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AMONDYS 45 casimersen 50 mg/mL Injection — NDC 60923-0227-02 package photo

AMONDYS 45 casimersen 50 mg/mL Injection

by Sarepta Therapeutics, Inc. · 1 VIAL, SINGLE-USE in 1 CARTON (60923-227-02) / 2 mL in 1 VIAL, SINGLE-USE
NDC 60923-0227-02
🏷️ FDA NDC (as labeled) 60923-227-02 billing pads the product segment with a zero
Rx only Brand On market Non-controlled
🗂️ Data synced 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

🆔 Identity & classification

FDA NDC (as labeled) 60923-227-02
Product NDC 60923-227
11-digit billing NDC 60923022702
NCPDP billing unit ML — per mL (volume)
RxCUI 2480100, 2480105
UNII X8UHF7SX0R
UPC 0360923227022
Application # NDA213026
SPL Set ID e9e5fd44-eeda-4580-bba1-a734828bbcc3
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 2021-02-25
Route INTRAVENOUS
Dosage form INJECTION
Substance CASIMERSEN
GPI-14 74600025002020
GPI class Amondys 45
GCN Seq No 081971
GCN 49253
HICL code 047149
Ingredient (HICL) Casimersen
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 AMONDYS-45 100 MG/2 ML VIAL
FDB brand name Amondys-45
Legend status F — Federal legend — prescription drug or device
Why two NDCs? The FDA registers this code as 60923-227-02 — 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 → 60923-0227-02. 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.

🏭 Manufacturer & labeler

LabelerSarepta Therapeutics, Inc.
Application holderSAREPTA THERAPEUTICS INC
FDA applicationNDA213026 (NDA)
Labeler code60923
First marketedFeb 2021
Product typeHuman Prescription Drug
Portfolio65 products on file
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.

🩺 Clinical

Label name AMONDYS-45 100 MG/2 ML VIAL Ingredient Casimersen

Supplement & herbal interactions

🌿 Drug–supplement interaction checker →
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.

🧪 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.

💲 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 $811.32 $811.32 / 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.

🔁 Therapeutic equivalents

ProductLabelerPackNADAC/unitTEStatusPrice vs. this
Amondys 45 50 mg/mLthis 60923-0227-02 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

🏛️
2021
First FDA approval
Feb 2021
📍
2026
Currently FDA-listed
5 years listed
🛡️
2030
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 Nov 2030. This may affect when a full generic version becomes widely available, but it is not a guaranteed launch date.
📅 FDA approved Feb 25, 2021 RLD RS ⏳ ~4.2 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 9758783 — method of use (U-3088)
US 9758783 — method of use (U-3089)
US 10781450 — method of use (U-3089)
US RE48960 — drug substance (U-3087)
US RE48960 — drug substance (U-3088)
US 10287586 — drug substance
US 9228187 — drug substance
Exclusivity NCE
Exclusivity ODE-347
2021 2023 2025 2027 2029 2031
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 (7)
PatentTypeUse codeExpires
US 9758783 ↗ Method of use U-3088 Nov 12, 2030
US 9758783 ↗ Method of use U-3089 Nov 12, 2030
US 10781450 ↗ Method of use U-3089 Nov 12, 2030
US RE48960 ↗ Drug substance U-3087 Feb 16, 2029
US RE48960 ↗ Drug substance U-3088 Feb 16, 2029
US 10287586 ↗ Drug substance — Nov 12, 2030
US 9228187 ↗ Drug substance — Nov 12, 2030
FDA exclusivity
CodeWhat it grantsExpires
NCENew Chemical Entity (5-year)Feb 25, 2026
ODE-347Orphan Drug Exclusivity (7-year)Feb 25, 2028
Common questions
Is there a generic version of AMONDYS-45 100 MG/2 ML VIAL?
No FDA-approved generic equivalent is currently listed in the FDA Orange Book for AMONDYS-45 100 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 Nov 2030 — 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.

🗺️ 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 60923-0227-02, 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 – Q4 2025 · 4 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
3K
Units reimbursed last 4 qtrs
178.4K
Gross reimbursed last 4 qtrs
$144.71M
Avg / prescription
$47,601.76
Avg / unit
$811.32
Latest quarter Q4 2025
687Rx
Fee-for-service vs managed care ⓘ
29% FFS 71% MCO
Fee-for-service · 882 Rx Managed care · 2,158 Rx
State Medicaid map
Alaska: no data reported AK Maine: 3,568 units · 256 per 100k residents ME Washington: 4,792 units · 61.3 per 100k residents WA Idaho: 5,724 units · 291 per 100k residents ID Montana: 2,662 units · 235 per 100k residents MT North Dakota: no data reported ND Minnesota: no data reported MN Wisconsin: 6,924 units · 117 per 100k residents WI Michigan: 7,088 units · 70.6 per 100k residents MI New York: 13,372 units · 68.3 per 100k residents NY Vermont: no data reported VT New Hampshire: no data reported NH Oregon: 5,786 units · 137 per 100k residents OR Nevada: no data reported NV Wyoming: no data reported WY South Dakota: no data reported SD Iowa: no data reported IA Illinois: 1,144 units · 9.1 per 100k residents IL Indiana: no data reported IN Ohio: 18,348 units · 156 per 100k residents OH Pennsylvania: 16,036 units · 124 per 100k residents PA New Jersey: 6,924 units · 74.5 per 100k residents NJ Massachusetts: no data reported MA California: 38,736 units · 99.4 per 100k residents CA Utah: no data reported UT Colorado: no data reported CO Nebraska: no data reported NE Missouri: no data reported MO Kentucky: no data reported KY West Virginia: no data reported WV Virginia: 1,332 units · 15.3 per 100k residents VA Maryland: 1,264 units · 20.5 per 100k residents MD Connecticut: no data reported CT Rhode Island: no data reported RI Arizona: no data reported AZ New Mexico: 1,124 units · 53.2 per 100k residents NM Kansas: no data reported KS Arkansas: no data reported AR Tennessee: 87 units · 1.2 per 100k residents TN North Carolina: 5,042 units · 46.5 per 100k residents NC South Carolina: no data reported SC Delaware: no data reported DE Oklahoma: no data reported OK Louisiana: 768 units · 16.8 per 100k residents LA Mississippi: no data reported MS Alabama: 1,344 units · 26.3 per 100k residents AL Georgia: 1,528 units · 13.9 per 100k residents GA D.C.: no data reported DC Hawaii: no data reported HI Texas: 18,434 units · 60.4 per 100k residents TX Florida: 16,336 units · 72.3 per 100k residents FL
Units reimbursed · per 100k residents
1.2291
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 Idaho 291 /100k
2 Maine 256 /100k
3 Montana 235 /100k
4 Ohio 156 /100k
5 Oregon 137 /100k
6 Pennsylvania 124 /100k
7 Wisconsin 117 /100k
8 California 99.4 /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 Amondys-45 — 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 Amondys-45. 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
$4.49M
Claims incl. refills
49
Beneficiaries
14
Spend / beneficiary
$320,999.83
Spend / claim
$91,714.24
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.

📦 Packaging — all sizes for this product

Package NDCDescription Marketing startStatus
60923-0227-02 You're viewing this 1 VIAL, SINGLE-USE in 1 CARTON (60923-227-02) / 2 mL in 1 VIAL, SINGLE-USE 2021-02-25 Active

🧭 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 FDA registers it as 60923-227-02, which is what is printed on the packaging and shown on DailyMed. Insurance claims use a fixed 11-digit 5-4-2 format, so the short segment is padded with a leading zero: 60923-0227-02, written without dashes as 60923022702. The Identity section at the top of this page lists every form of this code.
What do the three segments of this NDC mean?
In 60923-0227-02, the first segment (60923) is the labeler code FDA assigned to Sarepta Therapeutics, Inc.; the middle segment (0227) identifies this specific product — its ingredient, strength, and dosage form; and the last segment (02) identifies this exact package size and type. Together they name one specific package of one specific product.
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.

📄 Full prescribing information FDA SPL

The complete FDA label for this product — the official prescribing information, verbatim, section by section. 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
🎯 Indications and Usage 166 words ▾

1 INDICATIONS AND USAGE AMONDYS 45 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 45 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with AMONDYS 45 [see Clinical Studies ( 14 )] . Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.

AMONDYS 45 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 45 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with AMONDYS 45 [see Clinical Studies ( 14 )] . 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 AMONDYS 45 ( 2.1 ) 30 milligrams per kilogram of body weight once weekly ( 2.2 ) Administer as an intravenous (IV) 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 (UPCR) should be measured before starting AMONDYS 45. Consider measurement of glomerular filtration rate prior to initiation of AMONDYS 45. Monitoring for kidney toxicity during treatment is recommended. Obtain the urine sample prior to infusion of AMONDYS 45 or at least 48 hours after infusion [see Warnings and Precautions ( 5.2 )] .

2.2Dosing Information The recommended dosage of AMONDYS 45 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 AMONDYS 45 is missed, it may be administered as soon as possible after the scheduled dose.

2.3Preparation Instructions AMONDYS 45 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 AMONDYS 45 to be administered based on the patient's weight and the recommended dose of 30 milligrams per kilogram. Determine the volume of AMONDYS 45 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 AMONDYS 45.

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 AMONDYS 45 from the appropriate number of vials.

To avoid dulling the needle and fragmenting the stoppers, replace the needle periodically during preparation. Dilute the withdrawn AMONDYS 45 in 0.9% Sodium Chloride Injection, USP, to make a total volume of 100 to 150 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. AMONDYS 45 contains no preservatives and should be administered immediately after dilution. Complete infusion of diluted AMONDYS 45 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 AMONDYS 45.

2.4Administration Instructions Application of a topical anesthetic cream to the infusion site prior to administration of AMONDYS 45 may be considered. AMONDYS 45 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 AMONDYS 45 over 35 to 60 minutes via an in-line 0.2 micron filter. Do not mix other medication with AMONDYS 45 or infuse other medications concomitantly via the same intravenous access with AMONDYS 45. If a hypersensitivity reaction occurs, consider slowing the infusion, interrupting or discontinuing the AMONDYS 45 therapy [see Contraindications ( 4 ), Warning and Precautions ( 5.1 ) and Adverse Reactions ( 6.2 )] .

💊 Dosage Forms and Strengths 54 words ▾

3 DOSAGE FORMS AND STRENGTHS AMONDYS 45 is a clear to slightly opalescent, colorless liquid and may contain trace amounts of small, white to off-white amorphous particles and is available as: Injection: 100 mg/2 mL (50 mg/ mL) solution in a single-dose vial Injection: 100 mg/2 mL in a single-dose vial ( 3 )

⛔ Contraindications 72 words ▾

4 CONTRAINDICATIONS AMONDYS 45 is contraindicated in patients with known serious hypersensitivity to casimersen or to any of the inactive ingredients in AMONDYS 45. Instances of hypersensitivity, including angioedema and anaphylaxis, have occurred in patients receiving AMONDYS 45 [see Warnings and Precautions ( 5.1 )] . AMONDYS 45 is contraindicated in patients with serious hypersensitivity to casimersen or to any of the inactive ingredients in AMONDYS 45.

( 4 , 5.1 )

⚠️ Warnings and Cautions ~2 min read ▾

5 WARNINGS AND PRECAUTIONS Hypersensitivity Reactions: Hypersensitivity reactions, including angioedema and anaphylaxis, have occurred in patients who were treated with AMONDYS 45. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion, interrupting or discontinuing the AMONDYS 45 infusion ( 2.4 , 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 angioedema and anaphylaxis, have occurred in patients who were treated with AMONDYS 45. If a hypersensitivity reaction occurs, institute appropriate medical treatment, and consider slowing the infusion [see Dosage and Administration ( 2.4 )] , interrupting, or discontinuing the AMONDYS 45 infusion and monitor until the condition resolves. AMONDYS 45 is contraindicated in patients with known serious hypersensitivity to casimersen or to any of the inactive ingredients in AMONDYS 45 [see Contraindications ( 4 )] .

5.2Kidney Toxicity Kidney toxicity was observed in animals who received casimersen [see Use in Specific Populations ( 8.4 ) and Nonclinical Toxicology ( 13.2 )] . Although kidney toxicity was not observed in the clinical studies with AMONDYS 45, kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. Kidney function should be monitored in patients taking AMONDYS 45.

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 AMONDYS 45. Consider also measuring glomerular filtration rate using an exogenous filtration marker before starting AMONDYS 45.

During treatment, monitor urine dipstick every month, and serum cystatin C and urine protein-to-creatinine ratio (UPCR) every three months. Only urine expected to be free of excreted AMONDYS 45 should be used for monitoring of urine protein. Urine obtained on the day of AMONDYS 45 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 AMONDYS 45 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 clinically significant adverse reactions are described elsewhere in the labeling: Hypersensitivity reactions [see Warnings and Precautions ( 5.1 )] The most common adverse reactions (incidence >20% and at least 5% higher than placebo) were upper respiratory tract infection, cough, pyrexia, headache, arthralgia, and oropharyngeal pain. ( 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 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 AMONDYS 45 clinical development program, 76 patients received at least one intravenous dose of AMONDYS 45 (30 mg/kg). All patients were male and had genetically confirmed Duchenne muscular dystrophy.

Age at study entry was 7 to 20 years (mean 9.9 years). Most (88%) patients were White, and 9% were Asian. AMONDYS 45 was studied in a double-blind, placebo-controlled study (Study 1).

Patients in ongoing Study 1 received AMONDYS 45 (n=57) 30 mg/kg or placebo (n=31) intravenously once weekly for up to 96 weeks, after which all patients received or will receive AMONDYS 45 30 mg/kg for up to 48 weeks. Adverse reactions observed in ≥20% of patients treated with AMONDYS 45 and 5% more frequently than in the placebo group in Study 1 are shown in Table 1 . Table 1.

Adverse Reactions Occurring in at Least 20% of Patients Treated with AMONDYS 45 and at a Rate at Least 5% More Frequently than in the Placebo Group in Study 1 *Includes upper respiratory infection, pharyngitis, nasopharyngitis, and rhinitis. Adverse Reaction AMONDYS 45 30 mg/kg Once Weekly (n = 57) % Placebo (n = 31) % Upper Respiratory Tract Infections* 65 55 Cough 33 26 Pyrexia 33 23 Headache 32 19 Arthralgia 21 10 Oropharyngeal Pain 21 7 Other adverse reactions that occurred in at least 10% of patients treated with AMONDYS 45, and that were reported at a rate at least 5% more frequently in the AMONDYS 45 group than in the placebo group, were: ear pain, nausea, ear infection, post-traumatic pain, and dizziness and light-headedness.

6.2Postmarketing Experience The following adverse reactions have been identified during postapproval use of AMONDYS 45. 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. Infusion-related reactions including rash, headache, cough, abdominal pain (including upper abdominal pain), and vomiting occurred within 24 hours from the start of an infusion of AMONDYS 45.

Hypersensitivity reactions, including angioedema and anaphylaxis, have occurred in patients treated with AMONDYS 45.

👥 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 AMONDYS 45 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 AMONDYS 45 on milk production, the presence of casimersen in milk, or the effects of AMONDYS 45 on the breastfed infant. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for AMONDYS 45 and any potential adverse effects on the breastfed infant from AMONDYS 45 or from the underlying maternal condition.

8.4Pediatric Use AMONDYS 45 is indicated for the treatment of DMD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping, including pediatric patients [see Clinical Studies ( 14 )]. Juvenile Animal Toxicity Data Intravenous administration of casimersen (0, 100, 300, and 900 mg/kg) to juvenile male rats once weekly for 10 weeks (postnatal days 14 to 77) resulted in renal tubular degeneration/necrosis at the highest dose tested. No effects were observed on the male reproductive system, neurobehavioral development, or immune function.

At the overall no-effect dose (300 mg/kg), plasma exposure (AUC) was 4 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 experience with AMONDYS 45 in geriatric DMD patients.

8.6Patients with Renal Impairment Renal clearance of casimersen is decreased 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 AMONDYS 45.

🤰 Pregnancy 45 words ▾

8.1Pregnancy Risk Summary There are no human or animal data available to assess the use of AMONDYS 45 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 115 words ▾

8.4Pediatric Use AMONDYS 45 is indicated for the treatment of DMD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping, including pediatric patients [see Clinical Studies ( 14 )]. Juvenile Animal Toxicity Data Intravenous administration of casimersen (0, 100, 300, and 900 mg/kg) to juvenile male rats once weekly for 10 weeks (postnatal days 14 to 77) resulted in renal tubular degeneration/necrosis at the highest dose tested. No effects were observed on the male reproductive system, neurobehavioral development, or immune function.

At the overall no-effect dose (300 mg/kg), plasma exposure (AUC) was 4 times that in humans at the recommended human dose of 30 mg/kg/week.

🧓 Geriatric Use 25 words ▾

8.5Geriatric Use DMD is largely a disease of children and young adults; therefore, there is no experience with AMONDYS 45 in geriatric DMD patients.

🧬 Clinical Pharmacology ~3 min read ▾

12 CLINICAL PHARMACOLOGY

12.1Mechanism of Action Casimersen is designed to bind to exon 45 of dystrophin pre-mRNA resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 45 skipping. Exon 45 skipping is intended to allow for production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 45 skipping [see Clinical Studies ( 14 )] .

12.2Pharmacodynamics In the interim analysis of muscle biopsy tissue obtained at baseline and at Week 48 from patients in Study 1, patients who received AMONDYS 45 (n=27) demonstrated a significant increase in skipping of exon 45 (p<0.001) compared to baseline, demonstrated by reverse transcription digital droplet polymerase chain reaction (RT-ddPCR). Patients who received placebo (n=16) did not demonstrate a significant increase in exon 45 skipping (p=0.808). The level of exon skipping is positively correlated with dystrophin protein expression [see Clinical Studies ( 14 )] .

In Study 1 [see Clinical Studies ( 14 )] , dystrophin levels as assessed by the Sarepta Western blot assay increased from 0.93% (SD 1.67) of normal at baseline to 1.74% (SD 1.97) of normal after 48 weeks of treatment with AMONDYS 45. The mean change from baseline in dystrophin after 48 weeks of treatment with AMONDYS 45 was 0.81% (SD 0.70) of normal levels (p<0.001). This increase in dystrophin protein expression after treatment with AMONDYS 45 positively correlated with the level of exon skipping.

The mean change from baseline in dystrophin after 48 weeks of treatment with placebo was 0.22% (SD 0.49). Patients who received AMONDYS 45 showed a significantly greater increase in dystrophin protein levels from baseline to Week 48 compared to those who received placebo (mean difference of 0.59%; p = 0.004). 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 dystrophin to the sarcolemma in patients treated with AMONDYS 45 was demonstrated by immunofluorescence staining.

12.3Pharmacokinetics The pharmacokinetics of casimersen was evaluated in DMD patients following administration of intravenous (IV) doses ranging from 4 mg/kg/week to 30 mg/kg/week (i.e., recommended dosage). Following a single IV dose of casimersen, C max was reached at the end of infusion. Casimersen exposure increased in a proportional manner with dose increment.

No accumulation of casimersen was observed in plasma following once weekly dosing. Inter-subject variability (as %CV) for casimersen C max and AUC ranged from 12% to 34% and 16% to 34%, respectively. Distribution Binding of casimersen to human plasma protein was not concentration-dependent and ranged from 8.4% to 31.6%.

The mean apparent volume of distribution at steady state (V ss ) was 367 mL/kg (%CV = 28.9) following a 30 mg/kg dose of casimersen administered intravenously. Elimination The plasma clearance (CL) of casimersen was 180 mL/hr/kg at the 30 mg/kg dose. The elimination half-life (t 1/2 ) was 3.5 hours (SD 0.4 hours).

Metabolism Casimersen is metabolically stable in human hepatic microsomal incubations. No metabolites were detected in plasma or urine. Excretion Casimersen is mostly excreted unchanged in the urine.

In a clinical study with radiolabeled casimersen, more than 90% of the drug was excreted in urine, with negligible fecal excretion. Specific Populations Age, Sex & Race The pharmacokinetics of AMONDYS 45 have been evaluated in male DMD patients 9 to 20 years of age. There is no experience with the use of AMONDYS 45 in DMD patients 65 years of age or older.

AMONDYS 45 has not been studied in female patients. The potential impact of race on the pharmacokinetics of…

🧬 Mechanism of Action 70 words ▾

12.1Mechanism of Action Casimersen is designed to bind to exon 45 of dystrophin pre-mRNA resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 45 skipping. Exon 45 skipping is intended to allow for production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 45 skipping [see Clinical Studies ( 14 )] .

📦 How Supplied / Storage and Handling 124 words ▾

16 HOW SUPPLIED/STORAGE AND HANDLING

16.1How Supplied AMONDYS 45 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/2 mL (50 mg/mL) NDC 60923-227-02

16.2Storage and Handling Store AMONDYS 45 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.

16.1How Supplied AMONDYS 45 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/2 mL (50 mg/mL) NDC 60923-227-02

📦 Storage and Handling 29 words ▾

16.2Storage and Handling Store AMONDYS 45 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 AMONDYS 45 (casimersen) injection is a sterile, aqueous, preservative-free, concentrated solution for dilution prior to intravenous administration. AMONDYS 45 is a clear to slightly opalescent, colorless liquid and may contain trace amounts of small, white to off-white amorphous particles. AMONDYS 45 is supplied in single-dose vials containing 100 mg casimersen (50 mg/mL).

AMONDYS 45 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 AMONDYS 45 contains: 50 mg casimersen; 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.

Casimersen 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). Casimersen contains 22 linked subunits. The sequence of bases from the 5' end to 3' end is CAATGCCATCCTGGAGTTCCTG.

The molecular formula of casimersen is C 268 H 424 N 124 O 95 P 22 and the molecular weight is 7584.5 daltons. The structure of casimersen is: Structural formula

💬 Information for Patients 139 words ▾

17 PATIENT COUNSELING INFORMATION Hypersensitivity Reactions Advise patients and/or caregivers that hypersensitivity reactions, including angioedema and anaphylaxis, have occurred in patients who were treated with AMONDYS 45. 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 AMONDYS 45.

Advise patients of the importance of monitoring for kidney toxicity by their healthcare providers during treatment with AMONDYS 45 [see Warnings and Precautions ( 5.2 )] . Manufactured for: Sarepta Therapeutics, Inc. Cambridge, MA 02142 USA Sarepta and Sarepta Therapeutics are trademarks of Sarepta Therapeutics, Inc. registered in the U.S.

Patent and Trademark Office and may be registered in various other jurisdictions. AMONDYS 45 and the AMONDYS 45 logo are trademarks of Sarepta Therapeutics, Inc. logo

Source: FDA Structured Product Labeling, mirrored from DailyMed / openFDA. Prefer the government’s original formatting? View this label on DailyMed ↗
← 60913-0999-02 · PHOENIX 60923-0284-10 · 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.