Home › NDC Lookup › Ingredients › Golodirsen › 60923-0465-02
Vyondys 53 golodirsen 50 mg/mL Injection — NDC 60923-0465-02 package photo
Label image from the product's FDA listing (DailyMed) — may show a different pack size or an older label revision.

Vyondys 53 golodirsen 50 mg/mL Injection — NDC 60923-465-02 (Billing 60923-0465-02)

by Sarepta Therapeutics, Inc. · 1 VIAL, SINGLE-USE in 1 CARTON / 2 mL in 1 VIAL, SINGLE-USE

This is a package of Vyondys 53 golodirsen 50 mg/mL Injection from Sarepta Therapeutics, Inc., marketed since Dec 2019 and currently FDA-listed. It is this product's only package size.

NDC 60923-0465-02
🏷️ FDA NDC (as labeled) 60923-465-02 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 Jul 24, 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 60923-465-02 alone.

Record
FDA NDC Directory package listing · Human prescription drug
Code segments
60923 labeler · 465 product · 02 package
Package marketed since
Dec 12, 2019
Sample package
No — commercial package
Listing certified through
Dec 31, 2026
Barcode (UPC-A, from the NDC)
3 6092346502 8
Medicaid fills, this package
816 prescriptions in the last four reported quarters
FDA record last changed
Jul 24, 2026

Identity & classification

Regulatory identifiers FDA, NLM and CMS codes for this package

FDA NDC (as labeled) 60923-465-02
Product NDC 60923-465
11-digit billing NDC 60923046502
NCPDP billing unit ML — per mL (volume)
RxCUI 2267211, 2267216
UNII 033072U4MZ
Application # NDA211970
SPL Set ID 35c227d1-5b24-44b0-b5d3-f0f6b1c46bd5
Established class (EPC) Antisense Oligonucleotide
Physiologic effect Increased Protein Synthesis
Chemical class Oligonucleotides, Antisense
DEA schedule Non-controlled
Marketing category NDA
Marketing status On market
FDA listing status Listed (active directory)
Marketing start 2019-12-12
Route INTRAVENOUS
Dosage form INJECTION
Substance GOLODIRSEN

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

GPI-14 74600042002020
GPI class Vyondys 53
GCN Seq No 080548
GCN 47426
HICL code 046254
Ingredient (HICL) Golodirsen
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 Z1R
Therapeutic class — specific (HIC3) Genetic D/O Tx-Exon Skipping Antisense Oligonucleo
AHFS code 92:18.00.00
AHFS class Antisense Oligonucleotides
FDB label name VYONDYS-53 100 MG/2 ML VIAL
FDB brand name Vyondys-53
Legend status F — Federal legend — prescription drug or device
Quick answers
  • GSN (GCN sequence number): 080548
  • GCN: 47426
  • GPI-14 (Medi-Span): 74600042002020
  • HICL (First Databank): 046254
  • AHFS class code: 92:18.00.00
  • RxCUI (RxNorm): 2267211
Why two NDCs? The FDA registers this code as 60923-465-02 — 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 → 60923-0465-02. 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.

RxNorm drug class

This medicine belongs to the Antisense Oligonucleotide class.

Pharmacologic class Antisense Oligonucleotide
Drug family (ATC) Other drugs for disorders of the musculo-skeletal system
Where does this data come from?
Therapeutic classes from RxNorm RxClass (U.S. National Library of Medicine) — Established Pharmacologic Class (FDA), ATC drug family (WHO) and mechanism of action, matched by this product’s RxCUI.

Clinical

Label name VYONDYS-53 100 MG/2 ML VIAL Ingredient Golodirsen
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 $744.83 $744.83 / 1 vial
Medicare drug plans payPart D · quarterly No Part D plan price is available for this NDC in our data.
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
60923-0465-02 You're viewing this Main listing 1 VIAL, SINGLE-USE in 1 CARTON / 2 mL in 1 VIAL, SINGLE-USE 2019-12-12 — Active

Therapeutic equivalents

ProductLabelerPackNADAC/unitTEStatusPrice vs. this
Vyondys 53 50 mg/mLthis 60923-0465-02 Sarepta 1 vial — — 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

🏛️
2019
First FDA approval
Dec 2019
📍
2026
Currently FDA-listed
7 years listed
🛡️
2028
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 Jun 2028. This may affect when a full generic version becomes widely available, but it is not a guaranteed launch date.
📅 FDA approved Dec 12, 2019 RLD RS ⏳ ~1.7 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 RE47691 — drug product
Exclusivity ODE-280
2019 2021 2023 2025 2027
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 (1)
PatentTypeUse codeExpires
US RE47691 ↗ Drug product — Jun 28, 2028
FDA exclusivity
CodeWhat it grantsExpires
ODE-280Orphan Drug Exclusivity (7-year)Dec 12, 2026
Common questions
Is there a generic version of VYONDYS-53 100 MG/2 ML VIAL?
No FDA-approved generic equivalent is currently listed in the FDA Orange Book for VYONDYS-53 100 MG/2 ML VIAL. Based on the patents and exclusivity currently listed, the Orange Book estimate is that full-label generic entry may be delayed until Jun 2028 — 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.

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.

  • UNII QTT17582CB
    A strong acid used to adjust and maintain the proper pH level in liquid medicines, ensuring stability and preventing breakdown of active ingredients.
  • UNII 660YQ98I10
    Potassium chloride is a mineral salt used in medicines as a buffer and to help maintain the proper pH balance and stability of the formulation during storage and use.
  • UNII 4J9FJ0HL51
    Potassium phosphate, monobasic is a salt compound used in medicines as a pH buffer and electrolyte source. It helps stabilize the acidity level of liquid and powdered formulations.
  • UNII 451W47IQ8X
    Sodium chloride is common table salt. It's used in medicines as a buffer to maintain proper pH, as a filler to add bulk, or to adjust the osmotic balance in liquid formulations.
  • UNII 55X04QC32I
    A strong alkaline chemical used to adjust and maintain the pH balance of liquid medicines. It helps keep the medicine stable and ensures it stays effective during storage.
  • UNII 22ADO53M6F
    A mineral salt derived from phosphoric acid, used as a buffer to maintain the pH balance of the medication and help stabilize the active ingredients.
  • UNII 059QF0KO0R
    Water is a liquid solvent that dissolves and mixes ingredients together in liquid medicines, syrups, and injections. It helps distribute the active drug evenly throughout the product.

7 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

LabelerSarepta Therapeutics, Inc.
Application holderSAREPTA THERAPEUTICS INC
FDA applicationNDA211970 (NDA)
Labeler code60923
First marketedDec 2019
Product typeHuman Prescription Drug
Portfolio65 products on file

More NDCs from Sarepta Therapeutics, Inc. labeler code 60923

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 ⚠️ Warnings and Cautions 🤒 Adverse Reactions 👥 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 📄 Recent Major Changes 📄 Package Label / Principal Display Panel
🎯 Indications and Usage 159 words ▾

1 INDICATIONS AND USAGE VYONDYS 53 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VYONDYS 53 [see Clinical Studies ( 14 )] . Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.

VYONDYS 53 is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VYONDYS 53. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.

( 1 )

⏱️ Dosage and Administration ~3 min read ▾

2 DOSAGE AND ADMINISTRATION Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting VYONDYS 53 ( 2.1 ) 30 milligrams per kilogram once weekly ( 2.2 ) Administer as an intravenous infusion over 35 to 60 minutes via an in-line 0.2 micron filter ( 2.2 , 2.4 ) Dilution required prior to administration ( 2.3 )

2.1Monitoring to Assess Safety Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting VYONDYS 53. Consider measurement of glomerular filtration rate prior to initiation of VYONDYS 53. Monitoring for kidney toxicity during treatment is recommended. Obtain the urine samples prior to infusion of VYONDYS 53 or at least 48 hours after the most recent infusion [see Warnings and Precautions ( 5.2 )] .

2.2Dosing Information The recommended dosage of VYONDYS 53 is 30 milligrams per kilogram administered once weekly as a 35 to 60-minute intravenous infusion via an in-line 0.2 micron filter. If a dose of VYONDYS 53 is missed, it may be administered as soon as possible after the scheduled dose.

2.3Preparation Instructions VYONDYS 53 is supplied in single-dose vials as a preservative-free concentrated solution that requires dilution prior to administration. Parenteral drug products should be inspected visually for particulate matter and discoloration prior to administration, whenever solution and container permit. Use aseptic technique.

Calculate the total dose of VYONDYS 53 to be administered based on the patient's weight and the recommended dose of 30 milligrams per kilogram. Determine the volume of VYONDYS 53 needed and the correct number of vials to supply the full calculated dose. Allow the vials to warm to room temperature.

Mix the contents of each vial by gently inverting 2 or 3 times. Do not shake. Visually inspect each vial of VYONDYS 53.

The solution is a clear to slightly opalescent, colorless liquid, and may contain trace amounts of small, white to off-white amorphous particles. Do not use if the solution in the vials is cloudy, discolored or contains extraneous particulate matter other than trace amounts of small, white to off-white amorphous particles. With a syringe fitted with a 21-gauge or smaller bore non-coring needle, withdraw the calculated volume of VYONDYS 53 from the appropriate number of vials.

Dilute the withdrawn VYONDYS 53 in 0.9% Sodium Chloride Injection, USP, to make a total volume of 100 to150 mL. Gently invert 2 to 3 times to mix. Do not shake.

Visually inspect the diluted solution. Do not use if the solution is cloudy, discolored or contains extraneous particulate matter other than trace amounts of small, white to off-white amorphous particles. Administer the diluted solution via an in-line 0.2 micron filter.

VYONDYS 53 contains no preservatives and should be administered immediately after dilution. Complete infusion of diluted VYONDYS 53 within 4 hours of dilution. If immediate use is not possible, the diluted product may be stored for up to 24 hours at 2°C to 8°C (36°F to 46°F).

Do not freeze. Discard unused VYONDYS 53.

2.4Administration Instructions Application of a topical anesthetic cream to the infusion site prior to administration of VYONDYS 53 may be considered. VYONDYS 53 is administered via intravenous infusion. Flush the intravenous access line with 0.9% Sodium Chloride Injection, USP, prior to and after infusion.

Infuse the diluted VYONDYS 53 over 35 to 60 minutes via an in-line 0.2 micron filter. Do not mix other medications with VYONDYS 53 or infuse other medications concomitantly via the same intravenous access line with VYONDYS 53. If a hypersensitivity reaction occurs, consider slowing the infusion, interrupting, or discontinuing the VYONDYS 53 therapy [see Contraindications ( 4 ), Warnings and Precautions ( 5.1 ,) and Adverse Reactions ( 6.1 )] .

💊 Dosage Forms and Strengths 54 words ▾

3 DOSAGE FORMS AND STRENGTHS VYONDYS 53 is a clear to slightly opalescent, colorless liquid, and may contain trace amounts of small, white to off-white amorphous particles, and available as: Injection: 100 mg/2 mL (50 mg/mL) solution in a single-dose vial Injection: 100 mg/2 mL (50 mg/mL) in a single-dose vial ( 3 )

⛔ Contraindications 67 words ▾

4 CONTRAINDICATIONS VYONDYS 53 is contraindicated in patients with a serious hypersensitivity reaction to golodirsen or to any of the inactive ingredients in VYONDYS 53. Anaphylaxis has occurred in patients receiving VYONDYS 53 [see Warnings and Precautions ( 5.1 )] . VYONDYS 53 is contraindicated in patients with serious hypersensitivity to golodirsen or to any of the inactive ingredients in VYONDYS 53. ( 4 , 5.1 )

⚠️ Warnings and Cautions ~2 min read ▾

5 WARNINGS AND PRECAUTIONS Hypersensitivity Reactions: Hypersensitivity reactions, including anaphylaxis, rash, pyrexia, pruritus, urticaria, dermatitis, and skin exfoliation have occurred in patients who were treated with VYONDYS 53. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion, interrupting or discontinuing the VYONDYS 53 therapy. ( 2.3 , 4 , 5.1 ) Kidney Toxicity: Based on animal data, may cause kidney toxicity.

Kidney function should be monitored; creatinine may not be a reliable measure of renal function in DMD patients. ( 5.2 , 13.2 )

5.1Hypersensitivity Reactions Hypersensitivity reactions, including anaphylaxis, rash, pyrexia, pruritus, urticaria, dermatitis, and skin exfoliation have occurred in VYONDYS 53-treated patients, some requiring treatment. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion, interrupting, or discontinuing the VYONDYS 53 therapy and monitor until the condition resolves [see Dosage and Administration ( 2.4 )]. VYONDYS 53 is contraindicated in patients with a history of a serious hypersensitivity reaction to golodirsen or to any of the inactive ingredients in VYONDYS 53 [see Contraindications ( 4 )].

5.2Kidney Toxicity Kidney toxicity was observed in animals who received golodirsen [see Use in Specific Populations ( 8.4 )] . Although kidney toxicity was not observed in the clinical studies with VYONDYS 53, the clinical experience with VYONDYS 53 is limited, and kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. Kidney function should be monitored in patients taking VYONDYS 53.

Because of the effect of reduced skeletal muscle mass on creatinine measurements, creatinine may not be a reliable measure of kidney function in DMD patients. Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting VYONDYS 53. Consider also measuring glomerular filtration rate using an exogenous filtration marker before starting VYONDYS 53.

During treatment, monitor urine dipstick every month, and serum cystatin C and urine protein-to-creatinine ratio every three months. Only urine expected to be free of excreted VYONDYS 53 should be used for monitoring of urine protein. Urine obtained on the day of VYONDYS 53 infusion prior to the infusion, or urine obtained at least 48 hours after the most recent infusion, may be used.

Alternatively, use a laboratory test that does not use the reagent pyrogallol red, as this reagent has the potential to cross react with any VYONDYS 53 that is excreted in the urine and thus lead to a false positive result for urine protein. If a persistent increase in serum cystatin C or proteinuria is detected, refer to a pediatric nephrologist for further evaluation.

🤒 Adverse Reactions ~2 min read ▾

6 ADVERSE REACTIONS The following serious adverse reactions are described below and elsewhere in the labeling: Hypersensitivity Reactions [see Warnings and Precautions ( 5.1 )] The most common adverse reactions (incidence ≥20% and higher than placebo) were headache, pyrexia, fall, abdominal pain, nasopharyngitis, cough, vomiting, and nausea. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Sarepta Therapeutics, Inc. at 1-888-SAREPTA (1-888-727-3782) 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 the clinical trials of another drug and may not reflect the rates observed in practice. In the VYONDYS 53 clinical development program, 58 patients received at least one intravenous dose of VYONDYS 53, ranging between 4 mg/kg (0.13 times the recommended dosage) and 30 mg/kg (the recommended dosage). All patients were male and had genetically confirmed Duchenne muscular dystrophy.

Age at study entry was 6 to 13 years. Most (86%) patients were Caucasian. VYONDYS 53 was studied in 2 double-blind, placebo-controlled studies.

In Study 1 Part 1, patients were randomized to receive once-weekly intravenous infusions of VYONDYS 53 (n=8) in four increasing dose levels from 4 mg/kg to 30 mg/kg or placebo (n=4), for at least 2 weeks at each level. All patients who participated in Study 1 Part 1 (n=12) were continued into Study 1 Part 2, an open-label extension, during which they received VYONDYS 53 at a dose of 30 mg/kg IV once weekly [see Clinical Studies ( 14 )] . In Study 2, patients received VYONDYS 53 (n=33) 30 mg/kg or placebo (n=17) IV once weekly for up to 96 weeks, after which all patients received VYONDYS 53 at a dose of 30 mg/kg.

Adverse reactions observed in at least 20% of treated patients in the placebo-controlled sections of Studies 1 and 2 are shown in Table 1 . Table 1: Adverse Reactions That Occurred in At Least 20% of VYONDYS 53-Treated Patients and at a Rate Greater than Placebo in Studies 1 and 2 Adverse Reaction VYONDYS 53 (N = 41) % Placebo (N = 21) % Headache 41 10 Pyrexia 41 14 Fall 29 19 Abdominal pain 27 10 Nasopharyngitis 27 14 Cough 27 19 Vomiting 27 19 Nausea 20 10 Other adverse reactions that occurred at a frequency greater than 5% of VYONDYS 53-treated patients and at a greater frequency than placebo were: administration site pain, back pain, pain, diarrhea, dizziness, ligament sprain, contusion, influenza, oropharyngeal pain, rhinitis, skin abrasion, ear infection, seasonal allergy, tachycardia, catheter site related reaction, constipation, and fracture.

Hypersensitivity reactions have occurred in patients treated with VYONDYS 53 [see Warnings and Precautions ( 5.1 )].

6.2Postmarketing Experience The following adverse reactions have been identified during postapproval use of VYONDYS 53. Because these reactions are reported voluntarily from a population of uncertain size, it is not always possible to reliably estimate their frequency or establish a causal relationship to drug exposure. Immune System Disorders: anaphylaxis [see Contraindications ( 4 ) and Warnings and Precautions ( 5.1 )]

👥 Use in Specific Populations ~2 min read ▾

8 USE IN SPECIFIC POPULATIONS

8.1Pregnancy Risk Summary There are no human or animal data available to assess the use of VYONDYS 53 during pregnancy. In the U.S. general population, major birth defects occur in 2 to 4% and miscarriage occurs in 15 to 20% of clinically recognized pregnancies.

8.2Lactation Risk Summary There are no human or animal data to assess the effect of VYONDYS 53 on milk production, the presence of golodirsen in milk, or the effects of VYONDYS 53 on the breastfed infant. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for VYONDYS 53 and any potential adverse effects on the breastfed infant from VYONDYS 53 or from the underlying maternal condition.

8.4Pediatric Use VYONDYS 53 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping, including pediatric patients [see Clinical Studies ( 14 )]. Intravenous administration of golodirsen (0, 100, 300, or 900 mg/kg) to juvenile male rats once weekly for 10 weeks (postnatal days 14 to 77) did not result in postnatal developmental (e.g., neurobehavioral, immune function, or male reproductive) toxicity. However, at the highest dose tested (900 mg/kg/week), golodirsen resulted in the death of animals because of renal impairment or failure.

In surviving animals (including one animal at the lowest dose tested), there was a dose-dependent increase in the incidence and severity of renal tubular effects (including degeneration/regeneration, fibrosis, vacuolation, and dilatation), which correlated with changes in clinical pathology parameters, reflecting a dose-dependent impairment of renal function. In addition, decreases in bone area, mineral content, and mineral density were observed at the highest dose tested (900 mg/kg week) but with no effect on bone growth.

A no-effect dose for renal toxicity was not identified; the lowest dose tested (100 mg/kg/week) was associated with plasma exposures (AUC) approximately 2.5 times that in humans at the recommended human dose of 30 mg/kg/week.

8.5Geriatric Use DMD is largely a disease of children and young adults; therefore, there is no geriatric experience with VYONDYS 53.

8.6Patients with Renal Impairment Renal clearance of golodirsen is reduced in non-DMD adults with renal impairment, based on estimated glomerular filtration rate calculated using the Modification of Diet and Renal Disease (MDRD) equation [see Clinical Pharmacology ( 12.3 )]. However, because of the effect of reduced skeletal muscle mass on creatinine measurements in DMD patients, no specific dosage adjustment can be recommended for DMD patients with renal impairment based on estimated glomerular filtration rate. Patients with known renal function impairment should be closely monitored during treatment with VYONDYS 53.

🤰 Pregnancy 45 words ▾

8.1Pregnancy Risk Summary There are no human or animal data available to assess the use of VYONDYS 53 during pregnancy. In the U.S. general population, major birth defects occur in 2 to 4% and miscarriage occurs in 15 to 20% of clinically recognized pregnancies.

🧒 Pediatric Use 210 words ▾

8.4Pediatric Use VYONDYS 53 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping, including pediatric patients [see Clinical Studies ( 14 )]. Intravenous administration of golodirsen (0, 100, 300, or 900 mg/kg) to juvenile male rats once weekly for 10 weeks (postnatal days 14 to 77) did not result in postnatal developmental (e.g., neurobehavioral, immune function, or male reproductive) toxicity. However, at the highest dose tested (900 mg/kg/week), golodirsen resulted in the death of animals because of renal impairment or failure.

In surviving animals (including one animal at the lowest dose tested), there was a dose-dependent increase in the incidence and severity of renal tubular effects (including degeneration/regeneration, fibrosis, vacuolation, and dilatation), which correlated with changes in clinical pathology parameters, reflecting a dose-dependent impairment of renal function. In addition, decreases in bone area, mineral content, and mineral density were observed at the highest dose tested (900 mg/kg week) but with no effect on bone growth.

A no-effect dose for renal toxicity was not identified; the lowest dose tested (100 mg/kg/week) was associated with plasma exposures (AUC) approximately 2.5 times that in humans at the recommended human dose of 30 mg/kg/week.

🧓 Geriatric Use 22 words ▾

8.5Geriatric Use DMD is largely a disease of children and young adults; therefore, there is no geriatric experience with VYONDYS 53.

🧬 Clinical Pharmacology ~3 min read ▾

12 CLINICAL PHARMACOLOGY

12.1Mechanism of Action Golodirsen is designed to bind to exon 53 of dystrophin pre-mRNA resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping. Exon 53 skipping is intended to allow for production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 53 skipping [see Clinical Studies ( 14 )].

12.2Pharmacodynamics After treatment with VYONDYS 53, all patients evaluated (n=25) in Study 1 Part 2 [see Clinical Studies ( 14 )] had an increase in skipping of exon 53 demonstrated by reverse transcription polymerase chain reaction (RT-PCR), compared to baseline. In Study 1 Part 2 [see Clinical Studies ( 14 )], dystrophin levels as assessed by the Sarepta western blot assay increased from 0.10% (SD 0.07) of normal at baseline to 1.02% (SD 1.03) of normal after 48 weeks of treatment with VYONDYS 53. The mean change from baseline in dystrophin after 48 weeks of treatment with VYONDYS 53 was 0.92% (SD 1.01) of normal levels (p<0.001); the median change from baseline was 0.88%.

This increase in dystrophin protein expression positively correlated with the level of exon skipping. Dystrophin levels assessed by western blot can be meaningfully influenced by differences in sample processing, analytical technique, reference materials, and quantitation methodologies. Therefore, comparing dystrophin results from different assay protocols will require a standardized reference material and additional bridging studies.

Correct localization of truncated dystrophin to the sarcolemma in muscle fibers of patients treated with golodirsen was demonstrated by immunofluorescence staining.

12.3Pharmacokinetics The pharmacokinetics of golodirsen was evaluated in DMD patients following administration of intravenous doses ranging from 4 mg/kg/week to 30 mg/kg/week (i.e., recommended dosage). Golodirsen exposure increased proportionally with dose, with minimal accumulation with once-weekly dosing. Inter-subject variability (as %CV) for C max and AUC ranged from 38% to 72%, and 34% to 44%, respectively.

Distribution Steady-state volume of distribution was similar between DMD patients and healthy subjects. The mean golodirsen steady-state volume of distribution was 668 mL/kg (%CV=32.3) at a dose of 30 mg/kg. Golodirsen plasma protein binding ranged from 33% to 39% and is not concentration dependent.

Elimination Golodirsen elimination half-life (SD) was 3.4 (0.6) hours, and plasma clearance was 346 mL/hr/kg at the 30 mg/kg dose. Metabolism Golodirsen is metabolically stable. No metabolites were detected in plasma or urine.

Excretion Golodirsen is mostly excreted unchanged in the urine. The elimination half-life (t 1/2 ) was 3.4 hours. Specific Populations Age : The pharmacokinetics of golodirsen have been evaluated in male pediatric DMD patients.

There is no experience with the use of VYONDYS 53 in DMD patients 65 years of age or older. Sex : Sex effects have not been evaluated; VYONDYS 53 has not been studied in female patients. Race : The potential impact of race is not known because 92% of the patients in studies were Caucasians.

Patients with Renal Impairment : The effect of renal impairment on the pharmacokinetics of golodirsen was evaluated in non-DMD subjects aged 41 to 65 years with Stage 2 chronic kidney disease (CKD) (n=8, estimated glomerular filtration rate (eGFR) ≥60 and <90 mL/min/1.73 m 2 ) or Stage 3 CKD (n=8, eGFR ≥30 and <60 mL/min/1.73 m 2 ) and matched healthy subjects (n=8, eGFR ≥90 mL/min/1.73 m 2 ). Subjects received a single 30 mg/kg IV dose of golodirsen. In subjects with Stage 2 or Stage 3 CKD, exposure (AUC) increased approximately 1.2-fold and 1.9-fold, respectively.

There was no change in the C max in subjects with Stage 2 CKD; in subjects with Stage 3 CKD, there was a 1.2-fold increase in C max compared with subjects with normal renal function. The effect of Stage 4 or Stage 5 CKD… [Excerpted — this section continues on DailyMed.]

🧬 Mechanism of Action 69 words ▾

12.1Mechanism of Action Golodirsen is designed to bind to exon 53 of dystrophin pre-mRNA resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping. Exon 53 skipping is intended to allow for production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 53 skipping [see Clinical Studies ( 14 )].

📦 How Supplied / Storage and Handling 77 words ▾

16 HOW SUPPLIED/STORAGE AND HANDLING

16.1How Supplied VYONDYS 53 injection is supplied in single dose vials. The solution is a clear to slightly opalescent, colorless liquid, and may contain trace amounts of small, white to off-white amorphous particles. Single-dose vials containing 100 mg/2mL (50 mg/mL) NDC 60923-465-02

16.2Storage and Handling Store VYONDYS 53 at 2°C to 8°C (36°F to 46°F). Do not freeze. Store in original carton until ready for use to protect from light.

📦 Storage and Handling 29 words ▾

16.2Storage and Handling Store VYONDYS 53 at 2°C to 8°C (36°F to 46°F). Do not freeze. Store in original carton until ready for use to protect from light.

📋 Description ~1 min read ▾

11 DESCRIPTION VYONDYS 53 (golodirsen) injection is a sterile, aqueous, preservative-free, concentrated solution for dilution prior to intravenous administration. VYONDYS 53 is a clear to slightly opalescent, colorless liquid, and may contain trace amounts of small, white to off-white amorphous particles. VYONDYS 53 is supplied in single-dose vials containing 100 mg golodirsen (50 mg/mL).

VYONDYS 53 is formulated as an isotonic phosphate buffered saline solution with an osmolality of 260 to 320 mOSM and a pH of 7.5. Each milliliter of VYONDYS 53 contains: 50 mg golodirsen; 0.2 mg potassium chloride; 0.2 mg potassium phosphate monobasic; 8 mg sodium chloride; and 1.14 mg sodium phosphate dibasic, anhydrous, in water for injection. The product may contain hydrochloric acid or sodium hydroxide to adjust pH.

Golodirsen is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer (PMO) subclass. PMOs are synthetic molecules in which the five-membered ribofuranosyl rings found in natural DNA and RNA are replaced by a six-membered morpholino ring. Each morpholino ring is linked through an uncharged phosphorodiamidate moiety rather than the negatively charged phosphate linkage that is present in natural DNA and RNA.

Each phosphorodiamidate morpholino subunit contains one of the heterocyclic bases found in DNA (adenine, cytosine, guanine, or thymine). Golodirsen contains 25 linked subunits. The sequence of bases from the 5' end to 3' end is GTTGCCTCCGGTTCTGAAGGTGTTC.

The molecular formula of golodirsen is C 305 H 481 N 138 O 112 P 25 and the molecular weight is 8647.28 daltons. The structure of golodirsen is: Structure of Golodirsen

💬 Information for Patients 148 words ▾

17 PATIENT COUNSELING INFORMATION Hypersensitivity Reactions Advise patients and/or caregivers that hypersensitivity reactions, including anaphylaxis, rash, pyrexia, pruritus, urticaria, dermatitis, and skin exfoliation have occurred in patients who were treated with VYONDYS 53. Instruct them to seek immediate medical care should they experience signs and symptoms of hypersensitivity [see Warnings and Precautions ( 5.1 )] . Kidney Toxicity Inform patients nephrotoxicity has occurred with drugs similar to VYONDYS 53.

Advise patients of the importance of monitoring for kidney toxicity by their healthcare providers during treatment with VYONDYS 53 [see Warnings and Precautions ( 5.2 )] . Manufactured for: Sarepta Therapeutics, Inc. Cambridge, MA 02142 USA SAREPTA, SAREPTA THERAPEUTICS, and VYONDYS are trademarks of Sarepta Therapeutics, Inc. registered in the U.S.

Patent and Trademark Office and may be registered in various other jurisdictions. VYONDYS 53, and the Vyondys 53 logo are trademarks of Sarepta Therapeutics, Inc. Vyondys 53 Logo

🧬 Pharmacokinetics ~2 min read ▾

12.3Pharmacokinetics The pharmacokinetics of golodirsen was evaluated in DMD patients following administration of intravenous doses ranging from 4 mg/kg/week to 30 mg/kg/week (i.e., recommended dosage). Golodirsen exposure increased proportionally with dose, with minimal accumulation with once-weekly dosing. Inter-subject variability (as %CV) for C max and AUC ranged from 38% to 72%, and 34% to 44%, respectively.

Distribution Steady-state volume of distribution was similar between DMD patients and healthy subjects. The mean golodirsen steady-state volume of distribution was 668 mL/kg (%CV=32.3) at a dose of 30 mg/kg. Golodirsen plasma protein binding ranged from 33% to 39% and is not concentration dependent.

Elimination Golodirsen elimination half-life (SD) was 3.4 (0.6) hours, and plasma clearance was 346 mL/hr/kg at the 30 mg/kg dose. Metabolism Golodirsen is metabolically stable. No metabolites were detected in plasma or urine.

Excretion Golodirsen is mostly excreted unchanged in the urine. The elimination half-life (t 1/2 ) was 3.4 hours. Specific Populations Age : The pharmacokinetics of golodirsen have been evaluated in male pediatric DMD patients.

There is no experience with the use of VYONDYS 53 in DMD patients 65 years of age or older. Sex : Sex effects have not been evaluated; VYONDYS 53 has not been studied in female patients. Race : The potential impact of race is not known because 92% of the patients in studies were Caucasians.

Patients with Renal Impairment : The effect of renal impairment on the pharmacokinetics of golodirsen was evaluated in non-DMD subjects aged 41 to 65 years with Stage 2 chronic kidney disease (CKD) (n=8, estimated glomerular filtration rate (eGFR) ≥60 and <90 mL/min/1.73 m 2 ) or Stage 3 CKD (n=8, eGFR ≥30 and <60 mL/min/1.73 m 2 ) and matched healthy subjects (n=8, eGFR ≥90 mL/min/1.73 m 2 ). Subjects received a single 30 mg/kg IV dose of golodirsen. In subjects with Stage 2 or Stage 3 CKD, exposure (AUC) increased approximately 1.2-fold and 1.9-fold, respectively.

There was no change in the C max in subjects with Stage 2 CKD; in subjects with Stage 3 CKD, there was a 1.2-fold increase in C max compared with subjects with normal renal function. The effect of Stage 4 or Stage 5 CKD on golodirsen pharmacokinetics and safety has not been studied. Estimated GFR values derived from MDRD equations and the threshold definitions for various CKD stages in otherwise healthy adults would not be generalizable to pediatric patients with DMD.

Therefore, no specific dosage adjustment can be recommended for patients with renal impairment [see Use in Specific Populations ( 8.6 )]. Patients with Hepatic Impairment : VYONDYS 53 has not been studied in patients with hepatic impairment. Drug Interaction Studies Golodirsen did not inhibit CYP1A2, CYP2B6, CYP2C8, CYP2C9, CYP2C19, CYP2D6, or CYP3A4/5 in vitro .

Golodirsen was a weak inducer of CYP1A2 and did not induce CYP2B6 or CYP3A4. Golodirsen was not metabolized by human hepatic microsomes and was not a substrate or strong inhibitor of any of the key human drug transporters tested (OAT1, OAT3, OCT2, OATP1B1, MATE1, P-gp, BCRP, and MRP2, OATP1B3 and MATE2-K). Based on in vitro data, golodirsen has a low potential for drug-drug interactions in humans.

🧬 Pharmacodynamics 190 words ▾

12.2Pharmacodynamics After treatment with VYONDYS 53, all patients evaluated (n=25) in Study 1 Part 2 [see Clinical Studies ( 14 )] had an increase in skipping of exon 53 demonstrated by reverse transcription polymerase chain reaction (RT-PCR), compared to baseline. In Study 1 Part 2 [see Clinical Studies ( 14 )], dystrophin levels as assessed by the Sarepta western blot assay increased from 0.10% (SD 0.07) of normal at baseline to 1.02% (SD 1.03) of normal after 48 weeks of treatment with VYONDYS 53. The mean change from baseline in dystrophin after 48 weeks of treatment with VYONDYS 53 was 0.92% (SD 1.01) of normal levels (p<0.001); the median change from baseline was 0.88%.

This increase in dystrophin protein expression positively correlated with the level of exon skipping. Dystrophin levels assessed by western blot can be meaningfully influenced by differences in sample processing, analytical technique, reference materials, and quantitation methodologies. Therefore, comparing dystrophin results from different assay protocols will require a standardized reference material and additional bridging studies.

Correct localization of truncated dystrophin to the sarcolemma in muscle fibers of patients treated with golodirsen was demonstrated by immunofluorescence staining.

🔬 Clinical Studies ~2 min read ▾

14 CLINICAL STUDIES The effect of VYONDYS 53 on dystrophin production was evaluated in one study in DMD patients with a confirmed mutation of the DMD gene that is amenable to exon 53 skipping (Study 1; NCT02310906). Study 1 Part 1 was a double-blind, placebo-controlled, dose-titration study in 12 DMD patients. Patients were randomized 2:1 to receive VYONDYS 53 or matching placebo.

VYONDYS 53-treated patients received four escalating dose levels, ranging from 4 mg/kg/week (less than the recommended dosage) to 30 mg/kg/week, by intravenous infusion for 2 weeks at each dose level. Study 1 Part 2 was a 168-week, open-label study assessing the efficacy and safety of VYONDYS 53 at a dose of 30 mg/kg/week in the 12 patients enrolled in Part 1, plus 13 additional treatment-naive patients with DMD amenable to exon 53 skipping. At study entry (either in Part 1 or Part 2), patients had a median age of 8 years and were on a stable dose of corticosteroids for at least 6 months.

Efficacy was assessed based on change from baseline in the dystrophin protein level (measured as % of the dystrophin level in healthy subjects, i.e., % of normal) at Week 48 of Part 2. Muscle biopsies were obtained at baseline prior to treatment and at Week 48 of Part 2 in all VYONDYS 53-treated patients (n=25), and were analyzed for dystrophin protein level by Sarepta western blot. Mean dystrophin levels increased from 0.10% (SD 0.07) of normal at baseline to 1.02% (SD 1.03) of normal by Week 48 of Study 1 Part 2, with a mean change in dystrophin of 0.92% (SD 1.01) of normal levels (p<0.001); the median change from baseline was 0.88%.

Individual patient dystrophin levels from Study 1 are shown in Table 2 . Table 2: Dystrophin Expression Sarepta Western Blot by Individual Patient From Study 1 Patient Number Sarepta Western Blot % Normal Dystrophin Patient number Sarepta Western Blot % Normal Dystrophin Baseline Part 2 Week 48 Change from baseline Baseline Part 2 Week 48 Change from baseline 1 0.08 0.09 0.01 14 0.22 0.28 0.06 2 0.11 0.11 0.01 15 0.14 0.21 0.07 3 0.21 0.22 0.01 16 0.05 0.42 0.37 4 0.05 0.12 0.08 17 0.07 1.03 0.97 5 0.03 0.12 0.09 18 0.02 1.57 1.55 6 0.06 0.14 0.09 19 0.12 1.17 1.05 7 0.12 0.37 0.25 20 0.03 1.72 1.69 8 0.11 1.06 0.95 21 0.11 1.77 1.66 9 0.06 0.54 0.48 22 0.31 4.30 3.99 10 0.05 0.97 0.92 23 0.11 0.36 0.25 11 0.06 1.55 1.49 24 0.03 0.91 0.88 12 0.07 1.91 1.84 25 0.07 1.29 1.22 13 0.10 3.25 3.15

🧪 Nonclinical Toxicology ~2 min read ▾

13 NONCLINICAL TOXICOLOGY

13.1Carcinogenesis, Mutagenesis, Impairment of Fertility Carcinogenesis Administration of golodirsen to male transgenic (Tg.rasH2) mice (0, 100, 300, or 1000 mg/kg) weekly for 26 weeks by subcutaneous injection and to male rats (0, 30, 100, or 300 mg/kg) weekly for up to 102 weeks by intravenous injection resulted in no increase in neoplasms. Mutagenesis Golodirsen was negative in in vitro (bacterial reverse mutation and chromosomal aberration in CHO cells) and in vivo (mouse bone marrow micronucleus) assays. Impairment of Fertility Fertility studies in animals were not conducted with golodirsen.

No effects of golodirsen on the male reproductive system were observed following weekly subcutaneous administration (0, 120, 300, or 600 mg/kg to male mice or weekly intravenous administration (0, 80, 200, or 400 mg/kg) to male monkeys. Plasma exposure (AUC) at the highest doses tested in mouse and monkey are approximately 10 and 45 times that in humans at the recommended weekly intravenous dose of 30 mg/kg.

13.2Animal Toxicology and/or Pharmacology Kidney toxicity was observed in studies in male mice and rats; findings in urinary bladder were observed in male mice. In male mice, golodirsen was administered weekly for 12 weeks by intravenous injection (0, 12, 120, or 960 mg/kg) or for 26 weeks by subcutaneous injection (0, 120, 300, or 600 mg/kg). In the 12-week study, microscopic findings in kidney (tubular dilatation, basophilic or eosinophilic casts, vacuolation), correlated with increases in serum markers of renal function (e.g., urea nitrogen, creatinine), were observed primarily at the highest dose tested; hypertrophy of the transitional epithelium of the ureter or urinary bladder was observed at all doses.

In the 26-week study, renal tubular degeneration and degeneration of the transitional epithelium of the urinary bladder were observed at all doses. In male rats, intravenous administration of golodirsen (0, 60, 100, 300, or 600 mg/kg) weekly for 13 weeks resulted in tubular degeneration at all but the lowest dose tested; at the high dose, the microscopic changes were accompanied by increases in serum urea nitrogen. In male monkeys, intravenous administration of golodirsen (0, 80, 200, or 400 mg/kg) weekly for 39 weeks resulted in microscopic changes in kidney (basophilia, dilatation, or mononuclear cell infiltration) at all doses, which correlated with increases in serum markers of renal function (urea nitrogen, creatinine) at the highest dose tested.

📄 Carcinogenesis, Mutagenesis, Impairment of Fertility 156 words ▾

13.1Carcinogenesis, Mutagenesis, Impairment of Fertility Carcinogenesis Administration of golodirsen to male transgenic (Tg.rasH2) mice (0, 100, 300, or 1000 mg/kg) weekly for 26 weeks by subcutaneous injection and to male rats (0, 30, 100, or 300 mg/kg) weekly for up to 102 weeks by intravenous injection resulted in no increase in neoplasms. Mutagenesis Golodirsen was negative in in vitro (bacterial reverse mutation and chromosomal aberration in CHO cells) and in vivo (mouse bone marrow micronucleus) assays. Impairment of Fertility Fertility studies in animals were not conducted with golodirsen.

No effects of golodirsen on the male reproductive system were observed following weekly subcutaneous administration (0, 120, 300, or 600 mg/kg to male mice or weekly intravenous administration (0, 80, 200, or 400 mg/kg) to male monkeys. Plasma exposure (AUC) at the highest doses tested in mouse and monkey are approximately 10 and 45 times that in humans at the recommended weekly intravenous dose of 30 mg/kg.

📄 Recent Major Changes 19 words ▾

Dosage and Administration ( 2.4 ) 06/2024 Contraindications ( 4 ) 06/2024 Warnings and Precautions ( 5.1 ) 06/2024

📄 Package Label / Principal Display Panel 79 words ▾

Principal Display Panel - Carton Label NDC: 60923-465-02 Rx Only VYONDYS 53 (golodirsen) Injection 100 mg/2 mL (50 mg/mL) For Intravenous Infusion After Dilution Use a 0.2 micron in-line filter during infusion Single Dose 1 vial SAREPTA THERAPEUTICS Principal Display Panel - Carton Label

Principal Display Panel - Vial Label NDC: 60923-465-02 VYONDYS 53 (golodirsen) Injection 100 mg/2 mL (50 mg/mL) Single Dose. Rx Only Mfg for: Sarepta Therapeutics, Inc., Cambridge, MA 02142 Principal Display Panel - Vial Label

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
816
Units reimbursed last 4 qtrs
41.4K
Gross reimbursed last 4 qtrs
$30.8M
Avg / prescription
$37,745.54
Avg / unit
$744.83
Latest quarter Q1 2026
70Rx
Fee-for-service vs managed care ⓘ
29% FFS 71% MCO
Fee-for-service · 233 Rx Managed care · 583 Rx
State Medicaid map
Alaska: no data reported AK Maine: no data reported ME Washington: no data reported WA Idaho: no data reported ID Montana: no data reported MT North Dakota: no data reported ND Minnesota: no data reported MN Wisconsin: no data reported WI Michigan: no data reported MI New York: 1,612 units · 8.2 per 100k residents NY Vermont: no data reported VT New Hampshire: no data reported NH Oregon: no data reported OR Nevada: no data reported NV Wyoming: no data reported WY South Dakota: no data reported SD Iowa: 3,344 units · 104 per 100k residents IA Illinois: no data reported IL Indiana: 5,664 units · 82.5 per 100k residents IN Ohio: no data reported OH Pennsylvania: 2,566 units · 19.8 per 100k residents PA New Jersey: 318 units · 3.4 per 100k residents NJ Massachusetts: 5,588 units · 79.8 per 100k residents MA California: 8,218 units · 21.1 per 100k residents CA Utah: no data reported UT Colorado: no data reported CO Nebraska: no data reported NE Missouri: 1,718 units · 27.7 per 100k residents MO Kentucky: no data reported KY West Virginia: no data reported WV Virginia: no data reported VA Maryland: no data reported MD Connecticut: no data reported CT Rhode Island: no data reported RI Arizona: no data reported AZ New Mexico: no data reported NM Kansas: no data reported KS Arkansas: 1,976 units · 64.4 per 100k residents AR Tennessee: no data reported TN North Carolina: no data reported NC South Carolina: no data reported SC Delaware: 3,576 units · 347 per 100k residents DE Oklahoma: no data reported OK Louisiana: no data reported LA Mississippi: no data reported MS Alabama: no data reported AL Georgia: no data reported GA D.C.: no data reported DC Hawaii: no data reported HI Texas: 6,772 units · 22.2 per 100k residents TX Florida: no data reported FL
Units reimbursed · per 100k residents
3.4347
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 Delaware 347 /100k
2 Iowa 104 /100k
3 Indiana 82.5 /100k
4 Massachusetts 79.8 /100k
5 Arkansas 64.4 /100k
6 Missouri 27.7 /100k
7 Texas 22.2 /100k
8 California 21.1 /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 Vyondys-53 — 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 Vyondys-53. 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
$1.98M
Claims incl. refills
14
Beneficiaries
—
Spend / beneficiary
—
Spend / claim
$141,335.04
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 Vyondys 53 (this brand).

Top reported reactions

Poor Venous Access63
Intentional Dose Omission34
Device Issue30
Covid-1923
Malaise18
Pyrexia18
Vomiting16

Reporter sex

435 reports
Male · 100%

Serious outcomes

Hospitalization67
Life-threatening2
Reports over time (by year) — tap or hover for the count & year
2022 2023 2024 2026 91 0
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 Sarepta Therapeutics, Inc.. Listing status can change — the directory data on this page refreshes weekly.
Who lists this product with the FDA?
Sarepta Therapeutics, 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.
← 60923-0363-02 · Sarepta 60923-0501-10 · Sarepta →
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.