NDC 60923-0363-02 — Exondys 51 eteplirsen 50 mg/mL Injection

🆔 Identity & classification

FDA NDC (as labeled) 60923-363-02

Product NDC 60923-363

11-digit billing NDC 60923036302

NCPDP billing unit ML — per mL (volume)

RxCUI 1810573, 1810578, 1810581, 1810582

UNII AIW6036FAS

UPC 0360923284100, 0360923363027

Application # NDA206488

SPL Set ID 33bff678-7829-479e-9110-b8e33a0bc0aa

Established class (EPC) Antisense Oligonucleotide

Chemical class Oligonucleotides, Antisense

DEA schedule Non-controlled

Marketing category NDA

Marketing status On market

FDA listing status Listed (active directory)

Marketing start 2016-09-19

Route INTRAVENOUS

Dosage form INJECTION

Substance ETEPLIRSEN

GPI-14 74600035002020

GPI class Exondys 51

GCN Seq No 076605

GCN 42295

HICL code 043770

Ingredient (HICL) Eteplirsen

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 EXONDYS-51 100 MG/2 ML VIAL

FDB brand name Exondys-51

Legend status F — Federal legend — prescription drug or device

Why two NDCs? The FDA registers this code as 60923-363-02 — 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 → 60923-0363-02. 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 applicationNDA206488 (NDA)

Labeler code60923

First marketedSep 2016

Product typeHuman Prescription Drug

Portfolio65 products on file

🔎 All NDCs from Sarepta Therapeutics, Inc.

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 EXONDYS-51 100 MG/2 ML VIAL Ingredient Eteplirsen

Patient education

📘 MedlinePlus patient leaflet ↗

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 system	Per each	Per package
Retail pharmacies payNADAC · weekly	Not in the retail survey — common for institutional, discontinued, or low-volume packs.
Medicaid paysCMS SDUD · 12 mo	$784.83	$784.83 / 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.

🔁 Therapeutic equivalents

Product	Labeler	Pack	NADAC/unit	TE	Status	Price vs. this
Exondys 51 50 mg/mLthis 60923-0363-02	Sarepta	1 vial	—	—	FDA listed	—
Exondys 51 50 mg/mL 60923-0284-10	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

🏛️

2016

First FDA approval

Sep 2016

📍

2026

Currently FDA-listed

10 years listed

🛡️

2034

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 Mar 2034. This may affect when a full generic version becomes widely available, but it is not a guaranteed launch date.

📅 FDA approved Sep 19, 2016 RLD RS ⏳ ~7.5 yr to latest listed protection

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

Patents & exclusivity — FDA Orange Book

US 9506058 — method of use (U-1918)

US 9506058 — method of use (U-1919)

US 10364431 — method of use (U-1919)

US 10364431 — method of use (U-1918)

US 10364431 — method of use (U-1919)

US 10364431 — method of use (U-1918)

US 10337003 — method of use (U-1918)

US RE48468 — method of use (U-2097)

US RE47769 — drug product

2016 2018 2020 2022 2024 2026 2028 2030 2032 2034

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 (14)

Patent	Type	Use code	Expires
US 9506058 ↗	Method of use	U-1918	Mar 14, 2034
US 9506058 ↗	Method of use	U-1918	Mar 14, 2034
US 9506058 ↗	Method of use	U-1919	Mar 14, 2034
US 9506058 ↗	Method of use	U-1919	Mar 14, 2034
US 10364431 ↗	Method of use	U-1919	Mar 14, 2034
US 10364431 ↗	Method of use	U-1918	Mar 14, 2034
US 10364431 ↗	Method of use	U-1919	Mar 14, 2034
US 10364431 ↗	Method of use	U-1918	Mar 14, 2034
US 10337003 ↗	Method of use	U-1918	Mar 14, 2034
US 10337003 ↗	Method of use	U-1918	Mar 14, 2034
US RE48468 ↗	Method of use	U-2097	Oct 27, 2028
US RE48468 ↗	Method of use	U-2097	Oct 27, 2028
US RE47769 ↗	Drug product	—	Feb 2, 2029
US RE47769 ↗	Drug product	—	Feb 2, 2029

Common questions

Is there a generic version of EXONDYS-51 100 MG/2 ML VIAL?

No FDA-approved generic equivalent is currently listed in the FDA Orange Book for EXONDYS-51 100 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 Mar 2034 — 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 60923-0363-02, 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

2.8K

Units reimbursed last 4 qtrs

29.9K

Gross reimbursed last 4 qtrs

$23.49M

Avg / prescription

$8,327.41

Avg / unit

$784.82

Latest quarter Q4 2025

624Rx

Fee-for-service vs managed care ⓘ

35% FFS 65% MCO

Fee-for-service · 978 Rx Managed care · 1,843 Rx

State Medicaid map

Units reimbursed · per 100k residents

1.341.3

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 Arizona 41.3 /100k

2 Missouri 32.2 /100k

3 Tennessee 25.9 /100k

4 Indiana 21.9 /100k

5 Virginia 18.6 /100k

6 Michigan 16.8 /100k

7 California 16.0 /100k

8 Illinois 13.8 /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 Exondys-51 — 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 Exondys-51. 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

$7.16M

Claims incl. refills

123

Beneficiaries

19

Spend / beneficiary

$376,657.23

Spend / claim

$58,182.82

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 Exondys 51 (this brand).

Top reported reactions

Intentional Dose Omission279

Poor Venous Access187

Device Issue182

Pyrexia108

Covid-1987

Malaise74

Pneumonia62

Age at onset

Adult1

Reporter sex

Male · 98%

Female · 2%

Unknown · 0%

Serious outcomes

Hospitalization343

Disabling2

Reports over time (by year) — tap or hover for the count & year

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.

📦 Packaging — all sizes for this product

Package NDC	Description	Marketing start	Status
60923-0363-02 You're viewing this	1 VIAL, SINGLE-USE in 1 CARTON (60923-363-02) / 2 mL in 1 VIAL, SINGLE-USE	2016-09-19	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 60923-363-02, 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: 60923-0363-02, written without dashes as 60923036302. 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 60923-0363-02, the first segment (60923) is the labeler code FDA assigned to Sarepta Therapeutics, Inc.; the middle segment (0363) 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 165 words ▾

1 INDICATIONS AND USAGE EXONDYS 51 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 51 skipping. This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with EXONDYS 51 [see Clinical Studies ( 14 )] . Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.

EXONDYS 51 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 51 skipping. This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with EXONDYS 51 [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 ~2 min read ▾

2 DOSAGE AND ADMINISTRATION 30 milligrams per kilogram of body weight once weekly ( 2.1 ) Administer as an intravenous infusion over 35 to 60 minutes via an in-line 0.2 micron filter ( 2.1 , 2.3 ) Dilution required prior to administration ( 2.2 )

2.1Dosing Information The recommended dose of EXONDYS 51 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 EXONDYS 51 is missed, it may be administered as soon as possible after the scheduled time.

2.2Preparation Instructions EXONDYS 51 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 EXONDYS 51 to be administered based on the patient's weight and the recommended dose of 30 milligrams per kilogram. Determine the volume of EXONDYS 51 needed and the correct number of vials to supply the full calculated dose. Allow 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 EXONDYS 51.

EXONDYS 51 is a clear, colorless solution that may have some opalescence, and may contain 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 white to off-white amorphous particles. With a syringe fitted with a 21-gauge or smaller non-coring needle, withdraw the calculated volume of EXONDYS 51 from the appropriate number of vials.

Dilute the withdrawn EXONDYS 51 in 0.9% Sodium Chloride Injection, USP, to make a total volume of 100-150 mL. Visually inspect the diluted solution. Do not use if the solution is cloudy, discolored or contains extraneous particulate matter other than white to off-white amorphous particles.

Administer the diluted solution via an in-line 0.2 micron filter. EXONDYS 51 contains no preservatives and should be administered immediately after dilution. Complete infusion of diluted EXONDYS 51 solution within 8 hours of dilution.

If immediate use is not possible, the diluted solution may be stored for up to 24 hours at 2ºC to 8ºC (36ºF to 46ºF). Do not freeze. Discard unused EXONDYS 51.

2.3Administration Instructions Application of a topical anesthetic cream to the infusion site prior to administration of EXONDYS 51 may be considered. EXONDYS 51 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 EXONDYS 51 solution over 35 to 60 minutes via an in-line 0.2 micron filter. Do not mix other medications with EXONDYS 51 or infuse other medications concomitantly via the same intravenous access line. If a hypersensitivity reaction occurs, consider slowing the infusion or interrupting the EXONDYS 51 therapy [see Warnings and Precautions ( 5.1 ) and Adverse Reactions ( 6.1 )].

💊 Dosage Forms and Strengths 76 words ▾

3 DOSAGE FORMS AND STRENGTHS EXONDYS 51 is a clear and colorless solution that may have some opalescence, and may contain white to off-white amorphous particles, and is available as follows: Injection: 100 mg/2 mL (50 mg/mL) solution in a single-dose vial Injection: 500 mg/10 mL (50 mg/mL) solution in a single-dose vial Injection: 100 mg/2 mL (50 mg/mL) in single-dose vial ( 3 ) 500 mg/10 mL (50 mg/mL) in single-dose vial ( 3 )

⛔ Contraindications 7 words ▾

4 CONTRAINDICATIONS None. None ( 4 )

⚠️ Warnings and Cautions 99 words ▾

5 WARNINGS AND PRECAUTIONS Hypersensitivity Reactions: Hypersensitivity reactions, including bronchospasm, chest pain, cough, tachycardia, and urticaria, have occurred in patients treated with EXONDYS 51. If hypersensitivity reactions occur, institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy. ( 2.3 , 5.1 )

5.1Hypersensitivity Reactions Hypersensitivity reactions, including bronchospasm, chest pain, cough, tachycardia, and urticaria, have occurred in patients who were treated with EXONDYS 51. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy [see Dosage and Administration ( 2.3 )] .

🤒 Adverse Reactions ~3 min read ▾

6 ADVERSE REACTIONS The most common adverse reactions (incidence ≥35% and higher than placebo) were balance disorder and vomiting ( 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. EXONDYS 51 was studied in a double-blind, placebo-controlled study for 24 weeks (Study 1), followed by an open label extension (Study 2). In Study 1, 12 patients were randomized to receive weekly intravenous infusions of EXONDYS 51 (n=8) or placebo (n=4) for 24 weeks.

All 12 patients continued in Study 2 and received open-label EXONDYS 51 weekly for up to 208 weeks. In Study 1, 4 patients received placebo, 4 patients received EXONDYS 51 30 mg/kg, and 4 patients received EXONDYS 51 50 mg/kg (1.7 times the recommended dosage). In Study 2, 6 patients received EXONDYS 51 30 mg/kg/week and 6 patients received EXONDYS 51 50 mg/kg/week [see Clinical Studies ( 14 )] .

Adverse reactions that occurred in 2 or more patients who received EXONDYS 51 and were more frequent than in the placebo group in Study 1 are presented in Table 1 (the 30 and 50 mg/kg groups are pooled). Because of the small numbers of patients, these represent crude frequencies that may not reflect the frequencies observed in practice. The 50 mg/kg once weekly dosing regimen of EXONDYS 51 is not recommended [see Dosage and Administration ( 2.1 )].

The most common adverse reactions were balance disorder and vomiting. Table 1. Adverse Reactions in DMD Patients Treated with 30 or 50 mg/kg/week 1 EXONDYS 51 with Incidence at Least 25% More than Placebo (Study 1) 1 50 mg/kg/week = 1.7 times the recommended dosage Adverse Reactions EXONDYS 51 (N=8) Placebo (N=4) % % Balance disorder 38 0 Vomiting 38 0 Contact dermatitis 25 0 Adverse Reactions from Observational Clinical Studies The following adverse reactions have been identified during observational studies that were conducted as part of the clinical development program and continued postapproval.

In open-label observational studies, 163 patients received at least one intravenous dose of EXONDYS 51, with doses ranging between 0.5 mg/kg (0.017 times the recommended dosage) and 50 mg/kg (1.7 times the recommended dosage). All patients were male and had genetically confirmed Duchenne muscular dystrophy. Age at study entry was 6 months to 19 years.

Most (85%) patients were Caucasian. The most common adverse reactions seen in greater than 10% of the study population were headache, cough, rash, and vomiting. Hypersensitivity reactions have occurred in patients treated with EXONDYS 51 [see Warnings and Precautions ( 5.1 )] .

6.2Postmarketing Experience The following adverse reactions have been identified during postapproval use of EXONDYS 51. 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. Postmarketing adverse reactions that occurred during infusion include bronchospasm, cyanosis of the lips, and malaise.

The following adverse reactions have also been reported in patients receiving EXONDYS 51: pyrexia, flushing, protein urine present, and dehydration.

👥 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 EXONDYS 51 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 EXONDYS 51 on milk production, the presence of eteplirsen in milk, or the effects of EXONDYS 51 on the breastfed infant. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for EXONDYS 51 and any potential adverse effects on the breastfed infant from EXONDYS 51 or from the underlying maternal condition.

8.4Pediatric Use EXONDYS 51 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 51 skipping, including pediatric patients [see Clinical Studies ( 14 )] . Intravenous administration of eteplirsen (0, 100, 300, or 900 mg/kg) to juvenile male rats once weekly for 10 weeks beginning on postnatal day 14 resulted in renal tubular necrosis at the highest dose tested and decreased bone densitometry parameters (mineral density, mineral content, area) at all doses.

The kidney findings were associated with clinical pathology changes (increased serum urea nitrogen and creatinine, decreased urine creatinine clearance). No effects were observed on the male reproductive system, neurobehavioral development, or immune function. An overall no-effect dose was not identified.

Plasma eteplirsen exposure (AUC) at the lowest dose tested (100 mg/kg) was similar to that in humans at the recommended human dose (30 mg/kg).

8.5Geriatric Use DMD is largely a disease of children and young adults; therefore, there is no geriatric experience with EXONDYS 51.

8.6Patients with Renal Impairment Renal clearance of eteplirsen is reduced in non-DMD adults with renal impairment based on estimated creatinine clearance [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.

🤰 Pregnancy 45 words ▾

8.1Pregnancy Risk Summary There are no human or animal data available to assess the use of EXONDYS 51 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 154 words ▾

8.4Pediatric Use EXONDYS 51 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 51 skipping, including pediatric patients [see Clinical Studies ( 14 )] . Intravenous administration of eteplirsen (0, 100, 300, or 900 mg/kg) to juvenile male rats once weekly for 10 weeks beginning on postnatal day 14 resulted in renal tubular necrosis at the highest dose tested and decreased bone densitometry parameters (mineral density, mineral content, area) at all doses.

The kidney findings were associated with clinical pathology changes (increased serum urea nitrogen and creatinine, decreased urine creatinine clearance). No effects were observed on the male reproductive system, neurobehavioral development, or immune function. An overall no-effect dose was not identified.

Plasma eteplirsen exposure (AUC) at the lowest dose tested (100 mg/kg) was similar to that in humans at the recommended human dose (30 mg/kg).

🧓 Geriatric Use 22 words ▾

8.5Geriatric Use DMD is largely a disease of children and young adults; therefore, there is no geriatric experience with EXONDYS 51.

🧬 Clinical Pharmacology ~3 min read ▾

12 CLINICAL PHARMACOLOGY

12.1Mechanism of Action Eteplirsen is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein, which was evaluated in Study 2 and Study 3 [ see Clinical Studies ( 14 ) ].

12.2Pharmacodynamics All EXONDYS 51-treated patients evaluated (n=36) were found to produce messenger ribonucleic acid (mRNA) for a truncated dystrophin protein by reverse transcription polymerase chain reaction. In Study 2, the average dystrophin protein level in muscle tissue after 180 weeks of treatment with EXONDYS 51 was 0.93% of normal (i.e., 0.93% of the dystrophin level in healthy subjects). Because of insufficient information on dystrophin protein levels before treatment with EXONDYS 51 in Study 1, it is not possible to estimate dystrophin production in response to EXONDYS 51 in Study 1.

In Study 3, the average dystrophin protein level was 0.16% of normal before treatment, and 0.44% of normal after 48 weeks of treatment with EXONDYS 51 [see Clinical Studies ( 14 )]. The median increase in truncated dystrophin in Study 3 was 0.1% [see Clinical Studies ( 14 )] . 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.

12.3Pharmacokinetics Following single or multiple intravenous infusions of EXONDYS 51 in male pediatric DMD patients, plasma concentration-time profiles of eteplirsen were generally similar and showed multi-phasic decline. The majority of drug elimination occurred within 24 hours. Approximate dose-proportionality and linearity in PK properties were observed following multiple-dose studies (0.5 mg/kg/week [0.017 times the recommended dosage] to 50 mg/kg/week [1.7 times the recommended dosage]).

There was no significant drug accumulation following weekly dosing across this dose range. The inter-subject variability for eteplirsen C max and AUC range from 20 to 55%. Following single or multiple intravenous infusions of EXONDYS 51, the peak plasma concentrations (C max ) of eteplirsen occurred near the end of infusion (i.e., 1.1 to 1.2 hours across a dose range of 0.5 mg/kg/week to 50 mg/kg/week).

Distribution In vitro investigation suggested that plasma protein binding of eteplirsen in human ranges between 6 to 17%. The mean apparent volume of distribution (Vss) of eteplirsen was 600 mL/kg following weekly intravenous infusion of EXONDYS 51 at 30 mg/kg. Twenty-four hours after the end of the infusion, mean concentrations of eteplirsen were 0.07% of C max .

Accumulation of eteplirsen during once weekly dosing has not been observed. Elimination The total clearance of eteplirsen was 339 mL/hr/kg following 12 weeks of therapy with 30 mg/kg/week. Metabolism Eteplirsen did not appear to be metabolized by hepatic microsomes of any species tested, including humans.

Excretion Renal clearance of eteplirsen accounts for approximately two-thirds of the administered dose within 24 hours of intravenous administration. Elimination half-life (t 1/2 ) of eteplirsen was 3 to 4 hours. Specific Populations Age: The pharmacokinetics of eteplirsen have been evaluated in male pediatric DMD patients.

There is no experience with the use of EXONDYS 51 in patients 65 years of age or older. Sex: Sex effects have not been evaluated; EXONDYS 51 has not been studied in female patients. Race: Potential impact of race is not known because 89% of the patients in studies were Caucasians.

Patients with Renal Impairment: The effect of renal impairment on the pharmacokinetics of eteplirsen was evaluated in non-DMD subjects aged 51 to 75 years with mild (n=8…

🧬 Mechanism of Action 67 words ▾

12.1Mechanism of Action Eteplirsen is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein, which was evaluated in Study 2 and Study 3 [ see Clinical Studies ( 14 ) ].

📦 How Supplied / Storage and Handling 89 words ▾

16 HOW SUPPLIED/STORAGE AND HANDLING

16.1How Supplied EXONDYS 51 injection is supplied in single-dose vials. The solution is clear and colorless, and may have some opalescence, and may contain white to off-white amorphous particles. Single-dose vials containing 100 mg/2 mL (50 mg/mL) eteplirsen NDC 60923-363-02 Single-dose vials containing 500 mg/10 mL (50 mg/mL) eteplirsen NDC 60923-284-10

16.2Storage and Handling Store EXONDYS 51 at 2°C to 8°C (36°F to 46°F). Do not freeze. Protect from light and store EXONDYS 51 in the original carton until ready for use.

📦 Storage and Handling 32 words ▾

16.2Storage and Handling Store EXONDYS 51 at 2°C to 8°C (36°F to 46°F). Do not freeze. Protect from light and store EXONDYS 51 in the original carton until ready for use.

📋 Description ~1 min read ▾

11 DESCRIPTION EXONDYS 51 (eteplirsen) injection is a sterile, aqueous, preservative-free, concentrated solution for dilution prior to intravenous administration. EXONDYS 51 is clear and colorless, and may have some opalescence, and may contain white to off-white amorphous particles. EXONDYS 51 is supplied in single dose vials containing 100 mg or 500 mg eteplirsen (50 mg/mL).

EXONDYS 51 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 EXONDYS 51 contains 50 mg eteplirsen; 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.

Eteplirsen 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). Eteplirsen contains 30 linked subunits. The molecular formula of eteplirsen is C 364 H 569 N 177 O 122 P 30 and the molecular weight is 10305.7 daltons.

The structure and base sequence of eteplirsen are: Structure and Base Sequence of Eteplirsen

💬 Information for Patients 95 words ▾

17 PATIENT COUNSELING INFORMATION Hypersensitivity Reactions Advise patients and/or caregivers that symptoms of hypersensitivity, including bronchospasm, chest pain, cough, tachycardia, and urticaria can occur with EXONDYS 51. Instruct them to seek immediate medical care should they experience signs and symptoms of hypersensitivity [see Warnings and Precautions ( 5.1 )] . Manufactured for: Sarepta Therapeutics, Inc.

Cambridge, MA 02142 USA SAREPTA, SAREPTA THERAPEUTICS, EXONDYS, EXONDYS 51, and the EXONDYS 51 Logo are trademarks of Sarepta Therapeutics, Inc. registered in the U.S. Patent and Trademark Office and may be registered in various other jurisdictions. Exondys 51 Logo

Glossary — plain-language definitions of medical terms used in this label:

in vitro: In a laboratory dish or test tube rather than in a living person or animal; results may not carry over to people.

accumulation: A buildup of a medicine in the body when new doses are taken before earlier ones are fully removed.

adverse reaction: An unwanted or harmful effect that a medicine causes in the body.

Adverse Reactions: Unwanted or harmful effects that people experienced while taking a medicine, listed on its official label.

AUC: A measure of total drug exposure over time, showing how much medicine the blood is exposed to overall.

bronchospasm: A sudden tightening of the muscles around the airways in the lungs that narrows them and makes breathing harder.

clearance: How quickly the body removes a medicine from the blood, usually through the liver and kidneys.

Clinical Pharmacology: The label section explaining how a medicine works in the body and how the body processes it.

Clinical Studies: The label section summarizing the research trials used to show that a medicine works and is acceptably safe.

contact dermatitis: A skin rash caused by touching something that irritates the skin or triggers an allergy.

Contraindications: A list of situations or health conditions in which a medicine should not be used because the chance of harm is too high.

creatinine clearance: An estimate of how fast the kidneys filter waste from the blood, often used to choose the right medicine dose.

cyanosis: A bluish color of the skin, lips, or nails caused by too little oxygen in the blood.

dermatitis: Inflammation of the skin, causing redness, itching, and sometimes swelling or blisters.

Distribution: How a medicine spreads from the blood into the body's tissues and organs after it is absorbed.

Dosage and Administration: The label section explaining how much of a medicine to use and how to take or give it.

double-blind: A study in which neither the patients nor their doctors know who is getting the real medicine and who is getting the dummy.

Elimination: The overall removal of a medicine from the body by breaking it down and passing it out.

Excretion: How the body removes a medicine or its leftover pieces, mainly through the urine or stool.

flushing: Temporary redness and warmth of the skin, often on the face and neck, from widening of small blood vessels.

Half-life: The time it takes for the amount of medicine in the blood to drop by half.

How Supplied/Storage and Handling: The label section describing the forms and strengths a medicine comes in and how to store it properly.

hypersensitivity: An overreaction of the body's defense system to a substance, causing symptoms such as rash, swelling, or trouble breathing.

incidence: How often a new event, such as a side effect, happens in a group of people over a set period of time.

Indications and Usage: The label section stating which medical conditions the FDA has officially approved the medicine to treat, prevent, or manage.

infusion: Slowly delivering fluid or medicine into a vein over a period of time, usually through a drip.

intravenous: Given directly into a vein through a needle or thin tube; often written as IV.

lactation: The making and release of milk from a mother's breasts after giving birth.

malaise: A general feeling of being unwell, uneasy, or low on energy, without one clear symptom.

Mechanism of Action: How a medicine produces its effect in the body, described at the level of cells or body chemistry.

Metabolism: How the body chemically changes a medicine, usually in the liver, into other substances.

NDC: A unique number that identifies a specific medicine, its maker, strength, and package size.

open-label: A study in which both the patients and their doctors know which treatment each person is receiving.

parenteral: Given by injection or drip into the body rather than swallowed by mouth.

Patient Counseling Information: The label section telling health professionals what to explain to patients about using a medicine safely.

Pharmacokinetics: The study of how the body takes in a medicine, spreads it around, breaks it down, and removes it over time.

placebo: An inactive treatment with no real medicine in it, such as a sugar pill, used for comparison in a study.

placebo-controlled: Describes a study that compares people taking the real medicine against people taking a dummy pill with no active ingredient.

plasma concentration: The amount of medicine measured in the liquid part of the blood at a given time.

protein binding: How much of a medicine sticks to proteins in the blood instead of moving freely through the body.

pyrexia: A raised body temperature; a fever.

randomized: Assigned to a treatment group purely by chance, like a coin flip, so the groups being compared start out fair and similar.

renal impairment: Reduced kidney function, meaning the kidneys do not filter waste and fluid from the blood as well as normal.

single-dose vial: A small container holding one dose of medicine, meant to be used once and then thrown away.

tachycardia: A faster-than-normal heart rate, usually over 100 beats per minute.

topical: Applied onto the skin, eyes, or another body surface to work mainly where it is placed.

urticaria: Raised, itchy welts on the skin, commonly called hives.

Use in Specific Populations: The label section describing how a medicine affects particular groups, such as pregnant people, children, or older adults.

volume of distribution: A calculated value showing how widely a medicine spreads into body tissues versus staying in the blood.

Warnings and Precautions: The label section describing serious risks to know about and safety steps to follow while using a medicine.

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

🔗 Related NDC lookups

🧪 All Eteplirsen NDCsSame ingredient 🏭 All Sarepta labeler NDCsLabeler code 60923 📚 All Antisense Oligonucleotide NDCsDrug class

💊 Related drugs in this class

Other drugs that share this product’s specific therapeutic class — Genetic D/O Tx-Exon Skipping Antisense Oligonucleo.

Casimersen 60923-0227-02 Golodirsen 60923-0465-02 Viltolarsen 73292-0011-01

Where does this data come from?

Other drugs sharing this product’s First Databank therapeutic class (HIC3). Each links to that NDC’s page in our directory.