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EVRYSDI Risdiplam 5 mg Tablet, 30-count — NDC 50242-0202-01 package photo
Label image from the product's FDA listing (DailyMed) — may show a different pack size or an older label revision.

EVRYSDI Risdiplam 5 mg Tablet, 30-count — NDC 50242-202-01 (Billing 50242-0202-01)

by Genentech, Inc. · 1 BOTTLE, PLASTIC in 1 CARTON / 30 TABLET in 1 BOTTLE, PLASTIC

This is a package of 30 tablets of EVRYSDI Risdiplam 5 mg Tablet from Genentech, Inc., marketed since Feb 2025 and currently FDA-listed. It is this product's only package size.

NDC 50242-0202-01
🏷️ FDA NDC (as labeled) 50242-202-01 billing pads the product segment with a zero
Rx only Brand On market Non-controlled ⇄ Compare with another NDC
🗂️ FDA directory synced Oct 8, 2026 · this listing last changed Oct 8, 2026 · sources: openFDA · FDA label (DailyMed) · FDA Orange & Purple Book · First Databank · CMS NADAC, ASP, Medicare & Medicaid · RxNorm
📋 All sources & update times →

This page is curated, coded, and maintained by

Compiled from public FDA, CMS, DailyMed, NADAC, and related drug data sources. Editorial policy

NDC database record

One package, one record: these facts belong to NDC 50242-202-01 alone.

Record
FDA NDC Directory package listing · Human prescription drug
Code segments
50242 labeler · 202 product · 01 package
Package marketed since
Feb 11, 2025
Sample package
No — commercial package
Listing certified through
Dec 31, 2027
Billing quantity
30 EA per package
Barcode (UPC-A, from the NDC)
3 5024220201 5
Medicaid fills, this package
2,121 prescriptions in the last four reported quarters
FDA record last changed
Oct 8, 2026

Identity & classification

Regulatory identifiers FDA, NLM and CMS codes for this package

FDA NDC (as labeled) 50242-202-01
Product NDC 50242-202
11-digit billing NDC 50242020201
NCPDP billing unit EA — each (per item)
Application # NDA219285
SPL Set ID eceb9a99-7191-4be5-87c3-0102707cf98e
Established class (EPC) Survival of Motor Neuron 2 Splicing Modifier; Survival of Motor Neuron 2 Splicing Modifie
Mechanism of action Multidrug and Toxin Extrusion Transporter 1 Inhibitors; Multidrug and Toxin Extrusion Transporter 2 K Inhibitors
Physiologic effect Increased Protein Synthesis
DEA schedule Non-controlled
Marketing category NDA
Marketing status On market
FDA listing status Listed (active directory)
Marketing start 2025-02-11
Route ORAL
Dosage form TABLET
Substance RISDIPLAM

Drug-database identifiers Medi-Span GPI and First Databank GCN / HICL / AHFS classification

GCN Seq No 087229
GCN 57085
HICL code 046765
Ingredient (HICL) Risdiplam
HIC1 code Z
Therapeutic class — broad (HIC1) Body As A Whole
HIC2 code Z1
Therapeutic class — intermediate (HIC2) Positive Effects On Wide-Spread Tissue
HIC3 code Z1T
Therapeutic class — specific (HIC3) Genetic D/O Tx - Smn Protein Deficiency Treatment
AHFS code 92:18.00.00
AHFS class Antisense Oligonucleotides
FDB label name EVRYSDI 5 MG TABLET
FDB brand name Evrysdi
Legend status F — Federal legend — prescription drug or device
Quick answers
  • GSN (GCN sequence number): 087229
  • GCN: 57085
  • HICL (First Databank): 046765
  • AHFS class code: 92:18.00.00
Why two NDCs? The FDA registers this code as 50242-202-01 — a 5-3-2 layout, and that's what's printed on the package and shown on DailyMed. For insurance claims, every NDC is standardized to a uniform 11-digit 5-4-2 format by adding a zero to the product segment → 50242-0202-01. Same drug, same package — only the format differs.
Where does this data come from?
Identifiers from the FDA openFDA NDC Directory and Structured Product Labeling; RxCUI from RxNorm (NLM); GPI from Medi-Span; GCN / HIC / AHFS / legend from First Databank.

Clinical

Label name EVRYSDI 5 MG TABLET Ingredient Risdiplam
📖 What it is MedlinePlus · NLM

Risdiplam is used for the treatment of spinal muscular atropy (SMA; an inherited condition that affected the nerves and causes muscle weakness). Risdiplam is in a class of medications called survival of motor neuron 2 (SMN2) splicing modifier. It works by increasing the amount of a protein in the body's nervous system to improve muscle strength and movement.

Read the full MedlinePlus article ↗
Where does this data come from?
Plain-language summary from MedlinePlus (U.S. National Library of Medicine); supplement & herbal interactions and nutrient depletion data from the Natural Medicines database; our full guide is HelloPharmacist editorial content.

Pricing

A drug doesn't have one price. Each row is a different public payment system, and none is what you'd pay at the counter — that depends on your insurance. The ⓘ on each row explains what it measures.

Price systemPer eachPer package
Retail pharmacies payNADAC · weekly Not in the retail survey — common for institutional, discontinued, or low-volume packs.
Medicaid paysCMS SDUD · 12 mo $1,135.82 $34,074.50 / 30 tablets
Medicare drug plans payPart D · Q2 2026 $1,180.93 $35,427.86 / 30 tablets
Where does this data come from?
NADAC (National Average Drug Acquisition Cost) is the CMS weekly pharmacy-acquisition-cost survey — what pharmacies pay. ASP (Average Sales Price) is the CMS Medicare Part B drug-payment file, published quarterly. Medicaid pays is computed by us from CMS State Drug Utilization Data (total reimbursed ÷ units, trailing 12 months) — gross of rebates and inclusive of dispensing fees, so it reflects what Medicaid paid, not an acquisition cost. Medicare drug plans pay is the median negotiated point-of-sale unit cost across plans listing this NDC in the CMS quarterly Prescription Drug Plan pricing files, before rebates. The VA pays is the federal contract price (FSS, and the statutory Big 4 ceiling where listed) from the VA National Acquisition Center pharmaceutical price file. All are free public government data; each measures a different payer, so the figures are not directly comparable.

Packaging — all sizes for this product

Package NDCDescription Marketing startMarketing endStatus
50242-0202-01 You're viewing this Main listing 1 BOTTLE, PLASTIC in 1 CARTON / 30 TABLET in 1 BOTTLE, PLASTIC 2025-02-11 — Active

Therapeutic equivalents

ProductLabelerPackNADAC/unitTEStatusPrice vs. this
Evrysdi 5 mgthis 50242-0202-01 Genentech, 30 tablets — — FDA listed —
About this product: this is the brand-name version. We did not find an FDA-approved generic match for this exact strength, form and route.
Where does this data come from?
Equivalents are other NDCs of the same ingredient, form and route from the openFDA NDC Directory, ranked least-expensive-first by NADAC. Therapeutic-equivalence (AB) ratings come from the FDA Orange Book; biologics use the FDA Purple Book for biosimilar & interchangeable status.

Availability & generic status

🏛️
2025
First FDA approval
Feb 2025
📍
2026
Currently FDA-listed
1 year listed
🛡️
2041
Latest patent/protection listed
not a guaranteed launch date
🔒No FDA-approved generic found

We did not find an FDA-approved generic match for this exact strength, form and route. Patent/protection dates below may affect future generic timing.

🛡️ Latest patent/protection date listed: FDA patent/protection data lists protections through Apr 2041. This may affect when a full generic version becomes widely available, but it is not a guaranteed launch date.
📅 FDA approved Feb 11, 2025 RLD RS ⏳ ~14.5 yr to latest listed protection

Why the date isn’t exact: Generic timing can change because patents may be challenged, settled, licensed, added, removed, or worked around with a narrower label — and FDA approval does not always mean a pharmacy can get the generic today.

Patents & exclusivity — FDA Orange Book
US 12350273 — method of use (U-1943)
US 11534444 — method of use (U-1943)
US 9969754 — drug substance (U-1943)
US 11827646 — method of use (U-1943)
US 9586955 — drug substance
US 12122789 — drug substance
Exclusivity M-270
2025 2027 2029 2031 2033 2035 2037 2039 2041
Today
LOE
Substance patent Formulation patent Method-of-use patent Exclusivity Pediatric +6mo
🏛️FDA exclusivity
FDA-granted marketing protection. It’s separate from patents and may be shorter than patent protection.
🧪Product / substance patents
Patents covering the active ingredient, product, formulation, or related drug features.
🎯Method-of-use patents
Patents covering specific approved uses. These can sometimes be carved out with a “skinny label,” but not always.
🛈 What do these terms mean?
Patent
Legal protection listed in the Orange Book that may delay generic approval or launch. Issued by the U.S. Patent & Trademark Office.
Substance patent
Covers the active drug molecule itself — the hardest to design around. A generic generally can’t launch until it expires.
Formulation (product) patent
Covers a specific formulation or dosage form. A generic can sometimes work around it with a different formulation.
Method-of-use patent
A patent covering one specific approved use of the drug — not necessarily the whole molecule. A generic can sometimes launch with a “skinny label” that carves out the protected use and keeps the others.
Skinny label
A generic label that omits a still-patented use when the FDA allows it — letting a generic reach the market for the unprotected uses.
Exclusivity
FDA-granted marketing protection, separate from patents — e.g. 5-yr new chemical entity, 7-yr orphan drug, or a +6-month pediatric extension.
Paragraph IV
A generic applicant’s formal challenge to a listed patent. It can potentially lead to earlier generic entry, but often involves litigation or a settlement.
RLD / RS
Reference Listed Drug — the brand product the FDA uses as the reference for generic applications. Reference Standard — the product the FDA expects generics to compare against in bioequivalence testing.
TE / AB rating
FDA therapeutic-equivalence rating. An AB rating generally means the FDA considers a generic therapeutically equivalent to — and substitutable for — the brand.
LOE (loss of exclusivity)
The latest patent or exclusivity currently listed — the loss-of-exclusivity / latest-listed-protection date shown on this page. Paragraph-IV challenges and settlements can move the real date earlier; FDA approval and a manufacturer’s decision to market can move it later.

Built from the FDA Orange Book. The bars above are scaled to each protection’s expiry; the red LOE marker is the last one to lapse.

Listed patents (6)
PatentTypeUse codeExpires
US 12350273 ↗ Method of use U-1943 Oct 1, 2038
US 11534444 ↗ Method of use U-1943 Oct 4, 2038
US 9969754 ↗ Drug substance U-1943 May 11, 2035
US 11827646 ↗ Method of use U-1943 Jan 25, 2036
US 9586955 ↗ Drug substance — Feb 8, 2033
US 12122789 ↗ Drug substance — Apr 15, 2041
FDA exclusivity
CodeWhat it grantsExpires
M-270New indication / labeling change (3-year)Oct 3, 2026
Common questions
Is there a generic version of EVRYSDI 5 MG TABLET?
No FDA-approved generic equivalent is currently listed in the FDA Orange Book for EVRYSDI 5 MG TABLET. Based on the patents and exclusivity currently listed, the Orange Book estimate is that full-label generic entry may be delayed until Apr 2041 — an estimate, not a guaranteed launch date.
The FDA approved a generic — why can’t I get it at my pharmacy yet?
FDA approval and pharmacy availability are two different things. The FDA can approve a generic years before it actually reaches pharmacies, because the brand company may still hold patents or have a settlement that delays the launch. A manufacturer also has to choose to make and sell it, and have supply ready. So a drug can be “FDA-approved generic exists” and still be brand-only at the counter today.
Why do different websites show different generic release dates?
Generic availability is not based on one single date. Some sources use the first exclusivity expiration, some use the last product patent, and others use the latest method-of-use patent. Patent challenges, settlements, licenses, and label carve-outs can also change the real-world launch date. This page shows the underlying Orange Book dates so you can see why estimates may differ.
What does “FDA listed” mean?
It means the product appears in the FDA’s official NDC directory. That’s a good sign a product exists and is intended for the U.S. market, but on its own it does not confirm a pharmacy can fill it today. Where we have recent retail pricing data (NADAC) for a product, we label it “Availability likely” instead.
What does a patent or protection date mean here?
It’s the latest date currently listed in the FDA Orange Book for a patent or exclusivity on the brand product. It can affect when a full generic version becomes widely available — but it is not a guaranteed generic launch date. Generics sometimes arrive earlier (through a settlement or patent challenge) or later (a manufacturer still has to make and sell one).
What does “current Orange Book estimate” mean?
It means we are using the latest patent and exclusivity dates currently listed in the FDA Orange Book. It is not a guaranteed launch date.
Can a generic come out before the last patent expires?
Sometimes. A generic company may challenge a patent, settle with the brand manufacturer, receive a license, or obtain approval with a narrower label that avoids a patented use. In other cases, the last listed protection may delay full-label generic competition.
Can a generic come out after the listed dates?
Yes. Even after patents or exclusivity expire, a generic still needs FDA approval and a manufacturer must choose to market it. Supply, litigation, business decisions, or regulatory issues can delay actual availability.
What is the difference between patents and exclusivity?
Patents are legal protections usually issued by the U.S. Patent and Trademark Office. FDA exclusivity is marketing protection granted by the FDA. They are separate, and either one can affect generic timing.
Why are there multiple patent dates?
One drug can have several patents covering different things: the active ingredient, a formulation, a manufacturing process, or a specific approved use. That is why a page may show several expiration dates instead of one simple generic date.
Built from FDA Orange Book patent and exclusivity data. Dates are refreshed from public FDA data when available; the marker is max(latest patent expiry, latest exclusivity expiry). Paragraph-IV settlements and first-filer 180-day exclusivity can shift the real date; a method-of-use patent may allow an earlier skinny-label generic for non-protected indications. Generic launch timing is an estimate, not a guarantee.
Where does this data come from?
Patents and exclusivity from the FDA Orange Book (small-molecule drugs), refreshed from public FDA data. Generic launch timing is an estimate, not a guarantee.

What it looks like

Color yellow
ShapeRound
ImprintEVR
Size7 mm
ScoringNot scored
FlavorStrawberry
One label can cover several strengths, so colors may be combined — always confirm a loose pill against the dispensed prescription label or a pharmacist.
Where does this data come from?
Physical description (imprint, shape, color, scoring, coating) from this product’s FDA Structured Product Labeling (SPL), mirrored from DailyMed / openFDA.

Inactive Ingredients / Excipients

Inactive ingredients, also called excipients, are components of the drug product other than the active ingredient. They may include fillers, dyes, coatings, preservatives, flavors, or other formulation ingredients.

💡 Tap an ingredient (hover on desktop) to see what it is and why it’s used.

  • 7.5 mg UNII 2S7830E561
    Crospovidone is a synthetic polymer derived from povidone. It acts as a disintegrant, helping the tablet or capsule break apart quickly in the stomach so the active ingredient can be absorbed.
  • 39.6 mg UNII 3OWL53L36A
    A natural sugar alcohol derived from seaweed or synthesized in the lab. It's used as a filler to add bulk, a sweetener in sugar-free formulas, and a disintegrant to help tablets break apart in the stomach.
  • 63.5 mg UNII OP1R32D61U
    Microcrystalline cellulose is a purified form of cellulose, a natural fiber from plant sources. It acts as a binder and filler in tablets and capsules, helping hold ingredients together and give the medicine its shape and size.
  • 0.5 mg UNII G2M7P15E5P
    Polyethylene glycol 3350 is a synthetic polymer used as a solvent, humectant, and thickening agent in medicines. It helps dissolve other ingredients, retain moisture in the product, and achieve the desired consistency.
  • 1 mg UNII 532B59J990
    Polyvinyl alcohol is a synthetic polymer made from plant-derived materials. It's used as a binder to hold ingredients together, a film-former in coatings, and a thickener in liquid formulations.
  • 2.5 mg UNII ETJ7Z6XBU4
    Silicon dioxide is a naturally occurring mineral used as a glidant and anti-caking agent. It helps powder ingredients flow smoothly and prevents clumping during manufacturing and storage.
  • 3.7 mg UNII 7CV7WJK4UI
    Sodium stearyl fumarate is a synthetic compound made from stearyl alcohol and fumaric acid. It acts as a lubricant and glidant in tablets and capsules, helping ingredients flow smoothly during manufacturing and preventing sticking.
  • 0.3 mg UNII 7SEV7J4R1U
    A powder made from a naturally occurring mineral. In medicines, talc works as a glidant and anti-caking agent, helping tablets and capsules flow smoothly during manufacturing and preventing clumping.
  • 3 mg UNII W4888I119H
    Tartaric acid is a natural organic acid found in grapes and tamarinds. In medicines, it works as a buffer to control acidity, an antioxidant to prevent spoilage, and sometimes a flavoring or binding agent.
  • 0.6 mg UNII 15FIX9V2JP
    Titanium dioxide is a bright white mineral powder commonly used as a colorant and opacifying agent. It makes pills and tablets white or lighter in color and helps make coatings non-transparent.

10 inactive ingredients listed in the exact product block matched to this NDC.

Where does this data come from?
Data sourced from official FDA Structured Product Labeling (SPL) via DailyMed — ingredient classCode="IACT" elements from the exact product block matched by this NDC. Label-section narrative from DailyMed / the openFDA label index is shown separately when available.

Inactive ingredient FAQ

Are inactive ingredients the same for every manufacturer?
No. Inactive ingredients can differ by manufacturer, dosage form, strength, and package / product version.
Why might an inactive ingredient be missing?
Some SPLs do not provide a complete structured inactive-ingredient list, and older or unusual labels may only include the information in narrative text.
Can inactive ingredients matter?
Yes. They can matter for allergies, intolerances, dyes, gluten / lactose concerns, preservatives, and formulation differences — but confirm with a pharmacist or the manufacturer when it’s clinically important.

Manufacturer & labeler

LabelerGenentech, Inc.
Application holderGENENTECH INC
FDA applicationNDA219285 (NDA)
Labeler code50242
First marketedFeb 2025
Product typeHuman Prescription Drug
Portfolio93 products on file

More NDCs from Genentech, Inc. labeler code 50242

The labeler markets the product; the application holder owns the FDA approval. They’re often the same company but can differ (e.g. a repackager or an authorized generic). A mailing address / phone appears here when the manufacturer includes it in the product’s FDA label (not all do).
Where does this data come from?
Labeler, application holder and registered establishment from the FDA openFDA NDC Directory and Drugs@FDA; address/contact from the product’s FDA label.

Full prescribing information FDA SPL

The complete FDA label for this product — the official prescribing information, verbatim, section by section. Very long sections are excerpted here and marked; the full text is on DailyMed (linked in the sources below). Jump with a chip, search within the label, or expand everything.
🎯 Indications and Usage ⏱️ Dosage and Administration 💊 Dosage Forms and Strengths ⛔ Contraindications 🤒 Adverse Reactions 🔄 Drug Interactions 👥 Use in Specific Populations 🤰 Pregnancy 🧒 Pediatric Use 🧓 Geriatric Use 🧬 Clinical Pharmacology 🧬 Mechanism of Action 📦 How Supplied / Storage and Handling 📦 Storage and Handling 📋 Description 💬 Information for Patients 🧬 Pharmacokinetics 🧬 Pharmacodynamics 🔬 Clinical Studies 🧪 Nonclinical Toxicology 📄 Carcinogenesis, Mutagenesis, Impairment of Fertility 📄 Patient Package Insert 📖 Instructions for Use 📄 Recent Major Changes 📄 Package Label / Principal Display Panel
🎯 Indications and Usage 48 words ▾

1 INDICATIONS AND USAGE EVRYSDI is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. EVRYSDI is a survival of motor neuron 2 (SMN2) splicing modifier indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. ( 1 )

⏱️ Dosage and Administration ~3 min read ▾

2 DOSAGE AND ADMINISTRATION Administer once daily with or without food per the table below ( 2.1 ): Age and Body Weight Recommended Daily Dosage Dosage Form Less than 2 months of age 0.15 mg/kg EVRYSDI for Oral Solution 2 months to less than 2 years of age 0.2 mg/kg 2 years of age and older weighing less than 20 kg 0.25 mg/kg 2 years of age and older weighing 20 kg or more 5 mg EVRYSDI for Oral Solution or EVRYSDI Tablet Swallow EVRYSDI tablet whole with water or dispersed in non-chlorinated drinking water (e.g., filtered water).

( 2.2 ) Administer EVRYSDI for oral solution with the provided oral syringe. ( 2.2 ) EVRYSDI for oral solution must be constituted by a healthcare provider prior to dispensing. ( 2.4 ) See Full Prescribing Information for important preparation and administration instructions.

( 2.2 , 2.4 )

2.1Dosing Information EVRYSDI is administered orally once daily with or without food at approximately the same time each day. The recommended dosage is determined by age and body weight (see Table 1 ). EVRYSDI tablets are available for patients prescribed the 5 mg dose.

Table 1 Adult and Pediatric Dosing Regimen by Age and Body Weight Age and Body Weight Recommended Daily Dosage Dosage Form Less than 2 months of age 0.15 mg/kg EVRYSDI for Oral Solution 2 months to less than 2 years of age 0.2 mg/kg 2 years of age and older weighing less than 20 kg 0.25 mg/kg 2 years of age and older weighing 20 kg or more 5 mg EVRYSDI for Oral Solution or EVRYSDI Tablet

2.2Important Administration Instructions It is recommended that a healthcare provider discuss with the patient or caregiver how to prepare the prescribed daily dose prior to administration of the first dose [see Instructions for Use for EVRYSDI for Oral Solution and EVRYSDI Tablets and Patient Information ]. EVRYSDI for Oral Solution In infants who are breastfed, EVRYSDI for oral solution can be administered before or after breastfeeding. EVRYSDI cannot be mixed with formula or milk.

Instruct patients or caregivers to administer the dose using the reusable oral syringe provided. EVRYSDI for oral solution must be taken immediately after it is drawn up into the oral syringe. If EVRYSDI is not taken within 5 minutes, EVRYSDI should be discarded from the oral syringe, and a new dose should be prepared.

Instruct patients to drink water after taking EVRYSDI for oral solution to ensure the drug has been completely swallowed. EVRYSDI for oral solution can be administered via a nasogastric or gastrostomy tube. The tube should be flushed with water after delivering EVRYSDI for oral solution [see Instructions for Use ].

EVRYSDI Tablets Swallow EVRYSDI tablets whole with water. Do not chew, cut, or crush the tablets. Alternatively, the EVRYSDI tablet can also be dispersed in one teaspoon (5 mL) of room temperature non-chlorinated drinking water (e.g., filtered water).

EVRYSDI tablets must not be dispersed in any liquid other than non-chlorinated drinking water. Do not expose the prepared dispersion to sunlight. Swirl the small cup gently for up to 3 minutes until fully mixed (though some particles will remain).

Administer the dispersed tablet immediately. To ensure no particles are left in the small cup, refill it with at least one tablespoon (15 mL) of non-chlorinated drinking water, swirl, and administer immediately again. EVRYSDI must be taken immediately after it is dispersed in non-chlorinated drinking water.

Discard the prepared dispersion if it is not used within 10 minutes of adding non-chlorinated drinking water. The dispersed EVRYSDI tablet can be administered via a nasogastric or gastrostomy tube that is 8 French or higher. Flush the tube with the non-chlorinated drinking water [at least one tablespoon (15 mL)] used to rinse the dispersion cup [see Instructions for Use ].

2.3Missed Dose If a dose of EVRYSDI is missed, EVRYSDI should be administered as soon as possible if still within 6 hours of the missed dose, and the usual dosing schedule can be resumed on the ne… [Excerpted — this section continues on DailyMed.]

💊 Dosage Forms and Strengths 91 words ▾

3 DOSAGE FORMS AND STRENGTHS EVRYSDI for oral solution: 60 mg as a light yellow, pale yellow, yellow, greyish yellow, greenish yellow, or light green powder for constitution. Following constitution, the volume of the greenish yellow to yellow solution is 80 mL, providing 60 mg/80 mL (0.75 mg/mL) risdiplam. EVRYSDI tablet: 5 mg as a pale yellow film-coated tablet, round and curved, with EVR debossed on one side.

For Oral Solution: 60 mg of risdiplam as a powder for constitution to provide 0.75 mg/mL solution. ( 3 ) Tablets: 5 mg

⛔ Contraindications 7 words ▾

4 CONTRAINDICATIONS None. None. ( 4 )

🤒 Adverse Reactions ~3 min read ▾

6 ADVERSE REACTIONS The most common adverse reactions in later-onset SMA (incidence at least 10% of patients treated with EVRYSDI and more frequent than control) were fever, diarrhea, and rash. ( 6.1 ) The most common adverse reactions in infantile-onset SMA were similar to those observed in later-onset SMA patients. Additionally, adverse reactions with an incidence of at least 10% were upper respiratory tract infection, lower respiratory tract infection, constipation, vomiting, and cough.

( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Genentech at 1-888-835-2555 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

6.1Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in clinical trials of another drug and may not reflect the rates observed in practice. In clinical trials including patients with infantile-onset SMA, later-onset SMA, and pre-symptomatic SMA, a total of 491 patients (51% female, 74% Caucasian) were exposed to EVRYSDI for up to a median duration of 48.1 months (range: 0.6 to 63.4 months), with 231 patients receiving treatment for more than 24 months.

At the time of first EVRYSDI dose, 90 (18%) patients were 18 years and older, 119 (24%) were 12 years to less than 18 years, 189 (39%) were 2 years to less than 12 years, 67 (14%) 2 months to less than 2 years, and 26 (5%) were less than 2 months. Clinical Trial in Later-Onset SMA The safety of EVRYSDI for later-onset SMA is based on data from a randomized, double-blinded, placebo-controlled study (Study 2 Part 2) in patients with SMA Type 2 or 3 (n = 180) [see Clinical Studies (14.2) ]. The patient population in Study 2 Part 2 ranged in age from 2 to 25 years at the time of the first dose.

The most common adverse reactions (reported in at least 10% of patients treated with EVRYSDI and at an incidence greater than on placebo) in Study 2 Part 2 were fever, diarrhea, and rash. Table 2 lists the adverse reactions that occurred in at least 5% of patients treated with EVRYSDI and at an incidence ≥ 5% greater than on placebo in Study 2 Part 2. Table 2 Adverse Reactions Reported in ≥ 5% of Patients Treated with EVRYSDI and with an Incidence ≥ 5% Greater Than on Placebo in Study 2 Part 2 Adverse Reaction EVRYSDI (N = 120) % Placebo (N = 60) % Fever Includes pyrexia and hyperpyrexia.

22 17 Diarrhea 17 8 Rash Includes rash, erythema, rash maculo-papular, rash erythematous, rash papular, dermatitis allergic, and folliculitis. 17 2 Mouth and aphthous ulcers 7 0 Arthralgia 5 0 Urinary tract infection Includes urinary tract infection and cystitis. 5 0 Clinical Trial in Infantile-Onset SMA The safety of EVRYSDI therapy for infantile-onset SMA is based on data from an open-label study in 62 patients (Study 1) [see Clinical Studies (14.1) ].

The patient population ranged in age from 2 to 7 months at the time of the first EVRYSDI dose (weight range: 4.1 to 10.6 kg). The most frequent adverse reactions reported in infantile-onset SMA patients treated with EVRYSDI in Study 1 were similar to those observed in later-onset SMA patients in Study 2. Additionally, the following adverse reactions reported in ≥ 10% of patients were: upper respiratory tract infection (including nasopharyngitis, rhinitis), lower respiratory tract infection (including pneumonia, bronchitis), constipation, vomiting, and cough.

Clinical Trial in Pre-Symptomatic SMA The safety of EVRYSDI therapy for pre-symptomatic SMA is based on data from an open-label, single-arm study in 26 patients (Study 3) [see Clinical Studies (14.3) ] . The patient population ranged in age from 16 to 41 days at the time of the first dose (weight range: 3.1 to 5.7 kg). The safety profile of EVRYSDI in pre-symptomatic patients in Study 3 is consistent with the safety profile for symptomatic SMA patients treated with EVRYSDI in clinical trials.

🔄 Drug Interactions 93 words ▾

7 DRUG INTERACTIONS Avoid coadministration with drugs that are substrates of multidrug and toxin extrusion (MATE) transporters. ( 7.1 )

7.1Effect of EVRYSDI on Substrates of Multidrug and Toxin Extrusion (MATE) Protein Transporters Based on in vitro data, EVRYSDI may increase plasma concentrations of drugs eliminated via MATE1 or MATE2-K [see Clinical Pharmacology (12.3) ] , such as metformin. Avoid coadministration of EVRYSDI with MATE substrates. If coadministration cannot be avoided, monitor for drug-related toxicities and consider dosage reduction of the coadministered drug (based on the labeling of that drug) if needed.

👥 Use in Specific Populations ~3 min read ▾

8 USE IN SPECIFIC POPULATIONS Pregnancy: Based on animal data, may cause fetal harm. ( 8.1 )

8.1Pregnancy Pregnancy Exposure Registry There is a pregnancy exposure registry that monitors pregnancy and fetal/neonatal/infant outcomes in women exposed to EVRYSDI during pregnancy. Physicians are encouraged to register patients and pregnant women are encouraged to register themselves by calling 1-833-760-1098 or visiting https://www.evrysdipregnancyregistry.com. Risk Summary There are no adequate data on the developmental risk associated with the use of EVRYSDI in pregnant women.

In animal studies, administration of risdiplam during pregnancy or throughout pregnancy and lactation resulted in adverse effects on development (embryofetal mortality, malformations, decreased fetal body weights, and reproductive impairment in offspring) at or above clinically relevant drug exposures [see Data ]. The estimated background risk of major birth defects and miscarriage for the indicated populations is unknown. In the U.S. general population, the estimated background risk of major birth defect and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively.

Based on animal data, advise pregnant women of the potential risk to the fetus. Data Animal Data Oral administration of risdiplam (0, 1, 3, or 7.5 mg/kg/day) to pregnant rats throughout organogenesis resulted in decreased fetal body weights and increased incidences of fetal structural variations at the highest dose tested, which was not associated with maternal toxicity. The no-effect level for adverse effects on embryofetal development (3 mg/kg/day) was associated with maternal plasma exposure (AUC) approximately 2 times that in humans at the maximum recommended human dose (MRHD) of 5 mg.

Oral administration of risdiplam (0, 1, 4, or 12 mg/kg/day) to pregnant rabbits throughout organogenesis resulted in embryofetal mortality, fetal malformations (hydrocephaly), and structural variations at the highest dose tested, which was associated with maternal toxicity. The no-effect dose for adverse effects on embryofetal development (4 mg/kg/day) was associated with maternal plasma exposure (AUC) approximately 4 times that in humans at the MRHD. When risdiplam (0, 0.75, 1.5, or 3 mg/kg/day) was orally administered to rats throughout pregnancy and lactation, gestation was prolonged in the dams, and delayed sexual maturation (vaginal opening) and impaired reproductive function (decreased numbers of corpora lutea, implantation sites, and live embryos) were observed in female offspring at the highest dose.

The no-effect dose for adverse effects on pre- and postnatal development in rats (1.5 mg/kg/day) was associated with maternal plasma exposure (AUC) similar to that in humans at the MRHD.

8.2Lactation Risk Summary There are no data on the presence of risdiplam in human milk, the effects on the breastfed infant, or the effects on milk production. Risdiplam was excreted in the milk of lactating rats orally administered risdiplam. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for EVRYSDI and any potential adverse effects on the breastfed infant from EVRYSDI or from the underlying maternal condition.

8.3Females and Males of Reproductive Potential Studies of risdiplam in juvenile and adult rats and in monkeys demonstrated adverse effects on the reproductive organs, including germ cells, in males at clinically relevant plasma exposures [see Use in Specific Populations (8.4) and Nonclinical Toxicology (13.1) ] . Pregnancy Testing Pregnancy testing is recommended for females of reproductive potential prior to initiating EVRYSDI [see Use in Specific Populations (8.1) ] . Contraception EVRYSDI may cause embryofetal harm when administered to a pregnant woman [see Use in Specific Populations (8.1) ].

Female Patients Advise female patients of reproductive potential to use effective contraception during treatment with… [Excerpted — this section continues on DailyMed.]

🤰 Pregnancy ~2 min read ▾

8.1Pregnancy Pregnancy Exposure Registry There is a pregnancy exposure registry that monitors pregnancy and fetal/neonatal/infant outcomes in women exposed to EVRYSDI during pregnancy. Physicians are encouraged to register patients and pregnant women are encouraged to register themselves by calling 1-833-760-1098 or visiting https://www.evrysdipregnancyregistry.com. Risk Summary There are no adequate data on the developmental risk associated with the use of EVRYSDI in pregnant women.

In animal studies, administration of risdiplam during pregnancy or throughout pregnancy and lactation resulted in adverse effects on development (embryofetal mortality, malformations, decreased fetal body weights, and reproductive impairment in offspring) at or above clinically relevant drug exposures [see Data ]. The estimated background risk of major birth defects and miscarriage for the indicated populations is unknown. In the U.S. general population, the estimated background risk of major birth defect and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively.

Based on animal data, advise pregnant women of the potential risk to the fetus. Data Animal Data Oral administration of risdiplam (0, 1, 3, or 7.5 mg/kg/day) to pregnant rats throughout organogenesis resulted in decreased fetal body weights and increased incidences of fetal structural variations at the highest dose tested, which was not associated with maternal toxicity. The no-effect level for adverse effects on embryofetal development (3 mg/kg/day) was associated with maternal plasma exposure (AUC) approximately 2 times that in humans at the maximum recommended human dose (MRHD) of 5 mg.

Oral administration of risdiplam (0, 1, 4, or 12 mg/kg/day) to pregnant rabbits throughout organogenesis resulted in embryofetal mortality, fetal malformations (hydrocephaly), and structural variations at the highest dose tested, which was associated with maternal toxicity. The no-effect dose for adverse effects on embryofetal development (4 mg/kg/day) was associated with maternal plasma exposure (AUC) approximately 4 times that in humans at the MRHD. When risdiplam (0, 0.75, 1.5, or 3 mg/kg/day) was orally administered to rats throughout pregnancy and lactation, gestation was prolonged in the dams, and delayed sexual maturation (vaginal opening) and impaired reproductive function (decreased numbers of corpora lutea, implantation sites, and live embryos) were observed in female offspring at the highest dose.

The no-effect dose for adverse effects on pre- and postnatal development in rats (1.5 mg/kg/day) was associated with maternal plasma exposure (AUC) similar to that in humans at the MRHD.

🧒 Pediatric Use ~2 min read ▾

8.4Pediatric Use The safety and effectiveness of EVRYSDI in pediatric patients (neonates and older) have been established . Use of EVRYSDI for SMA is supported by evidence from adequate and well-controlled studies of EVRYSDI in patients 2 months of age and older with SMA. Use of EVRYSDI for SMA in patients 2 months of age and younger is supported by pharmacokinetic and safety data from pediatric patients 16 days and older, and pharmacokinetic modeling and simulation to identify the dosing regimen [see Clinical Pharmacology (12.3) and Clinical Studies (14) ] .

Juvenile Animal Toxicity Data Oral administration of risdiplam (0, 0.75, 1.5, 2.5 mg/kg/day) to young rats from postnatal day (PND) 4 through PND 31 resulted in decreased growth (body weight, tibia length) and delayed sexual maturation in males at the mid and high dose. The skeletal and body weight deficits persisted after cessation of dosing. Ophthalmic changes consisting of vacuoles in the anterior vitreous were seen at the high dose.

Decreases in absolute B lymphocyte counts were observed at all doses after cessation of dosing. Decreases in testis and epididymis weights, which correlated with degeneration of the seminiferous epithelium in the testis, occurred at the mid and high doses; the histopathology findings were reversible, but organ weight persisted after cessation of dosing. Impaired female reproductive performance (decreased mating index, fertility index, and conception rate) was observed at the high dose.

A no-effect dose for adverse developmental effects on preweaning rats was not identified. The lowest dose tested (0.75 mg/kg/day) was associated with plasma exposures (AUC) lower than that in humans at the maximum recommended human dose (MRHD) of 5 mg/day. Oral administration of risdiplam (0, 1, 3, or 7.5 mg/kg/day) to young rats from PND 22 through PND 112 produced a marked increase in micronuclei in the bone marrow, male reproductive organ histopathology (degeneration/necrosis of the seminiferous tubule epithelium, oligo/aspermia in the epididymis, spermatic granulomas), and adverse effects on sperm parameters (decreased sperm concentration and motility, increased sperm morphology abnormalities) at the highest dose tested.

Increases in T lymphocytes (total, helper, and cytotoxic) were observed at the mid and high doses. The reproductive and immune effects persisted after cessation of dosing. The no-effect dose (1 mg/kg/day) for adverse effects on postweaning juvenile rats was associated with plasma exposures (AUC) lower than that in humans at the MRHD.

🧓 Geriatric Use 26 words ▾

8.5Geriatric Use Clinical studies of EVRYSDI did not include patients aged 65 years and older to determine whether they respond differently from younger adult patients.

🧬 Clinical Pharmacology ~3 min read ▾

12 CLINICAL PHARMACOLOGY

12.1Mechanism of Action Risdiplam is a survival of motor neuron 2 (SMN2) splicing modifier designed to treat patients with spinal muscular atrophy (SMA) caused by mutations in chromosome 5q that lead to SMN protein deficiency. Using in vitro assays and studies in transgenic animal models of SMA, risdiplam was shown to increase exon 7 inclusion in SMN2 messenger ribonucleic acid (mRNA) transcripts and production of full-length SMN protein in the brain. In vitro and in vivo data indicate that risdiplam may cause alternative splicing of additional genes, including FOXM1 and MADD.

FOXM1 and MADD are thought to be involved in cell cycle regulation and apoptosis, respectively, and have been identified as possible contributors to adverse effects seen in animals.

12.2Pharmacodynamics In clinical trials for infantile-onset SMA and later-onset SMA patients, EVRYSDI led to an increase in SMN protein with a greater than 2-fold median change from baseline within 4 weeks of treatment initiation across all SMA types studied. The increase was sustained throughout the treatment period (of at least 24 months). Cardiac Electrophysiology At the maximum recommended dose, clinically significant QTc interval prolongation was not observed.

12.3Pharmacokinetics Pharmacokinetics of EVRYSDI have been characterized in healthy adult subjects and in patients with SMA. After administration of EVRYSDI as an oral solution, pharmacokinetics of risdiplam were approximately linear between 0.6 and 18 mg in a single-ascending-dose study in healthy adult subjects, and between 0.02 and 0.25 mg/kg once daily in a multiple-ascending-dose study in patients with SMA. Following once-daily oral administration of risdiplam in healthy subjects, approximately 3-fold accumulation of peak plasma concentrations (C max ) and area under the plasma concentration-time curve (AUC 0-24h ) was observed.

Risdiplam exposures reach steady state 7 to 14 days after once daily administration. EVRYSDI tablet (swallowed whole or dispersed in water) demonstrated comparable bioavailability to EVRYSDI for oral solution in adult healthy volunteers under fasted and fed states. Absorption Following oral administration of risdiplam in fasted state, the median time to reach maximum plasma concentration (T max ) was 3.26 to 4 hours.

The T max was delayed by up to1 hour in fed state compared to that under fasted state. Effect of Food Food (high-fat, high calorie breakfast) had no relevant effect on the exposure of risdiplam. In the clinical efficacy studies (Study 1 and Study 2), risdiplam was administered with a morning meal or after breastfeeding.

Distribution The apparent volume of distribution at steady state is 190.4 L for a 31.3 kg patient. Risdiplam is predominantly bound to serum albumin, without any binding to alpha-1 acid glycoprotein, with a free fraction of 11%. Elimination The apparent clearance (CL/F) of risdiplam is

2.45L/h for a 31.3 kg patient. The terminal elimination half-life of risdiplam was approximately 50 hours in healthy adults. Metabolism Risdiplam is primarily metabolized by flavin monooxygenase 1 and 3 (FMO1 and FMO3) and also by CYPs 1A1, 2J2, 3A4, and 3A7.

Parent drug was the major component found in plasma, accounting for 83% of drug-related material in circulation. The pharmacologically-inactive metabolite M1 was identified as the major circulating metabolite. Excretion Following a dose of 18 mg, approximately 53% of the dose (14% unchanged risdiplam) was excreted in the feces and 28% in urine (8% unchanged risdiplam).

Specific Populations There were no clinically significant differences in the pharmacokinetics of EVRYSDI based on race or gender. Renal impairment is not expected to alter the exposures to risdiplam. The impact of geriatric age on the pharmacokinetics of EVRYSDI has not been studied.

Hepatic Impairment The pharmacokinetics and safety of risdiplam have been studied in subjects with mild or moderate hepatic impairment (as defined… [Excerpted — this section continues on DailyMed.]

🧬 Mechanism of Action 120 words ▾

12.1Mechanism of Action Risdiplam is a survival of motor neuron 2 (SMN2) splicing modifier designed to treat patients with spinal muscular atrophy (SMA) caused by mutations in chromosome 5q that lead to SMN protein deficiency. Using in vitro assays and studies in transgenic animal models of SMA, risdiplam was shown to increase exon 7 inclusion in SMN2 messenger ribonucleic acid (mRNA) transcripts and production of full-length SMN protein in the brain. In vitro and in vivo data indicate that risdiplam may cause alternative splicing of additional genes, including FOXM1 and MADD.

FOXM1 and MADD are thought to be involved in cell cycle regulation and apoptosis, respectively, and have been identified as possible contributors to adverse effects seen in animals.

📦 How Supplied / Storage and Handling 214 words ▾

16 HOW SUPPLIED/STORAGE AND HANDLING

16.1EVRYSDI for Oral Solution How Supplied Each amber glass bottle of EVRYSDI for oral solution is packaged with a bottle adapter, two 1 mL reusable oral syringes, two 6 mL reusable oral syringes, and one 12 mL reusable oral syringe. EVRYSDI for oral solution is a light yellow, pale yellow, yellow, greyish yellow, greenish yellow, or light green powder. Each bottle contains 60 mg of risdiplam (NDC 50242-175-07).

Storage and Handling Store the dry powder at 20°C to 25°C (68°F to 77°F), excursions permitted between 15°C to 30°C (59°F to 86°F) [see USP controlled room temperature] . Keep in the original carton. Keep the constituted oral solution of EVRYSDI in the original amber bottle to protect from light.

Store in a refrigerator at 2°C to 8°C (36°F to 46°F) [see Dosage and Administration (2.4) ].

16.2EVRYSDI Tablets How Supplied Pale yellow film-coated tablet, round and curved, with EVR debossed on one side; available in HDPE bottles of 30 tablets with a child-resistant cap (NDC 50242-202-01). Storage and Handling Store at 20°C to 25°C (68°F to 77°F); excursions permitted between 15°C to 30°C (59°F to 86°F) [see USP Controlled Room Temperature] . Keep in the original bottle. Keep the bottle tightly closed in order to protect from moisture.

📦 Storage and Handling 42 words ▾

Storage and Handling Store at 20°C to 25°C (68°F to 77°F); excursions permitted between 15°C to 30°C (59°F to 86°F) [see USP Controlled Room Temperature] . Keep in the original bottle. Keep the bottle tightly closed in order to protect from moisture.

📋 Description 206 words ▾

11 DESCRIPTION EVRYSDI for oral solution and EVRYSDI tablets for oral use contain risdiplam, which is a survival of motor neuron 2 (SMN2)-directed RNA splicing modifier. The chemical name of risdiplam is 7-(4,7-diazaspiro[2.5]octan-7-yl)-2-(2,8 dimethylimidazo[1,2-b]pyridazin-6-yl)pyrido-4H-[1,2-a]pyrimidin-4-one. Risdiplam has a molecular weight of 401.46 g/mol.

Risdiplam demonstrates pH-dependent aqueous solubility; the greatest solubility is at low pH, and solubility decreases with increasing pH. Risdiplam has a pKa 1 of 3.78 (base) and pKa 2 of 6.62 (base). The molecular formula of risdiplam is C 22 H 23 N 7 O and the chemical structure is shown below.

EVRYSDI for oral solution is supplied as a powder in an amber glass bottle. Each bottle contains 60 mg of risdiplam. The inactive ingredients of EVRYSDI are: ascorbic acid, disodium edetate dihydrate, isomalt, mannitol, polyethylene glycol 6000, sodium benzoate, strawberry flavor, sucralose, and tartaric acid.

The powder is constituted with purified water to yield 60 mg/80 mL (0.75 mg/mL) of risdiplam after constitution [see Dosage and Administration (2.4) ]. Each EVRYSDI tablet contains 5 mg of risdiplam. The inactive ingredients of EVRYSDI tablet are colloidal silicon dioxide, crospovidone, mannitol, microcrystalline cellulose, polyethylene glycol 3350, polyvinyl alcohol, sodium stearyl fumarate, strawberry flavor, talc, tartaric acid, titanium dioxide, and yellow iron oxide.

Chemical Structure

💬 Information for Patients ~2 min read ▾

17 PATIENT COUNSELING INFORMATION Advise the patient to read the FDA-approved patient labeling ( Patient Information and Instructions for Use ). Pregnancy and Fetal Risk Inform pregnant women and women of reproductive potential that, based on animal studies, EVRYSDI may cause fetal harm [see Use in Specific Populations (8.1) ]. Discuss with women of childbearing age whether they are pregnant, might be pregnant, or are trying to become pregnant.

Advise women of childbearing potential to use effective contraception during treatment with EVRYSDI and for at least 1 month after stopping EVRYSDI. Advise a female patient to immediately inform the prescriber if she is pregnant or planning to become pregnant [see Use in Specific Populations (8.3) ]. Pregnancy Registry Encourage patients to enroll in the EVRYSDI Pregnancy Registry if they become pregnant while taking EVRYSDI [see Use in Specific Populations (8.1) ] .

Potential Effects on Male Fertility Advise male patients that their fertility may be compromised while on treatment with EVRYSDI [see Use in Specific Populations (8.3) ]. Instructions for Preparation of Oral Solution Advise patients/caregivers to ensure that EVRYSDI oral solution is in liquid form when received from the pharmacy. Instruct patients/caregivers to take EVRYSDI oral solution with or without food or before or after breastfeeding at approximately the same time each day.

However, instruct caregivers not to mix EVRYSDI with formula or milk. Instruct patients/caregivers to take EVRYSDI oral solution immediately after it is drawn up into the reusable oral syringe [see Dosage and Administration (2.2) ]. Instructions for EVRYSDI Tablets Advise patients/caregivers to swallow EVRYSDI tablets whole with water.

Do not chew, cut, or crush the tablets. Alternatively, the tablet can be dispersed in one teaspoon (5 mL) of room temperature non-chlorinated drinking water (e.g., filtered water) and taken immediately. EVRYSDI tablets must not be dispersed in any liquid other than non-chlorinated drinking water.

Instruct the patient/caregivers that the dispersion must be administered within 10 minutes of adding non-chlorinated drinking water, or it must be discarded [see Dosage and Administration (2.2) ] . Advise patients/caregivers to wash their hands before and after preparing or taking EVRYSDI tablets. Advise patients/caregivers to avoid getting the dispersed tablet on their skin or in their eyes.

Advise patients/caregivers to wash the area with soap and water if the dispersed tablet gets on the skin. Advise patients/caregivers to rinse their eyes with water if the dispersed tablet gets in the eyes. Advise patients/caregivers to use a dry paper towel to dry the area if the dispersion is spilled and then clean with soap and water.

Advise patients/caregivers to throw the paper towel away in the trash and wash their hands with soap and water.

🧬 Pharmacokinetics ~3 min read ▾

12.3Pharmacokinetics Pharmacokinetics of EVRYSDI have been characterized in healthy adult subjects and in patients with SMA. After administration of EVRYSDI as an oral solution, pharmacokinetics of risdiplam were approximately linear between 0.6 and 18 mg in a single-ascending-dose study in healthy adult subjects, and between 0.02 and 0.25 mg/kg once daily in a multiple-ascending-dose study in patients with SMA. Following once-daily oral administration of risdiplam in healthy subjects, approximately 3-fold accumulation of peak plasma concentrations (C max ) and area under the plasma concentration-time curve (AUC 0-24h ) was observed.

Risdiplam exposures reach steady state 7 to 14 days after once daily administration. EVRYSDI tablet (swallowed whole or dispersed in water) demonstrated comparable bioavailability to EVRYSDI for oral solution in adult healthy volunteers under fasted and fed states. Absorption Following oral administration of risdiplam in fasted state, the median time to reach maximum plasma concentration (T max ) was 3.26 to 4 hours.

The T max was delayed by up to1 hour in fed state compared to that under fasted state. Effect of Food Food (high-fat, high calorie breakfast) had no relevant effect on the exposure of risdiplam. In the clinical efficacy studies (Study 1 and Study 2), risdiplam was administered with a morning meal or after breastfeeding.

Distribution The apparent volume of distribution at steady state is 190.4 L for a 31.3 kg patient. Risdiplam is predominantly bound to serum albumin, without any binding to alpha-1 acid glycoprotein, with a free fraction of 11%. Elimination The apparent clearance (CL/F) of risdiplam is

2.45L/h for a 31.3 kg patient. The terminal elimination half-life of risdiplam was approximately 50 hours in healthy adults. Metabolism Risdiplam is primarily metabolized by flavin monooxygenase 1 and 3 (FMO1 and FMO3) and also by CYPs 1A1, 2J2, 3A4, and 3A7.

Parent drug was the major component found in plasma, accounting for 83% of drug-related material in circulation. The pharmacologically-inactive metabolite M1 was identified as the major circulating metabolite. Excretion Following a dose of 18 mg, approximately 53% of the dose (14% unchanged risdiplam) was excreted in the feces and 28% in urine (8% unchanged risdiplam).

Specific Populations There were no clinically significant differences in the pharmacokinetics of EVRYSDI based on race or gender. Renal impairment is not expected to alter the exposures to risdiplam. The impact of geriatric age on the pharmacokinetics of EVRYSDI has not been studied.

Hepatic Impairment The pharmacokinetics and safety of risdiplam have been studied in subjects with mild or moderate hepatic impairment (as defined by Child-Pugh class A and B, respectively, n = 8 each) compared to subjects with normal hepatic function (n = 10). Following the administration of 5 mg EVRYSDI, the AUC inf and C max of risdiplam were approximately 20% and 5% lower, respectively, in subjects with mild hepatic impairment and were approximately 8% and 20% higher, respectively, in subjects with moderate hepatic impairment, versus matched healthy control subjects.

The magnitude of these changes is not considered to be clinically meaningful. The pharmacokinetics and safety in patients with severe hepatic impairment (Child-Pugh class C) have not been studied. Pediatric Patients Body weight and age were found to have significant effect on the pharmacokinetics of risdiplam.

The estimated exposure (mean AUC 0-24h ) in pre-symptomatic infants at the age of 1 to 2 months was 2090 ng.h/mL at the recommended dose of 0.15 mg/kg once daily. The estimated exposure for infantile-onset SMA patients (age 2 to 7 months at enrollment) at the recommended dose of 0.2 mg/kg once daily was 1930 ng.h/mL. The estimated exposure for later-onset SMA patients (2 to 25 years old at enrollment) at the recommended dose was 2070 ng.h/mL (0.25 mg/kg once daily for patients with a body weight < 20 kg and 5 mg o… [Excerpted — this section continues on DailyMed.]

🧬 Pharmacodynamics 68 words ▾

12.2Pharmacodynamics In clinical trials for infantile-onset SMA and later-onset SMA patients, EVRYSDI led to an increase in SMN protein with a greater than 2-fold median change from baseline within 4 weeks of treatment initiation across all SMA types studied. The increase was sustained throughout the treatment period (of at least 24 months). Cardiac Electrophysiology At the maximum recommended dose, clinically significant QTc interval prolongation was not observed.

🔬 Clinical Studies ~3 min read ▾

14 CLINICAL STUDIES The efficacy of EVRYSDI for the treatment of patients with infantile-onset, later-onset, and pre-symptomatic SMA was evaluated in three clinical studies, Study 1 (NCT02913482) and Study 2 (NCT02908685), and Study 3 (NCT03779334), respectively. The overall findings of these studies support the effectiveness of EVRYSDI in SMA pediatric and adult patients and appear to support the early initiation of treatment with EVRYSDI.

14.1Infantile-Onset SMA Study 1 was an open-label, 2-part study to investigate the efficacy, safety, pharmacokinetics, and pharmacodynamics of EVRYSDI for oral solution in patients with Type 1 SMA (symptom onset between 28 days and 3 months of age). All patients had genetic confirmation of homozygous deletion or compound heterozygosity predictive of loss of function of the SMN1 gene, and two SMN2 gene copies. Part 1 of Study 1 was designed as a dose-finding study.

Part 2 of Study 1 assessed the safety and efficacy of EVRYSDI at 0.20 mg/kg, the recommended dose determined in Part 1 [see Dosage and Administration (2.4) ] . Patients from Part 1 did not take part in Part 2. A total of 62 patients with symptomatic Type 1 SMA were enrolled in FIREFISH Part 1 (n = 21) and Part 2 (n = 41), of which 58 patients received the recommended dosage [see Dosage and Administration (2.1) ] .

The median age of onset of clinical signs and symptoms was 1.5 months (range: 0.9 to 3.0 months). The median age at enrollment was 5.6 months (range: 2.2 to 6.9 months), and the median time between onset of symptoms and the first dose was 3.7 months (range 1.0 to 6.0 months). Of these patients, 60% were female, 57% were Caucasian, and 29% were Asian.

The demographics and baseline disease characteristics were comparable between Part 1 and Part 2 of the study. Effectiveness was established based on the ability to sit without support for at least 5 seconds (as measured by Item 22 of the Bayley Scales of Infant and Toddler Development – Third Edition (BSID-III) gross motor scale) and on the basis of survival without permanent ventilation. Permanent ventilation was defined as requiring a tracheostomy or more than 21 consecutive days of either non-invasive ventilation (≥ 16 hours per day) or intubation, in the absence of an acute reversible event.

The primary endpoint was the proportion of patients with the ability to sit without support for at least 5 seconds (BSID-III gross motor scale, Item 22) after 12 months of treatment in Part 2; 29% of patients (n = 12/41) achieved this milestone. Other efficacy endpoints of EVRYSDI-treated patients in Study 1 (pooled Part 1 and Part 2) are shown in Table 3 . Table 3 Key Efficacy Results at Month 12 and Month 24 (Study 1, Parts 1 and Part 2) Efficacy Endpoints Proportion of Patients Parts 1 & 2 at Month 12 Proportion of Patients Parts 1 & 2 at Month 24 Motor Function and Development Milestones N = 58 Results were pooled from all patients who received the recommended dose of risdiplam (all patients in Part 2 and those in the high-dose cohort of Part 1; n = 58).

BSID-III, Item 22: sitting without support for at least 5 seconds 32.8% 60.3% Survival and Event-Free Survival N = 62 Results were pooled from all patients who received any dose of risdiplam in Part 1 and Part 2 (n = 62). Alive without Permanent Ventilation 87.1% 83.8% At Month 24, 40% (23/58) of patients who received the recommended dose achieved sitting without support for 30 seconds (BSID-III, Item 26). In addition at Month 24, patients continued to achieve additional motor milestones; 28% (16/58) of patients achieved a standing measure (16% [9/58] supporting weight and 12% [7/58] standing with support), as measured by Section 2 of the Hammersmith Infant Neurological Examination (HINE-2) which assesses motor milestones.

The proportion of patients alive without permanent ventilation (event-free survival) was 84% for all patients at Month 24 (Table 3). Out of 62 patients, 6 infants died (4 within the first 3 months following st… [Excerpted — this section continues on DailyMed.]

🧪 Nonclinical Toxicology ~3 min read ▾

13 NONCLINICAL TOXICOLOGY

13.1Carcinogenesis, Mutagenesis, Impairment of Fertility Carcinogenesis Risdiplam was not carcinogenic in Tg.rasH2 mice when administered at oral doses of up to 9 mg/kg/day for 26 weeks. In a 2-year carcinogenicity study in rats, oral administration of risdiplam (0, 0.3, 1, or 3 mg/kg/day) resulted in increased incidences of preputial gland squamous cell carcinomas in males and combined thyroid follicular cell adenomas and carcinomas in females at the highest dose tested. The higher dose not associated with an increase in tumors (1 mg/kg/day) was associated with plasma drug exposures (AUC) similar to that in humans at the maximum recommended human dose (MRHD) of 5 mg/day.

Mutagenesis Risdiplam was negative in an in vitro Ames assay. In an in vivo combined bone marrow micronucleus and comet assay in rat, risdiplam was clastogenic, as evidenced by an increase in micronuclei in bone marrow, but was negative in the comet assay. A pronounced increase in bone marrow micronuclei was also observed in toxicity studies in adult and juvenile rats [see Use in Specific Populations (8.4) ].

Impairment of Fertility Oral administration of risdiplam to rats for 4 (0, 1, 3, or 9 mg/kg/day) or 26 (0, 1, 3, or 7.5 mg/kg/day) weeks resulted in histopathological effects in the testis (degenerated spermatocytes, degeneration/atrophy of the seminiferous tubules) and epididymis (degeneration/necrosis of ductular epithelium) at the mid and/or high doses. At the high dose in the 26-week study, the testicular lesions persisted to the end of the recovery period, which corresponds, in rat, to approximately one spermatogenic cycle.

The no-effect dose for adverse reproductive system effects in adult male rats (1 mg/kg/day) was associated with plasma drug exposures (AUC) similar to that in humans at the maximum recommended human dose (MRHD) of 5 mg/day. Adverse effects of risdiplam on the testis could not be fully evaluated in the monkey because the majority of monkeys tested were sexually immature. However, oral administration of risdiplam (0, 2, 4, or 6 mg/kg/day) for 2 weeks resulted in histopathological changes in the testis (increases in multinucleate cells, germ cell degeneration) at the highest dose.

At the no-effect dose for testicular toxicity in monkeys, plasma exposures were approximately 3 times that in humans at the MRHD. Oral administration of risdiplam to postweaning juvenile rats resulted in male reproductive toxicity (degeneration/necrosis of the testis seminiferous epithelium with associated oligo/aspermia in the epididymis and abnormal sperm parameters). The no-effect dose for adverse reproductive effects in postweaning male juvenile rats was associated with plasma exposures approximately 4 times that in humans at the MRHD [see Use in Specific Populations (8.4) ] .

13.2Animal Toxicology and/or Pharmacology Retinal toxicity Risdiplam-induced functional and structural retinal abnormalities were seen in animal studies. In a 39-week toxicity study in monkeys, oral administration of risdiplam (0, 1.5, 3, or 7.5/5 mg/kg/day; high dose lowered after 4 weeks) produced functional abnormalities on the electroretinogram (ERG) in all mid- and high-dose animals at the earliest examination time (Week 20). These findings were associated with retinal degeneration, detected by optical coherence tomography (OCT), on Week 22, the first examination time.

The retinal degeneration, with peripheral photoreceptor loss, was irreversible. A no-effect dose for the retinal findings (1.5 mg/kg/day) was associated with plasma exposures (AUC) similar to that in humans at the maximum recommended human dose (MRHD) of 5 mg. Effect on Epithelial Tissues Oral administration of risdiplam to rats and monkeys resulted in histopathological changes in epithelium of the gastrointestinal (GI) tract (apoptosis/single cell necrosis), lamina propria (vacuolation), the exocrine pancreas (single cell necrosis), the skin, tongue, and larynx (parakeratosis/hyper… [Excerpted — this section continues on DailyMed.]

📄 Carcinogenesis, Mutagenesis, Impairment of Fertility ~2 min read ▾

13.1Carcinogenesis, Mutagenesis, Impairment of Fertility Carcinogenesis Risdiplam was not carcinogenic in Tg.rasH2 mice when administered at oral doses of up to 9 mg/kg/day for 26 weeks. In a 2-year carcinogenicity study in rats, oral administration of risdiplam (0, 0.3, 1, or 3 mg/kg/day) resulted in increased incidences of preputial gland squamous cell carcinomas in males and combined thyroid follicular cell adenomas and carcinomas in females at the highest dose tested. The higher dose not associated with an increase in tumors (1 mg/kg/day) was associated with plasma drug exposures (AUC) similar to that in humans at the maximum recommended human dose (MRHD) of 5 mg/day.

Mutagenesis Risdiplam was negative in an in vitro Ames assay. In an in vivo combined bone marrow micronucleus and comet assay in rat, risdiplam was clastogenic, as evidenced by an increase in micronuclei in bone marrow, but was negative in the comet assay. A pronounced increase in bone marrow micronuclei was also observed in toxicity studies in adult and juvenile rats [see Use in Specific Populations (8.4) ].

Impairment of Fertility Oral administration of risdiplam to rats for 4 (0, 1, 3, or 9 mg/kg/day) or 26 (0, 1, 3, or 7.5 mg/kg/day) weeks resulted in histopathological effects in the testis (degenerated spermatocytes, degeneration/atrophy of the seminiferous tubules) and epididymis (degeneration/necrosis of ductular epithelium) at the mid and/or high doses. At the high dose in the 26-week study, the testicular lesions persisted to the end of the recovery period, which corresponds, in rat, to approximately one spermatogenic cycle.

The no-effect dose for adverse reproductive system effects in adult male rats (1 mg/kg/day) was associated with plasma drug exposures (AUC) similar to that in humans at the maximum recommended human dose (MRHD) of 5 mg/day. Adverse effects of risdiplam on the testis could not be fully evaluated in the monkey because the majority of monkeys tested were sexually immature. However, oral administration of risdiplam (0, 2, 4, or 6 mg/kg/day) for 2 weeks resulted in histopathological changes in the testis (increases in multinucleate cells, germ cell degeneration) at the highest dose.

At the no-effect dose for testicular toxicity in monkeys, plasma exposures were approximately 3 times that in humans at the MRHD. Oral administration of risdiplam to postweaning juvenile rats resulted in male reproductive toxicity (degeneration/necrosis of the testis seminiferous epithelium with associated oligo/aspermia in the epididymis and abnormal sperm parameters). The no-effect dose for adverse reproductive effects in postweaning male juvenile rats was associated with plasma exposures approximately 4 times that in humans at the MRHD [see Use in Specific Populations (8.4) ] .

📄 Patient Package Insert ~3 min read ▾

This Patient Information has been approved by the U.S. Food and Drug Administration. Issued: 2/2026 Patient Information EVRYSDI ® [ev-RIZ-dee] (risdiplam) for oral solution EVRYSDI ® [ev-RIZ-dee] (risdiplam) tablets, for oral use What is EVRYSDI?

EVRYSDI is a prescription medicine used to treat spinal muscular atrophy (SMA) in children and adults. Before taking EVRYSDI, tell your healthcare provider about all of your medical conditions, including if you: are pregnant or plan to become pregnant. If you are pregnant, or are planning to become pregnant, ask your healthcare provider for advice before taking this medicine.

EVRYSDI may harm your unborn baby. are a woman who can become pregnant: Before you start your treatment with EVRYSDI, your healthcare provider may test you for pregnancy. Because EVRYSDI may harm your unborn baby, you and your healthcare provider will decide if taking EVRYSDI is right for you during this time. Talk to your healthcare provider about birth control methods that may be right for you.

Use birth control while on treatment and for at least 1 month after stopping EVRYSDI. Pregnancy Registry. There is a pregnancy registry for women who take EVRYSDI during pregnancy.

The purpose of this registry is to collect information about the health of the pregnant woman and her baby. If you are pregnant or become pregnant while receiving EVRYSDI, tell your healthcare provider right away. Talk to your healthcare provider about registering with the EVRYSDI Pregnancy Registry.

Your healthcare provider can enroll you in this registry or you can enroll by calling 1-833-760-1098 or visiting https://www.evrysdipregnancyregistry.com. are an adult male planning to have children: EVRYSDI may affect a man's ability to have children (fertility). If this is of concern to you, make sure to ask a healthcare provider for advice. are breastfeeding or plan to breastfeed. It is not known if EVRYSDI passes into breast milk and may harm your baby.

If you plan to breastfeed, discuss with your healthcare provider about the best way to feed your baby while on treatment with EVRYSDI. Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Keep a list of them to show your healthcare provider, including your pharmacist, when you get a new medicine.

How should I take EVRYSDI? For infants and children, your healthcare provider will determine the daily dose of EVRYSDI needed based on your child's age and weight. For adults, take 5 mg of EVRYSDI daily.

Your healthcare provider will either prescribe: EVRYSDI for oral solution Or EVRYSDI tablet Your healthcare provider will tell you how long you or your child needs to take EVRYSDI. Do not stop treatment with EVRYSDI unless your healthcare provider tells you to. Take EVRYSDI exactly as your healthcare provider tells you to take it.

Do not change the dose without talking to your healthcare provider. Avoid getting EVRYSDI on your skin or in your eyes. If it gets on your skin, wash the area with soap and water.

If it gets in your eyes, rinse your eyes with water. If you are taking EVRYSDI for oral solution: See the detailed Instructions for Use that comes with it for information on how to take or give EVRYSDI for oral solution. You should receive EVRYSDI from the pharmacy as a liquid that can be given by mouth or through a feeding tube.

The liquid solution is prepared by your pharmacist or other healthcare provider. If the medicine in the bottle is a powder, do not use it . Contact your pharmacist for a replacement.

Take EVRYSDI one time daily with or without a meal at about the same time each day. Drink water afterwards to make sure EVRYSDI has been completely swallowed. In infants who are breastfed, EVRYSDI can be given before or after breastfeeding.

Do not mix EVRYSDI with formula or milk. If you are unable to swallow and have a nasogastric (NG-tube) or gastrostomy tube (G-tube), EVRYSDI for oral solution… [Excerpted — this section continues on DailyMed.]

📖 Instructions for Use ~3 min read ▾

INSTRUCTIONS FOR USE EVRYSDI ® [ev-RIZ-dee] (risdiplam) for oral solution Please read and understand this Instructions for Use and the Patient Information leaflet before you start taking EVRYSDI for information about EVRYSDI and how to prepare and give EVRYSDI through an oral syringe, gastrostomy tube (G-tube), or nasogastric tube (NG-tube). If you have any questions about how to take EVRYSDI, contact your healthcare provider. EVRYSDI should come as a liquid in a bottle when you receive it from the pharmacy.

Do not take EVRYSDI and contact your pharmacist if the medicine in the bottle is a powder. Each EVRYSDI carton contains (see Figure A ): 1 Cap Figure A 1 Bottle adapter 1 EVRYSDI bottle 1 or 2 Reusable oral syringe(s) 1 Instructions for Use (not shown) 1 Prescribing Information and Patient Information (not shown) Reusable Oral Syringe Overview (see Figure B ) Figure B Important information about EVRYSDI Ask your healthcare provider to show you the correct oral syringe you should use and how to measure your prescribed daily dose.

Always use the reusable oral syringes that come with EVRYSDI to measure your prescribed daily dose. If your carton does not contain two identical syringes, contact your pharmacist. Always take EVRYSDI exactly as your healthcare provider tells you to take it.

Take EVRYSDI 1 time daily with or without a meal at approximately the same time each day. Do not take EVRYSDI if the bottle adapter is not in the bottle. If the bottle adapter is not in the bottle, contact your pharmacist.

Do not mix EVRYSDI into food or liquids. Do not mix EVRYSDI with formula or milk. Do not take EVRYSDI if the bottle or oral syringes are damaged.

Avoid getting EVRYSDI on your skin or in your eyes. If EVRYSDI gets on your skin, wash the area with soap and water. If EVRYSDI gets in your eyes, rinse your eyes with water.

If you spill EVRYSDI, dry the area with a dry paper towel and then clean with water. Throw away the paper towel in the trash and wash your hands well with soap and water. If there is not enough EVRYSDI left in the bottle for your prescribed dose, throw away (discard) the bottle with remaining EVRYSDI and used oral syringes according to your local requirements.

Use a new bottle of EVRYSDI to get your prescribed dose. Do not mix EVRYSDI from the new bottle with the bottle you are currently using. How to store EVRYSDI Store EVRYSDI in the refrigerator between 36°F to 46°F (2°C to 8°C).

Do not freeze. If necessary, EVRYSDI can be kept at room temperature up to 104°F (up to 40°C) for a combined total of 5 days. EVRYSDI can be removed from, and returned to, a refrigerator.

The total combined time out of refrigeration should not be more than 5 days. Throw away EVRYSDI if it has been kept at room temperature for more than 5 days. Store EVRYSDI in the original amber bottle in an upright position with the cap tightly closed.

Throw away (discard) any unused portion of EVRYSDI 64 days after mixed by the pharmacist (constitution) when stored in the refrigerator at 36°F to 46°F (2°C to 8°C). Please see the Discard After date written on the bottle label (see Figure C ). Ask your pharmacist for the Discard After date if it is not written on the bottle label.

Throw away any unused portion of EVRYSDI that has been kept above 104°F (40°C). Keep EVRYSDI, all medicines and syringes out of the reach of children. Figure C A) Preparing and withdrawing your dose How to prepare your dose of EVRYSDI Figure D Step A1 Remove the cap by pushing it down and then twisting the cap to the left (counterclockwise) (See Figure D ).

Do not throw away the cap. Figure E Step A2 Push the plunger of the oral syringe all the way down to remove any air in the oral syringe (See Figure E ). Figure F Step A3 Place the EVRYSDI bottle on a flat surface.

While keeping the bottle in an upright position, insert the syringe tip into the bottle adapter (See Figure F ). Figure G Step A4 Carefully turn the bottle upside down with the syringe tip firmly inserted into… [Excerpted — this section continues on DailyMed.]

📄 Recent Major Changes 9 words ▾

Dosage and Administration ( 2.1 , 2.2 ) 2/2026

📄 Package Label / Principal Display Panel 94 words ▾

PRINCIPAL DISPLAY PANEL - 60 mg/80 mL Bottle Carton NDC 50242-175-07 Evrysdi ® (risdiplam) for oral solution 60 mg/80 mL (0.75 mg/mL) Attention pharmacist: Evrysdi must be constituted with water prior to dispensing. 80 mL (2.71 fl oz) total volume after constitution Rx only Genentech 11044976 PRINCIPAL DISPLAY PANEL - 60 mg/80 mL Bottle Carton

PRINCIPAL DISPLAY PANEL - 5 mg Tablet Bottle Carton NDC 50242-202-01 Evrysdi ® (risdiplam) Tablets 5 mg Do not chew, cut, or crush tablet. 30 Tablets Rx only Genentech 11044980 PRINCIPAL DISPLAY PANEL - 5 mg Tablet Bottle Carton

Source: FDA Structured Product Labeling, mirrored from DailyMed / openFDA. Prefer the government’s original formatting? View this label on DailyMed ↗

Medicaid utilization & spend

📍 This exact package only: Medicaid data is reported per full 11-digit NDC — labeler, product and pack size — so every number here is for this package alone, not the drug overall. Other pack sizes report separately.
💊 Pharmacy benefit only: These are Medicaid outpatient pharmacy claims, billed by NDC. They exclude the medical benefit — clinic- or hospital-administered drugs billed under HCPCS J-codes — so drugs used mostly that way (e.g. Avastin, Lucentis, Keytruda) can look low or missing here. That’s expected, not an error.
📅 Q1 2025 – Q1 2026 · 5 quarters of data
ⓘ The newest quarter is usually incomplete when first published; states restate recent quarters in later CMS releases, so the latest figures typically revise upward. State coverage-policy changes can also shift quarter-to-quarter totals.
Prescriptions last 4 qtrs
2.1K
Units reimbursed last 4 qtrs
63.9K
Gross reimbursed last 4 qtrs
$72.63M
Avg / prescription
$34,241.04
Avg / unit
$1,135.82
Latest quarter Q1 2026
737Rx
Fee-for-service vs managed care ⓘ
52% FFS 48% MCO
Fee-for-service · 1,095 Rx Managed care · 1,026 Rx
State Medicaid map
Alaska: no data reported AK Maine: no data reported ME Washington: 1,650 units · 21.1 per 100k residents WA Idaho: 750 units · 38.2 per 100k residents ID Montana: no data reported MT North Dakota: no data reported ND Minnesota: no data reported MN Wisconsin: 1,170 units · 19.8 per 100k residents WI Michigan: 3,690 units · 36.8 per 100k residents MI New York: 3,660 units · 18.7 per 100k residents NY Vermont: no data reported VT New Hampshire: no data reported NH Oregon: 840 units · 19.8 per 100k residents OR Nevada: no data reported NV Wyoming: no data reported WY South Dakota: no data reported SD Iowa: 1,350 units · 42.1 per 100k residents IA Illinois: 780 units · 6.2 per 100k residents IL Indiana: 1,800 units · 26.2 per 100k residents IN Ohio: 5,610 units · 47.6 per 100k residents OH Pennsylvania: 3,120 units · 24.1 per 100k residents PA New Jersey: 1,350 units · 14.5 per 100k residents NJ Massachusetts: no data reported MA California: 7,830 units · 20.1 per 100k residents CA Utah: no data reported UT Colorado: 1,110 units · 18.9 per 100k residents CO Nebraska: no data reported NE Missouri: no data reported MO Kentucky: 330 units · 7.3 per 100k residents KY West Virginia: no data reported WV Virginia: 1,140 units · 13.1 per 100k residents VA Maryland: no data reported MD Connecticut: no data reported CT Rhode Island: no data reported RI Arizona: 3,670 units · 49.4 per 100k residents AZ New Mexico: 360 units · 17.0 per 100k residents NM Kansas: no data reported KS Arkansas: 1,081 units · 35.2 per 100k residents AR Tennessee: 1,590 units · 22.3 per 100k residents TN North Carolina: 4,590 units · 42.4 per 100k residents NC South Carolina: no data reported SC Delaware: no data reported DE Oklahoma: 2,010 units · 49.6 per 100k residents OK Louisiana: 390 units · 8.5 per 100k residents LA Mississippi: no data reported MS Alabama: 390 units · 7.6 per 100k residents AL Georgia: 990 units · 9.0 per 100k residents GA D.C.: no data reported DC Hawaii: no data reported HI Texas: 4,320 units · 14.2 per 100k residents TX Florida: 6,090 units · 26.9 per 100k residents FL
Units reimbursed · per 100k residents
6.249.6
gray = no data reported ⓘ
Colors are per 100,000 residents, so big states don’t automatically dominate. Tap or hover a state for its actual totals.
Tap or hover a state
…for its Medicaid breakdown
🏆 Top states by units · per 100k residents
1 Oklahoma 49.6 /100k
2 Arizona 49.4 /100k
3 Ohio 47.6 /100k
4 North Carolina 42.4 /100k
5 Iowa 42.1 /100k
6 Idaho 38.2 /100k
7 Michigan 36.8 /100k
8 Arkansas 35.2 /100k
National units — by quarter
💵 About the dollar figures: “reimbursed” is what Medicaid paid pharmacies before confidential manufacturer rebates, so the program’s real net cost is lower than these numbers. Fee-for-service and managed-care claims are combined unless split above. Source: CMS State Drug Utilization Data; per-100k rates use 2023 Census population estimates.

Medicare Part D spend CMS · PART D · 2026 (Q1)

Medicare Part D (outpatient prescription) spending for Evrysdi — the program that covers self-administered drugs. 1 manufacturer.
⚠️ Drug-level data: CMS publishes Part D spending by drug, not by NDC — these figures combine every manufacturer, strength and package size sold under the name Evrysdi. That’s a different level of aggregation than the Medicaid card above, which is specific to this exact 11-digit NDC (pack size included), so the two aren’t directly comparable.
Period
Total Part D spend
$51.15M
Claims incl. refills
1.5K
Beneficiaries
519
Spend / beneficiary
$98,557.13
Spend / claim
$33,410.29
Trend by period
💵 About the dollar figures: spending is what Part D plans paid before confidential manufacturer rebates, so the program’s real net cost is lower. A blank patient count means fewer than 11 people — CMS hides counts that small to protect privacy. Source: CMS Medicare Quarterly Part D Spending by Drug (data.cms.gov), updated quarterly.

Reported adverse events (FAERS)

Read carefully: FAERS reports are voluntary and unverified. Counts are not incidence, do not establish causation, are subject to reporting bias, and cannot be used to compare one drug to another. Shown for signal context only. Reports for EVRYSDI (this brand).

Top reported reactions

Diarrhoea296
Asthenia225
Pneumonia221
Death205
Pyrexia182
Fatigue158
Muscular Weakness145

Age at onset

Neonate42
Infant189
Child453
Adolescent186
Adult798
Elderly58

Reporter sex

4,081 reports
Male · 45%
Female · 55%
Reports over time (by year) — tap or hover for the count & year
2021 2022 2024 2026 1,378 229
Most recent year is provisional (FAERS lags ~3 months).
Where does this data come from?
Adverse-event reports from the FDA Adverse Event Reporting System (FAERS) via openFDA. FAERS reports are voluntary and unverified — counts are not incidence and don’t establish causation.

About this NDC listing & data coverage

Finished prescription product
What data is (and isn’t) available for this NDC — tap to expand
NDC identity (package / product / labeler codes) ✓ Available
Labeler ✓ Available
Product & package description ✓ Available
Marketing category & status ✓ Available
Active ingredient / dosage form / route ✓ Available
FDA label (SPL via DailyMed) ✓ Available
Package photos ✓ Available
Inactive ingredients (structured) ✓ Available
NADAC pharmacy acquisition price (CMS) — Not published for this NDC CMS publishes NADAC only for NDCs reported in its retail-pharmacy survey.
Orange Book / therapeutic-equivalence data ✓ Available
HCPCS J-code billing crosswalk — Not published for this NDC Most self-administered / retail products have no J-code — that is normal.
Medicaid utilization (CMS SDUD) ✓ Available
“Not published” reflects what the public FDA / CMS / NLM sources provide for this exact package code — it is a property of the data feeds, not a judgment about the product.

Questions about this listing

Why is there no price listed?
The pricing shown on our NDC pages comes from CMS NADAC, a voluntary survey of retail community pharmacy invoices. CMS does not publish a NADAC for every NDC — packages outside the retail survey (institutional and hospital products, bulk packages, discontinued items, and many OTC items) may never receive one. A missing price reflects the survey's scope, not this product's actual cost, and does not mean the product is free or unavailable.
Is the NDC printed on the package the same as the 11-digit billing NDC?
Yes, they identify this exact package in different formats. The form printed on the packaging and shown on DailyMed is the one the FDA registered. Insurance claims use a fixed 11-digit 5-4-2 format, so the short segment is padded with a leading zero and the dashes are dropped. The Identity section at the top of this page lists each form of this code.
Is this package still being marketed?
Yes, per the latest FDA NDC Directory data on this page: this package is listed as actively marketed, with no marketing end date reported by Genentech, Inc.. Listing status can change — the directory data on this page refreshes weekly.
Who lists this product with the FDA?
Genentech, Inc. is the labeler of record for this NDC — the company under whose FDA-assigned code the package is listed. The labeler may be the manufacturer itself or a distributor marketing the product under its own code.
Do I need a prescription for this product?
This NDC is listed with FDA as a prescription product, so it is dispensed under a prescriber's order. Your pharmacist can tell you whether any over-the-counter forms of the same medication exist.
This page identifies an FDA-listed package (the NDC) and reports public regulatory and pricing data about the listing. It is reference information, not a medical recommendation — talk to your pharmacist or prescriber about your own medication.
Where does this data come from?
Listing facts (marketing category, packager status, marketing dates) from the FDA openFDA NDC Directory; label availability from DailyMed; pricing coverage from CMS NADAC; equivalence scope from the FDA Orange Book.
← 50242-0201-01 · Genentech, 50242-0210-60 · Genentech, →
For educational and professional reference only — not medical advice. Pricing reflects published NADAC and CMS ASP (free public data) and may differ from your acquisition cost; always verify before billing or dispensing.