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ELEVIDYS delandistrogene moxeparvovec-rokl Kit — NDC 60923-0536-45 package photo

ELEVIDYS delandistrogene moxeparvovec-rokl Kit

by Sarepta Therapeutics, Inc. · 1 KIT in 1 CARTON (60923-536-45) * 10 mL in 1 VIAL, SINGLE-USE (60923-562-01) * 45 POUCH in 1 BOX / 1 SWAB in 1 POUCH
NDC 60923-0536-45
🏷️ FDA NDC (as labeled) 60923-536-45 billing pads the product segment with a zero
Rx only Brand On market Non-controlled
🗂️ Data synced Sep 17, 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) 60923-536-45
Product NDC 60923-536
11-digit billing NDC 60923053645
NCPDP billing unit EA — each (per item)
RxCUI 797544, 2641679, 2641685, 2641687, 2641688, 2641689, 2641690, 2641691,
UPC 0360923501108, 0360923528372, 0360923540497, 0360923521304 +10 more
Application # BLA125781
SPL Set ID 3525abcd-fbd2-46c5-9706-cffdf2e8a361
DEA schedule Non-controlled
Marketing category BLA
Marketing status On market
FDA listing status Listed (active directory)
Marketing start 2023-06-22
Route INTRAVENOUS
Dosage form KIT
GCN Seq No 084931
GCN 54344
HICL code 049019
Ingredient (HICL) Delandistrogene Moxeparvc-Rokl
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 Z16
Therapeutic class — specific (HIC3) Gene Therapy Agents - Protein Deficiency
AHFS code 26:12.00.00
AHFS class Gene Therapy
FDB label name ELEVIDYS 44.5-45.4 KG(10MLX45)
FDB brand name Elevidys
Legend status F — Federal legend — prescription drug or device
Biologic (Purple Book) 351(a)
Why two NDCs? The FDA registers this code as 60923-536-45 — 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 → 60923-0536-45. 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 Other antiseptics and disinfectants class.

Drug family (ATC) Other antiseptics and disinfectants
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.
FDA applicationBLA125781 (BLA)
Labeler code60923
First marketedJun 2023
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 ELEVIDYS 44.5-45.4 KG(10MLX45) Ingredient Delandistrogene Moxeparvc-Rokl
📖 What it is MedlinePlus · NLM

Delandistrogene moxeparvovec-rokl injection is used to treat certain patients with Duchenne muscular dystrophy (DMD; a progressive disease in which the muscles do not function properly). It is in a class of medications called gene therapy. It works by replacing a defective gene in muscles which helps improve muscle function.

Read the full MedlinePlus article ↗

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.

A current SPL was checked, but it does not contain a structured or narrative inactive-ingredient list for this product. This does not mean the product has no inactive ingredients.
Where does this data come from?
Source: official FDA Structured Product Labeling (SPL) via DailyMed and the openFDA label index. Structured IACT rows and label-wide narrative are kept separate; availability and product-level specificity depend on the submitted label.

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 No recent Medicaid claims on file for this NDC — rare and low-volume NDCs are suppressed in the public data.
Medicare drug plans payPart D · quarterly No Part D plan price is available for this NDC in our data.
Medicare Part B allowsASP · J1413 No ASP payment limit on file for J1413 this quarter.
ℹ️
No price is published for this exact package yet. CMS surveys NADAC per package size, so a different pack of the same drug often has one.
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.

🧾 Billing & reimbursement

FDA NDC (as labeled)60923-536-45
11-digit billing NDC60923-0536-45
Format5-3-2 as registered → padded to 5-4-2 for billing (zero added to the product segment)
HCPCS J-codeJ1413
DescriptorINJECTION, DELANDISTROGENE MOXEPARVOVEC-ROKL, PER THERAPEUTIC DOSE
Billing units / pkg1 units
Crosswalk sourcePDAC NDC-HCPCS crosswalk (DME MAC / DMEPOS)
Where does this data come from?
The HCPCS J-code crosswalk comes from the CMS ASP NDC-HCPCS crosswalk and the DMEPDAC (DME MAC) NDC-HCPCS crosswalk — free public CMS data. Billing units are derived from the code’s descriptor and the package amount.

🔁 Therapeutic equivalents

ProductLabelerPackNADAC/unitTEStatusPrice vs. this
Elevidys 60923-0518-27 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0547-56 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0552-61 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0561-70 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0523-32 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0529-38 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0557-66 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0505-14 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0507-16 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0522-31 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0526-35 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0533-42 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0543-52 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0544-53 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0503-12 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0508-17 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0513-22 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0516-25 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0521-30 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0525-34 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0538-47 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0555-64 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0558-67 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0560-69 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0532-41 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0542-51 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0545-54 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0548-57 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0551-60 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0554-63 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0510-19 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0524-33 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0534-43 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0550-59 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0559-68 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0502-11 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0509-18 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0512-21 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0514-23 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0530-39 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0537-46 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0556-65 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0504-13 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0506-15 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0515-24 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0520-29 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0527-36 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0528-37 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0546-55 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0553-62 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0501-10 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0511-20 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0531-40 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0535-44 Sarepta 1 kit — — FDA listed —
Elevidysthis 60923-0536-45 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0540-49 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0541-50 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0517-26 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0519-28 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0539-48 Sarepta 1 kit — — FDA listed —
Elevidys 60923-0549-58 Sarepta 1 kit — — FDA listed —
About this product: this is a biologic. Biologics don't have small-molecule generics — competition comes from FDA-licensed biosimilars (shown above), not generics.
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 & biosimilar status

🏛️
2023
First FDA approval
Jun 2023
📍
2026
Currently FDA-listed
3 years listed
🛡️
2035
Latest patent/protection listed
not a guaranteed launch date
🧬Biologic — competition comes from biosimilars

Biologics have no small-molecule generics; biosimilar competition is tracked in the FDA Purple Book.

🛡️ Latest patent/protection date listed: FDA patent/protection data lists protections through Jun 2035. This may affect when biosimilars become widely available, but it is not a guaranteed launch date.
📅 FDA approved Jun 22, 2023 ⏳ ~8.8 yr to latest listed protection

Why the date isn’t exact: Biosimilar timing can change because patents may be challenged, settled, licensed, added or removed, and litigation can move the real date earlier or later.

FDA Purple Book — biosimilars & interchangeables ⓘ
Reference product
Elevidysdelandistrogene moxeparvovec-rokl
🔒 No FDA-licensed biosimilars or interchangeable biosimilars are listed yet for this biologic. It currently has no biosimilar competition in the FDA Purple Book.
Source: FDA Purple Book (purplebooksearch.fda.gov), matched on the reference product’s active ingredient.
Patents & exclusivity — FDA Purple Book
Exclusivity RefProduct
2023 2025 2027 2029 2031 2033 2035
Today
LOE
Biologic patent Exclusivity
🏛️Reference-product exclusivity
A flat 12 years of FDA market protection from first licensure. No biosimilar can be licensed before it ends — regardless of patents.
🧪Listed biologic patents
Patents the reference maker lists covering the molecule, formulation, or manufacturing. A biosimilar generally can’t launch until these resolve.
🔁Interchangeability
An interchangeable biosimilar may be substituted at the pharmacy (state laws vary). The first one can earn its own exclusivity period.
🛈 What do these terms mean?
Biologic patent
A patent the reference product’s maker has publicly listed. A biosimilar generally can’t launch until these expire — unless they’re invalidated or resolved in a settlement.
Reference-product exclusivity
A flat 12 years of FDA market protection from the biologic’s first licensure (the BPCIA). No biosimilar can be licensed before it ends, regardless of patents.
Interchangeable exclusivity
The first interchangeable biosimilar can earn a period as the only interchangeable version (pharmacists can substitute it without the prescriber).
Earliest biosimilar (LOE)
The latest of all the dates above — the soonest a biosimilar can realistically reach the market. Litigation and settlements can move it earlier.

Biologics have no small-molecule “generics” — competition comes from FDA-licensed biosimilars, tracked in the FDA Purple Book.

FDA exclusivity
CodeWhat it grantsExpires
RefProductReference-product exclusivity (12-year, BPCIA) — no biosimilar can be licensed before this dateJun 22, 2035
Common questions
Is there a biosimilar for ELEVIDYS 44.5-45.4 KG(10MLX45)?
No FDA-licensed biosimilar is currently listed for this biologic in the FDA Purple Book.
Why do different websites show different biosimilar dates?
Biosimilar availability isn’t based on one single date. Some sources use the reference-product exclusivity, some use the last listed patent, and patent litigation, settlements, and licenses can all change the real-world launch date. This page shows the underlying Purple Book dates so you can see why estimates differ.
Can a biosimilar launch before the last patent expires?
Sometimes. A biosimilar maker may settle with the reference manufacturer or receive a license to launch earlier. In other cases, the last listed protection delays competition.
What does “current Purple Book estimate” mean?
It means we’re using the latest patent and exclusivity dates currently listed in the FDA Purple Book. It is not a guaranteed launch date.
What does “FDA listed” mean?
It means the product appears in the FDA’s official directory. That’s a good sign a product exists for the U.S. market, but on its own it does not confirm a pharmacy can get it today. Where we have recent retail pricing data, 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 Purple Book for a patent or exclusivity on the reference biologic. It can affect when a biosimilar becomes widely available — but it is not a guaranteed launch date. Settlements and licenses can move the real date earlier or later.
Built from the FDA Purple Book Patent List (patents the reference-product sponsor has publicly listed under the BPCIA) plus reference-product exclusivity. Biosimilars cannot launch until these clear; patent litigation and settlements can shift the real date. Biologics have no small-molecule generics — competition comes from FDA-licensed biosimilars, not the Orange Book.
Where does this data come from?
Patents and exclusivity from the FDA Purple Book (biologics), refreshed from public FDA data. Biosimilar launch timing is an estimate, not a guarantee.

🔬 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 ELEVIDYS (this brand).

Top reported reactions

Vomiting63
Gamma-glutamyltransferase Increased43
Nausea43
Alanine Aminotransferase Increased35
Aspartate Aminotransferase Increased32
Hepatic Enzyme Increased29
Liver Injury29

Reporter sex

0 reports

Serious outcomes

Hospitalization160
Death7
Life-threatening2
Disabling1
Reports over time (by year) — tap or hover for the count & year
2023 2024 2025 2026 165 0
Most recent year is provisional (FAERS lags ~3 months).
Where does this data come from?
Adverse-event reports from the FDA Adverse Event Reporting System (FAERS) via openFDA. FAERS reports are voluntary and unverified — counts are not incidence and don’t establish causation.

📦 Packaging — all sizes for this product

Package NDCDescription Marketing startStatus
60923-0536-45 You're viewing this 1 KIT in 1 CARTON (60923-536-45) * 10 mL in 1 VIAL, SINGLE-USE (60923-562-01) * 45 POUCH in 1 BOX / 1 SWAB in 1 POUCH 2023-06-22 Active

🧭 About this NDC listing & data coverage

Finished prescription product Kit / multi-component package

Kit / multi-component package

This NDC identifies a kit — a package containing more than one component. Structured data (pricing, ingredients, equivalents) is often reported per component rather than for the kit NDC itself, which can make this page look thinner than the components' own pages.

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) — Not published for this NDC The labeler did not submit a structured excipient list, or no SPL is 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 ✓ Available
Medicaid utilization (CMS SDUD) — Not published for this NDC CMS reports utilization only for NDCs with Medicaid claims above its privacy threshold.
“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 60923-536-45, 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: 60923-0536-45, written without dashes as 60923053645. 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 60923-0536-45, the first segment (60923) is the labeler code FDA assigned to Sarepta Therapeutics, Inc.; the middle segment (0536) identifies this specific product — its ingredient, strength, and dosage form; and the last segment (45) 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.
Does this product have a billing J-code?
Yes — this NDC cross-references HCPCS code J1413 for medical-claim billing (typically used when a product is administered in a clinical setting rather than dispensed at a retail pharmacy). See the Billing section on this page.
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.
🚨 Boxed Warning 🎯 Indications and Usage ⏱️ Dosage and Administration 💊 Dosage Forms and Strengths ⛔ Contraindications ⚠️ Warnings and Cautions 🤒 Adverse Reactions 🔄 Drug Interactions 👥 Use in Specific Populations 🤰 Pregnancy 🧒 Pediatric Use 🧓 Geriatric Use 🧬 Clinical Pharmacology 🧬 Mechanism of Action 📦 How Supplied / Storage and Handling 📦 Storage and Handling 📋 Description 💬 Information for Patients 💬 Medication Guide
🚨 Boxed Warning ~1 min read ▾

WARNING: ACUTE SERIOUS LIVER INJURY AND ACUTE LIVER FAILURE Acute serious liver injury, including life-threatening and fatal acute liver failure, has occurred with ELEVIDYS [see Warnings and Precautions ( 5.1 )]. Patients with preexisting liver impairment may be at higher risk [see Warnings and Precautions ( 5.1 )]. Prior to infusion, assess liver function by clinical examination and laboratory testing.

Administer systemic corticosteroids before and after ELEVIDYS infusion. Continue to monitor liver function weekly for the first 3 months after infusion and continue until results are unremarkable [see Dosage and Administration (2.1, 2.2, 2.4)]. Instruct patients to maintain proximity to an appropriate healthcare facility, as determined by the healthcare provider, for at least 2 months following ELEVIDYS infusion [see Dosage and Administration ( 2.1 )].

Obtain prompt consultation with a specialist (e.g., gastroenterologist or hepatologist) if acute serious liver injury or impending acute liver failure is suspected [see Dosage and Administration ( 2.2 ), Warnings and Precautions ( 5.1 )]. WARNING: ACUTE SERIOUS LIVER INJURY AND ACUTE LIVER FAILURE See full prescribing information for complete boxed warning. Acute serious liver injury, including life-threatening and fatal acute liver failure, has occurred with ELEVIDYS.

( 5.1 ) Patients with preexisting liver impairment may be at higher risk. ( 5.1 ) Prior to infusion, assess liver function by clinical examination and laboratory testing. Administer systemic corticosteroids before and after ELEVIDYS infusion.

Continue to monitor liver function weekly for the first 3 months after infusion and continue until results are unremarkable. ( 2.1 , 2.2 , 2.4 ) Instruct patients to maintain proximity to an appropriate healthcare facility, as determined by the healthcare provider, for at least 2 months following ELEVIDYS infusion. ( 2.1 ) Obtain prompt consultation with a specialist (e.g., gastroenterologist or hepatologist) if acute serious liver injury or impending acute liver failure is suspected.

( 2.2 , 5.1 )

🎯 Indications and Usage ~1 min read ▾

1 INDICATIONS AND USAGE ELEVIDYS is indicated for the treatment of patients 4 years of age and older with Duchenne muscular dystrophy (DMD), who are ambulatory and have a confirmed mutation in the DMD gene [see Clinical Pharmacology ( 12.2 ), Clinical Studies ( 14 )] . ELEVIDYS is an adeno-associated virus vector-based gene therapy indicated for the treatment of patients 4 years of age and older with Duchenne muscular dystrophy (DMD) who are ambulatory and have a confirmed mutation in the DMD gene. ( 1 , 12.2 , 14 ) Limitations of Use: ELEVIDYS is not recommended in patients with: Preexisting liver impairment (defined as gamma-glutamyl transferase [GGT] > 2 x upper limit of normal or total bilirubin > the upper limit of normal not due to Gilbert's syndrome) or active hepatic viral infection due to the high risk of acute serious liver injury and acute liver failure.

Recent vaccination (within 4 weeks of treatment) due to immunogenicity and potential safety concerns. Active or recent (within 4 weeks) infections due to safety concerns. Limitations of Use: ELEVIDYS is not recommended in patients with: Preexisting liver impairment (defined as gamma-glutamyl transferase [GGT] > 2 x upper limit of normal or total bilirubin > the upper limit of normal not due to Gilbert's syndrome) or active hepatic viral infection due to the high risk of acute serious liver injury and acute liver failure.

Recent vaccination (within 4 weeks of treatment) due to immunogenicity and potential safety concerns. Active or recent (within 4 weeks) infections due to safety concerns.

⏱️ Dosage and Administration ~3 min read ▾

2 DOSAGE AND ADMINISTRATION For single-dose intravenous infusion only. ELEVIDYS is for single-dose intravenous infusion only. Select patients for treatment with ELEVIDYS with anti-AAVrh74 total binding antibody titers <1:400, as measured by an FDA authorized test.

( 2.1 ) Postpone in patients with active or recent (within 4 weeks) infections. ( 2.1 ) Assess liver function, platelet counts and troponin-I before ELEVIDYS infusion. ( 2.1 ) Recommended dosage: 10 to 70 kg: 1.33 × 10 14 vector genomes (vg) per kg of body weight; 70 kg or greater: 9.31 × 10 15 vg.

( 2.2 ) One day prior to infusion, initiate a corticosteroid regimen for a minimum of 60 days. Recommend modifying corticosteroid dose for patients with liver function abnormalities. ( 2.2 ) Administer as an intravenous infusion over 1-2 hours.

Infuse at a rate of less than 10 mL/kg/hour. ( 2.4 )

2.1Critical Dosing Information Instruct patients to maintain proximity to an appropriate healthcare facility, as determined by the healthcare provider, for at least 2 months following ELEVIDYS infusion. Prior to ELEVIDYS infusion: Select patients for treatment with ELEVIDYS with anti-AAVrh74 total binding antibody titers <1:400, as measured by an FDA authorized test. Information on FDA authorized tests is available at http://www.fda.gov/companiondiagnostics.

Do not administer ELEVIDYS to patients with elevated anti-AAVrh74 total binding antibody titers (≥1:400) [Clinical Pharmacology ( 12.6 )] . Due to the increased risk of serious systemic immune response, postpone ELEVIDYS in patients with active or recent (within 4 weeks) infections [see Warnings and Precautions ( 5.2 )]. Assess liver function (clinical examination and laboratory testing including aspartate aminotransferase (AST), alanine aminotransferase (ALT), gamma-glutamyl transferase (GGT), albumin, activated partial thromboplastin time (aPTT), international normalized ratio (INR), and total bilirubin) [see Dosage and Administration ( 2.4 ), Warnings and Precautions ( 5.1 ), Use in Specific Populations ( 8.6 )] .

Obtain platelet count and troponin-I levels [see Dosage and Administration ( 2.4 ), Warnings and Precautions ( 5.3 )] . Do not re-administer ELEVIDYS.

2.2Recommended Dose The recommended dose of ELEVIDYS is 1.33 × 10 14 vector genomes per kilogram (vg/kg) of body weight (or 10 mL/kg body weight) for patients weighing less than 70 kg or 9.31 × 10 15 vg total fixed dose for patients weighing 70 kg or greater. For the number of vials required, refer to Table 10 [see How Supplied/Storage and Handling ( 16.1 )] . Calculate the dose as follows: ELEVIDYS dose (in mL) = patient body weight (rounded to the nearest kilogram) x 10 The multiplication factor 10 represents the per kilogram dose (1.33 × 10 14 vg/kg) divided by the amount of vector genome copies per mL of the ELEVIDYS suspension (1.33 × 10 13 vg/mL).

Number of ELEVIDYS vials needed = ELEVIDYS dose (in mL) divided by 10. Example: Calculation of volume needed for a 19.5 kg patient 19.5 kg rounded to the nearest kilogram = 20 kg 20 kg × 10 = 200 mL Number of ELEVIDYS vials needed = 200 divided by 10, rounded to the nearest number of vials = 20 vials Administer corticosteroids to reduce the risk of immune responses to the AAVrh74 vector after administration of ELEVIDYS [see Clinical Pharmacology ( 12.6 )] . Start corticosteroids 1 day prior to ELEVIDYS infusion based on the schedule outlined in Table 1 below.

Continue this regimen for a minimum of 60 days after the infusion, unless earlier tapering is clinically indicated. Table 1: Recommended pre- and post-infusion oral corticosteroid dosing a Patient continues to receive this dose b Corticosteroids other than prednisone and prednisolone have not been studied for use as a peri-ELEVIDYS infusion corticosteroid Baseline corticosteroid dosing a Peri-ELEVIDYS infusion corticosteroid dose (prednisone equivalent) b Recommended maximum total daily oral dose (prednisone equivalent) b Daily or intermittent dose Start 1 day…

💊 Dosage Forms and Strengths 130 words ▾

3 DOSAGE FORMS AND STRENGTHS ELEVIDYS is a preservative-free, sterile, clear, colorless liquid that may have some opalescence and may contain white to off-white particles. ELEVIDYS is a suspension for intravenous infusion with a nominal concentration of 1.33 × 10 13 vg/mL. ELEVIDYS is provided in a customized kit containing ten to seventy 10 mL single-dose vials, with each kit constituting a dosage unit based on the patient's body weight [see How Supplied/Storage and Handling ( 16.1 )] .

ELEVIDYS is a suspension for intravenous infusion with a nominal concentration of 1.33 × 10 13 vg/mL. ( 3 ) ELEVIDYS is provided in a customized kit containing ten to seventy 10 mL single-dose vials, with each kit constituting a dosage unit based on the patient's body weight. ( 3 )

⛔ Contraindications 73 words ▾

4 CONTRAINDICATIONS ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9, including a deletion of any portion or the entirety of these exons, in the DMD gene [see Warnings and Precautions ( 5.5 )] . ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9, including a deletion of any portion or the entirety of these exons, in the DMD gene. ( 4 )

⚠️ Warnings and Cautions ~3 min read ▾

5 WARNINGS AND PRECAUTIONS Serious Infections: Serious infections with fatal outcomes may occur due to concomitant administration of corticosteroids, additional immunosuppressants, and ELEVIDYS. Monitor patients for signs and symptoms of infection; treat appropriately. ( 5.2 ) Myocarditis: Acute, serious, life-threatening myocarditis and troponin-I elevations have been observed.

Monitor troponin-I before ELEVIDYS infusion, and weekly for the first month after ELEVIDYS infusion. ( 5.3 ) Infusion-related Reactions: Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred. Monitor during administration and for at least 3 hours after end of infusion.

If symptoms occur, slow or stop the infusion and give appropriate treatment. Once symptoms resolve, restart infusion at a slower infusion rate. Discontinue infusion for anaphylaxis.

( 2.4 , 5.4 ) Immune-mediated Myositis: Severe to life-threatening immune-mediated myositis has been reported with ELEVIDYS in patients with deletions including portions of exons 1 to 17 and /or exons 59 to 71 of the DMD gene. Consider additional immunomodulatory treatment if symptoms of myositis occur (e.g., unexplained increased muscle pain, tenderness, or weakness). ( 5.5 ) Pre-existing Immunity against AAVrh74: Perform baseline testing for presence of anti-AAVrh74 total binding antibodies prior to ELEVIDYS administration.

( 5.6 )

5.1Acute Serious Liver Injury and Acute Liver Failure Acute serious liver injury marked by elevations of liver enzymes (e.g., GGT, ALT) and total bilirubin and acute liver failure, has occurred with ELEVIDYS. Onset of the liver injury typically begins within 8 weeks after administration of ELEVIDYS. In non-ambulatory patients treated with ELEVIDYS, acute liver failure with fatal outcome has occurred in the clinical and post-marketing settings.

Life-threatening mesenteric vein thrombosis, complicated by bowel ischemia and necrosis, and portal hypertension have been reported following acute liver injury associated with ELEVIDYS in a non-ambulatory patient [see Adverse Reactions ( 6.2 )] . Patients with preexisting liver impairment, chronic hepatic condition, or acute liver disease (e.g., acute hepatic viral infection) may be at higher risk of acute serious liver injury or acute liver failure. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled.

Patients with hepatic impairment, acute liver disease, chronic hepatic condition or elevated GGT have not been studied in clinical trials with ELEVIDYS [see Specific Populations ( 8.6 )] . In clinical studies, liver function test increased (including increases in GGT, ALT, AST, or total bilirubin) was commonly reported typically within 8 weeks following ELEVIDYS infusion, with the majority of cases being asymptomatic [see Adverse Reactions ( 6.1 )] . Most cases resolved spontaneously or with systemic corticosteroids and resolved without clinical sequelae within 2 months.

Prior to ELEVIDYS administration, perform liver enzyme test [see Dosage and Administration ( 2.1 )] . Monitor liver function (clinical examination, AST, ALT, GGT, albumin, aPTT, INR, and total bilirubin) weekly for the first 3 months following ELEVIDYS infusion. Continue monitoring if clinically indicated, until results are unremarkable (e.g., normal clinical exam, GGT and total bilirubin levels return to near baseline levels) [see Dosage and Administration ( 2.4 )] .

Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion [see Dosage and Administration ( 2.2 )] . Adjust corticosteroid regimen when indicated [see Dosage and Administration ( 2.2 )] . Obtain prompt consultation with a specialist (e.g., gastroenterologist or hepatologist) if acute serious liver injury or impending acute liver failure is suspected.

5.2Serious Infections Increased susceptibility to serious infections may occur due to concomitant administration of corticosteroid regim…

🤒 Adverse Reactions ~3 min read ▾

6 ADVERSE REACTIONS Most common adverse reactions (incidence ≥5%) were vomiting and nausea, liver injury, pyrexia, thrombocytopenia, and troponin-I increased. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Sarepta Therapeutics, Inc., at 1-888-SAREPTA (1-888-727-3782) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

6.1Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The safety data described in this section reflect exposure to a one-time intravenous infusion of ELEVIDYS in 156 male patients with a confirmed mutation of the DMD gene in four clinical studies, including one completed open-label study, one ongoing open-label study, and two studies that included a double-blind, placebo-controlled period.

Prior to ELEVIDYS infusion, patients in the ELEVIDYS treatment group had a mean age of 6.7 years (range: 3 to 20) and mean weight of 24.6 kg (range: 12.5 to 80.1). 144 patients received the recommended dose of 1.33 × 10 14 vg/kg, and 12 received a lower dose. Table 3 below presents adverse reactions from these four clinical studies.

The most common adverse reactions (incidence ≥5%) across all studies are summarized in Table 3 . Adverse reactions were typically seen within the first 2 weeks (nausea, vomiting, thrombocytopenia, pyrexia), the first month (myocarditis, troponin-I increased) or within the first 2 months (immune-mediated myositis, liver injury). Vomiting may occur as early as on the day of the infusion.

Table 3. Adverse reactions (Incidence ≥5%) following treatment with ELEVIDYS in Clinical Studies Adverse reactions ELEVIDYS (N=156) % a Includes: AST increased, ALT increased, GGT increased, GLDH increased, GLDH level abnormal, Hepatotoxicity, Hepatic enzyme increased, Hypertransaminasemia, Liver function test increased, Liver injury, Transaminases increased, Blood bilirubin increased b Includes: Thrombocytopenia, Platelet count decreased c Transient, mild, asymptomatic decrease in platelet counts d Includes: Troponin I increased, Troponin increased, Troponin I abnormal Vomiting 65 Nausea 43 Liver injury a 40 Pyrexia 28 Thrombocytopenia b c 8 Troponin-I increased d 8 In clinical trials, immune-mediated myositis was observed in 2 of 6 patients with deletion mutations involving exon 8 and/or 9 in the DMD gene [see Contraindications ( 4 ), and Warnings and Precautions ( 5.5 )] .

In the double-blind, placebo-controlled trial, Study 3 Part 1, patients 4 to 7 years of age (N=125) received either ELEVIDYS (N=63) at the recommended dose of 1.33 × 10 14 vg/kg or placebo (N=62). Table 4 below presents the most frequent adverse reactions from Study 3 Part 1. Table 4.

Adverse reactions occurring in ELEVIDYS-treated patients and at least twice more frequently than with placebo in Study 3 Part 1 a Includes: AST increased, ALT increased, GGT increased, GLDH increased, GLDH level abnormal, Hepatotoxicity, Hepatic enzyme increased, Hypertransaminasemia, Liver function test increased, Liver injury, Transaminases increased. b Includes: platelet count decreased, thrombocytopenia c Transient, mild, asymptomatic decrease in platelet counts Adverse reactions ELEVIDYS (N=63) % Placebo (N=62) % Vomiting 64 19 Nausea 40 13 Liver injury a 41 8 Pyrexia 32 24 Thrombocytopenia bc 3 0

6.2Postmarketing Experience The following adverse reactions have been identified during post-approval use of ELEVIDYS. 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. Hepatobiliary Disorders: Acute liver injury, acute liver failure, including fatal outcome and life-threatening mesenteric vein thrombosis [see Warnings and Precautions ( 5.1 )] Infections and Infestations : B…

🔄 Drug Interactions 51 words ▾

7 DRUG INTERACTIONS Prior to initiating the corticosteroid regimen required before ELEVIDYS administration, consider the patient's vaccination status. Patients should, if possible, be brought up to date with all immunizations in agreement with current immunization guidelines. Vaccinations should be completed at least 4 weeks prior to initiation of the corticosteroid regimen.

👥 Use in Specific Populations ~1 min read ▾

8 USE IN SPECIFIC POPULATIONS

8.1Pregnancy Risk Summary ELEVIDYS is not intended for use in pregnant women. In the U.S. general population, the estimated background risks of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively.

8.2Lactation Risk Summary There is no information available on the presence of ELEVIDYS in human milk, the effects on the breastfed infant, or the effects on milk production.

8.4Pediatric Use The safety and effectiveness of ELEVIDYS for the treatment of Duchenne muscular dystrophy has been established in pediatric patients at least 4 years of age with a confirmed mutation in the DMD gene. The use of ELEVIDYS in pediatric patients was supported by evidence from three adequate and well controlled clinical studies which included 144 pediatric patients aged 4 years of age and older [see Adverse Reactions ( 6 ), Clinical Pharmacology ( 12.2 ), Clinical Studies ( 14 )] .

8.5Geriatric Use The safety and efficacy of ELEVIDYS in geriatric patients with DMD have not been studied.

8.6Hepatic Impairment The safety and efficacy of ELEVIDYS in patients with hepatic impairment or elevated GGT have not been studied. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled. Treatment with ELEVIDYS should be carefully considered in patients with preexisting liver impairment or chronic hepatic viral infection.

These patients may be at increased risk of acute serious liver injury or acute liver failure [see Warnings and Precautions ( 5.1 )] . In clinical trials, liver function test increase was commonly reported in patients following ELEVIDYS infusion [see Warnings and Precautions ( 5.1 ), Adverse Reactions ( 6.1 )] .

🤰 Pregnancy 41 words ▾

8.1Pregnancy Risk Summary ELEVIDYS is not intended for use in pregnant women. In the U.S. general population, the estimated background risks of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively.

🧒 Pediatric Use 84 words ▾

8.4Pediatric Use The safety and effectiveness of ELEVIDYS for the treatment of Duchenne muscular dystrophy has been established in pediatric patients at least 4 years of age with a confirmed mutation in the DMD gene. The use of ELEVIDYS in pediatric patients was supported by evidence from three adequate and well controlled clinical studies which included 144 pediatric patients aged 4 years of age and older [see Adverse Reactions ( 6 ), Clinical Pharmacology ( 12.2 ), Clinical Studies ( 14 )] .

🧓 Geriatric Use 18 words ▾

8.5Geriatric Use The safety and efficacy of ELEVIDYS in geriatric patients with DMD have not been studied.

🧬 Clinical Pharmacology ~3 min read ▾

12 CLINICAL PHARMACOLOGY

12.1Mechanism of Action ELEVIDYS is the recombinant gene therapy product that is comprised of a non-replicating, recombinant, adeno-associated virus (AAV) serotype rh74 (AAVrh74) capsid and a single-stranded DNA expression cassette flanked by inverted terminal repeats (ITRs) derived from AAV2. The cassette contains: 1) an MHCK7 gene regulatory component comprising a creatine kinase 7 promoter and an α-myosin heavy chain enhancer, and 2) the DNA transgene encoding the engineered micro-dystrophin protein. Vector/Capsid: Clinical and nonclinical studies have demonstrated AAVrh74 serotype transduction in skeletal muscle cells.

Additionally, in nonclinical studies, AAVrh74 serotype transduction has been demonstrated in cardiac and diaphragm muscle cells. Promoter: The MHCK7 promoter/enhancer drives transgene expression and has been shown in animal models to drive transgenic micro-dystrophin protein expression predominantly in skeletal muscle (including diaphragm) and cardiac muscle. In clinical studies, muscle biopsy analyses have confirmed micro-dystrophin expression in skeletal muscle.

Transgene: DMD is caused by a mutation in the DMD gene resulting in lack of functional dystrophin protein. ELEVIDYS carries a transgene encoding a micro-dystrophin protein consisting of selected domains of dystrophin expressed in normal muscle cells. ELEVIDYS micro-dystrophin has been demonstrated to localize to the sarcolemma.

12.2Pharmacodynamics In 92 patients who received ELEVIDYS in clinical studies, micro-dystrophin protein expression from muscle biopsies (gastrocnemius or biceps brachii) was quantified by western blot and localized by immunofluorescence staining (fiber intensity and percentage micro-dystrophin). Micro-dystrophin expression (expressed as change from baseline) in ELEVIDYS-treated patients as measured by western blot was the primary objective of Study 1 and Study 2, and a key secondary objective for Study 3. Muscle biopsies were obtained at baseline prior to ELEVIDYS infusion and at Week 12 after ELEVIDYS infusion in all patients.

The absolute quantity of micro-dystrophin was measured by western blot assay, adjusted by muscle content and expressed as a percent of control (levels of wild-type dystrophin in patients without DMD or Becker muscular dystrophy) in muscle biopsy samples. Study 1 and 2 results of patients receiving 1.33 × 10 14 vg/kg ELEVIDYS are presented in Table 5 . Table 5.

Micro-Dystrophin Expression in Study 1 and Study 2 at Week 12 from Baseline (Western Blot Assay) abc Western blot (% of micro-dystrophin compared to control) Study 1 Part 1 (n=6) Study 1 Part 2 (n=21) Study 2 Ambulatory (n=40) a All patients received 1.33 x 10 14 vg/kg, as measured by ddPCR b Change from baseline was statistically significant c Adjusted for muscle content. Control was level of wild-type (normal) dystrophin in normal muscle. Mean change from baseline (SD) 43.4 (48.6) 40.7 (32.3) 51.0 (47) Median change from baseline (Min, Max) 24.3 (1.6, 116.3) 40.8 (0.0, 92.0) 46.9 (1.9, 197.3) In Study 3 Part 1, muscle biopsies were obtained at Week 12 in 31 patients.

For the ELEVIDYS-treated patients, the mean micro-dystrophin expression at Week 12 was 34.3% (N=17, SD: 41.0%), compared to placebo patients of 0% (N=14, SD: 0%). Assessment of micro-dystrophin levels can be meaningfully influenced by differences in sample processing, analytical technique, reference materials, and quantitation methodologies. Therefore, valid comparisons of micro-dystrophin measurements obtained from different assays cannot be made.

12.3Pharmacokinetics Vector Distribution and Vector Shedding Nonclinical Data Biodistribution of ELEVIDYS was evaluated in tissue samples collected from healthy mice and DMD mdx mice following intravenous administration in toxicology studies. At 12 weeks following ELEVIDYS administration at dose levels of 1.33 ×10 14 to 4.02 ×10 14 vg/kg, vector DNA was detected in all major organs with the highest quantities…

🧬 Mechanism of Action 190 words ▾

12.1Mechanism of Action ELEVIDYS is the recombinant gene therapy product that is comprised of a non-replicating, recombinant, adeno-associated virus (AAV) serotype rh74 (AAVrh74) capsid and a single-stranded DNA expression cassette flanked by inverted terminal repeats (ITRs) derived from AAV2. The cassette contains: 1) an MHCK7 gene regulatory component comprising a creatine kinase 7 promoter and an α-myosin heavy chain enhancer, and 2) the DNA transgene encoding the engineered micro-dystrophin protein. Vector/Capsid: Clinical and nonclinical studies have demonstrated AAVrh74 serotype transduction in skeletal muscle cells.

Additionally, in nonclinical studies, AAVrh74 serotype transduction has been demonstrated in cardiac and diaphragm muscle cells. Promoter: The MHCK7 promoter/enhancer drives transgene expression and has been shown in animal models to drive transgenic micro-dystrophin protein expression predominantly in skeletal muscle (including diaphragm) and cardiac muscle. In clinical studies, muscle biopsy analyses have confirmed micro-dystrophin expression in skeletal muscle.

Transgene: DMD is caused by a mutation in the DMD gene resulting in lack of functional dystrophin protein. ELEVIDYS carries a transgene encoding a micro-dystrophin protein consisting of selected domains of dystrophin expressed in normal muscle cells. ELEVIDYS micro-dystrophin has been demonstrated to localize to the sarcolemma.

📦 How Supplied / Storage and Handling ~3 min read ▾

16 HOW SUPPLIED/STORAGE AND HANDLING

16.1How Supplied ELEVIDYS is shipped frozen (≤ -60ºC [-76ºF]) in 10 mL vials. ELEVIDYS is supplied as a customized kit to meet dosing requirements for each patient [see Dosage and Administration ( 2.2 )] . Each kit contains: Ten (10) to seventy (70) single-dose vials of ELEVIDYS One alcohol wipe per vial Each ELEVIDYS pack may contain a maximum of two different drug product lots.

The total number of vials in each kit corresponds to the dosing requirement for the individual patient, based on the patient's body weight, and is specified on the package [see Dosage and Administration ( 2.2 )] . Each kit includes a specified number of ELEVIDYS vials (with a minimum of 10 vials for a patient with 10.0 – 10.4 kg body weight range, and a maximum of 70 vials for a patient with body weight of 69.5 kg and above). Kit sizes and National Drug Codes (NDC) are provided in Table 10 .

Table 10: ELEVIDYS Multi-vial Kits Patient Weight (kg) Total Vials per Kit Total Dose Volume per Kit (mL) NDC Number 10.0 – 10.4 10 100 60923-501-10 10.5 – 11.4 11 110 60923-502-11 11.5 – 12.4 12 120 60923-503-12 12.5 – 13.4 13 130 60923-504-13 13.5 – 14.4 14 140 60923-505-14 14.5 – 15.4 15 150 60923-506-15 15.5 – 16.4 16 160 60923-507-16 16.5 – 17.4 17 170 60923-508-17 17.5 – 18.4 18 180 60923-509-18 18.5 – 19.4 19 190 60923-510-19 19.5 – 20.4 20 200 60923-511-20 20.5 – 21.4 21 210 60923-512-21 21.5 – 22.4 22 220 60923-513-22 22.5 – 23.4 23 230 60923-514-23 23.5 – 24.4 24 240 60923-515-24 24.5 – 25.4 25 250 60923-516-25 25.5 – 26.4 26 260 60923-517-26 26.5 – 27.4 27 270 60923-518-27 27.5 – 28.4 28 280 60923-519-28 28.5 – 29.4 29 290 60923-520-29 29.5 – 30.4 30 300 60923-521-30 30.5 – 31.4 31 310 60923-522-31 31.5 – 32.4 32 320 60923-523-32 32.5 – 33.4 33 330 60923-524-33 33.5 – 34.4 34 340 60923-525-34 34.5 – 35.4 35 350 60923-526-35 35.5 – 36.4 36 360 60923-527-36 36.5 – 37.4 37 370 60923-528-37 37.5 – 38.4 38 380 60923-529-38 38.5 – 39.4 39 390 60923-530-39 39.5 – 40.4 40 400 60923-531-40 40.5 – 41.4 41 410 60923-532-41 41.5 – 42.4 42 420 60923-533-42 42.5 – 43.4 43 430 60923-534-43 43.5 – 44.4 44 440 60923-535-44 44.5 – 45.4 45 450 60923-536-45 45.5 – 46.4 46 460 60923-537-46 46.5 – 47.4 47 470 60923-538-47 47.5 – 48.4 48 480 60923-539-48 48.5 – 49.4 49 490 60923-540-49 49.5 – 50.4 50 500 60923-541-50 50.5 – 51.4 51 510 60923-542-51 51.5 – 52.4 52 520 60923-543-52 52.5 – 53.4 53 530 60923-544-53 53.5 – 54.4 54 540 60923-545-54 54.5 – 55.4 55 550 60923-546-55 55.5 – 56.4 56 560 60923-547-56 56.5 – 57.4 57 570 60923-548-57 57.5 – 58.4 58 580 60923-549-58 58.5 – 59.4 59 590 60923-550-59 59.5 – 60.4 60 600 60923-551-60 60.5 – 61.4 61 610 60923-552-61 61.5 – 62.4 62 620 60923-553-62 62.5 – 63.4 63 630 60923-554-63 63.5 – 64.4 64 640 60923-555-64 64.5 – 65.4 65 650 60923-556-65 65.5 – 66.4 66 660 60923-557-66 66.5 – 67.4 67 670 60923-558-67 67.5 – 68.4 68 680 60923-559-68 68.5 – 69.4 69 690 60923-560-69 69.5 and above 70 700 60923-561-70 A 10 mL single-dose vial carton for ELEVIDYS (NDC 60923-562-01) is not sold individually.

16.2Storage and Handling ELEVIDYS is shipped and delivered at ≤ −60ºC [−76ºF]. ELEVIDYS can be refrigerated for up to 14 days when stored at 2°C to 8°C (36º F to 46º F) in the upright position. Do not refreeze. Do not shake. Do not place back in the refrigerator once brought to room temperature. Follow local guidelines on handling of biological waste.

16.1How Supplied ELEVIDYS is shipped frozen (≤ -60ºC [-76ºF]) in 10 mL vials. ELEVIDYS is supplied as a customized kit to meet dosing requirements for each patient [see Dosage and Administration ( 2.2 )] . Each kit contains: Ten (10) to seventy (70) single-dose vials of ELEVIDYS One alcohol wipe per vial Each ELEVIDYS pack may contain a maximum of two different drug product lots.

The total number of vials in each kit corresponds to the dosing requirement for the individual patient, based on the patient's body weight, and is specified on the package [see Dos…

📦 Storage and Handling 63 words ▾

16.2Storage and Handling ELEVIDYS is shipped and delivered at ≤ −60ºC [−76ºF]. ELEVIDYS can be refrigerated for up to 14 days when stored at 2°C to 8°C (36º F to 46º F) in the upright position. Do not refreeze. Do not shake. Do not place back in the refrigerator once brought to room temperature. Follow local guidelines on handling of biological waste.

📋 Description 182 words ▾

11 DESCRIPTION ELEVIDYS (delandistrogene moxeparvovec-rokl) is a recombinant gene therapy designed to deliver the gene encoding the ELEVIDYS micro-dystrophin protein. ELEVIDYS is a non-replicating, recombinant, adeno-associated virus serotype rh74 (AAVrh74) based vector containing the ELEVIDYS micro-dystrophin transgene under the control of the MHCK7 promoter. The genome within the ELEVIDYS AAVrh74 vector contains no viral genes and consequently is incapable of replication or reversion to a replicating form.

The micro-dystrophin protein expressed by ELEVIDYS is a shortened version (138 kDa, compared to 427 kDa size of dystrophin expressed in normal muscle cells) that contains selected domains of dystrophin expressed in normal muscle cells. ELEVIDYS is a preservative-free, sterile, clear, colorless liquid that may have some opalescence and may contain white to off-white particles. ELEVIDYS is a suspension for intravenous infusion with a nominal concentration of 1.33 ×10 13 vg/mL and supplied in a single-dose 10 mL vial.

Each vial contains an extractable volume of 10 mL and the following excipients: 200mM sodium chloride, 13 mM tromethamine HCl, 7 mM tromethamine, 1mM magnesium chloride, 0.001% poloxamer 188, with a pH of 8.0 ± 0.3.

💬 Information for Patients ~2 min read ▾

17 PATIENT COUNSELING INFORMATION Advise the patient to read the FDA-approved Medication Guide. Provide a copy of the Medication Guide and review the contents with the patient. Inform patients or caregivers that: ELEVIDYS can increase certain liver enzyme levels and cause acute serious liver injury or acute liver failure, and death.

Management of acute serious liver injury may require hospitalization. Patients will receive oral corticosteroid medication before and after infusion with ELEVIDYS. Weekly blood tests will be required to monitor liver enzyme levels for 3 months after treatment.

Contact a healthcare provider immediately if the patient's skin and/or whites of the eyes appear yellowish, if the patient misses a dose of corticosteroid or vomits it up, or if the patient experiences a change in mental status [see Warnings and Precautions ( 5.1 )] . Due to the concomitant administration of corticosteroids, an infection (e.g., cold, flu, gastroenteritis, otitis media, bronchiolitis, pneumonia, etc.) before or after ELEVIDYS infusion could lead to more serious complications, including death. Contact a healthcare provider immediately if symptoms suggestive of infection are observed (e.g., coughing, wheezing, sneezing, runny nose, sore throat, or fever) [see Warnings and Precautions ( 5.2 )] .

Myocarditis (inflammation of the heart) has been observed within days to more than a year following ELEVIDYS infusion. Weekly monitoring of troponin-I for the first month after treatment is required. Contact a healthcare provider immediately if the patient begins to experience chest pain and/or shortness of breath [see Warnings and Precautions ( 5.3 )] .

Infusion-related reactions including hypersensitivity and anaphylaxis have occurred during and after ELEVIDYS infusion. Possible symptoms of infusion-related reactions are fast heart rate, fast breathing, swollen lips, being short of breath, nostrils widening, hives, red and blotchy skin, itchy or inflamed lips, rash, vomiting, nausea, chills and fever. Contact a healthcare provider immediately if the patient experiences such a reaction [see Warnings and Precautions ( 5.4 )].

Immune-mediated myositis (an immune response affecting muscles) was observed in some patients following ELEVIDYS infusion. Contact a physician immediately if the patient experiences any unexplained increased muscle pain, tenderness, or weakness, including difficulty swallowing, difficulty breathing or difficulty speaking, as these may be symptoms of myositis [see Warnings and Precautions ( 5.5 )] . Patient's immunizations should be up to date with current immunization guidelines prior to initiation of the corticosteroid regimen required before ELEVIDYS infusion.

Vaccinations should be completed at least 4 weeks prior to initiation of the corticosteroid regimen [see Drug Interactions ( 7 )] . Vector shedding of ELEVIDYS occurs primarily through body waste. Practice proper hand hygiene, such as hand washing, when coming into direct contact with patient body waste.

Place potentially contaminated materials that may have the patient's bodily fluids/waste in a sealable bag and dispose into regular trash. These precautions should be followed for one month after ELEVIDYS infusion. Manufactured for: Sarepta Therapeutics, Inc.

Cambridge, MA 02142 USA U.S. license number 2308 SAREPTA, SAREPTA THERAPEUTICS and ELEVIDYS are trademarks of Sarepta Therapeutics, Inc. ©2026 Sarepta Therapeutics, Inc. Elevidys delandistrogene moxeparvovec-rokl suspension for intracenous infusion

💬 Medication Guide ~3 min read ▾

This Medication Guide has been approved by the U.S. Food and Drug Administration. Revised: 11/2025 MEDICATION GUIDE ELEVIDYS (Ah-LEV-ah-dis) (delandistrogene moxeparvovec-rokl) suspension, for intravenous use What is the most important information that you/your child should know about ELEVIDYS?

Rapid serious liver injury and rapid liver failure. ELEVIDYS can increase certain liver lab test levels and cause rapid serious liver injury, rapid liver failure, and death. Complication of blood clots in the blood vessel in the abdomen that helps carry blood from the intestines to the liver has happened.

Patients with preexisting liver problems may be at higher risk. You/your child will receive oral corticosteroid medication before and after ELEVIDYS infusion and will need weekly blood tests to monitor liver function for 3 months or longer after treatment. For at least 2 months following ELEVIDYS infusion, stay close to a healthcare facility that your/your child's doctor recommends.

Contact your/your child's doctor immediately if your/your child's skin and/or whites of the eyes appear yellowish or if your/your child misses a dose of corticosteroid or vomits it up. Serious Infection. Because you/your child will be taking corticosteroids as part of ELEVIDYS treatment, this may lower the ability of your/your child's immune system to fight infections and make it easier to get an infection.

Getting an infection (like a cold, flu, stomach flu, ear infection, chest infection) before or after ELEVIDYS infusion could lead to more serious health problems, including death. Contact your/your child's doctor right away if you notice any signs of infection such as: coughing wheezing sneezing runny nose sore throat fever Vaccinations should be completed at least 4 weeks before starting the corticosteroids that are part of the ELEVIDYS treatment. ELEVIDYS should not be given if you have/your child has an infection.

Inflammation of the heart muscle (Myocarditis). Serious and life-threatening inflammation of the heart muscle has happened following ELEVIDYS infusion. In individuals who develop inflammation of the heart muscle after receiving ELEVIDYS, they may be at a higher risk of serious complications if they already had a heart condition that has affected how well the heart pumps.

You/Your child will need weekly blood tests for a heart protein that can detect damage to muscle cells in the heart (troponin-I) for the first month after ELEVIDYS infusion. Contact your/your child's doctor right away if you notice: chest pain trouble breathing or shortness of breath Infusion-related reactions. Reactions from the infusion, including serious allergic reactions, may happen during or after ELEVIDYS infusion.

Contact your/your child's doctor right away if you notice: fast heart rate fast breathing swollen lips shortness of breath nostrils widening hives red and blotchy skin itchy or inflamed lips rash vomiting nausea chills fever Immune response affecting muscles (Immune-mediated myositis). Immune response affecting muscles, including serious and life-threatening reactions, has happened in patients about 1 month after receiving ELEVIDYS infusion. Contact your/your child's doctor right away if you notice: Unexplained increased muscle pain Tenderness Weakness, including trouble swallowing, breathing or speaking See “ What are the possible side effects of ELEVIDYS? ” for more information about side effects.

What is ELEVIDYS? ELEVIDYS is a prescription gene therapy used to treat ambulatory individuals at least 4 years old with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene. ELEVIDYS is not recommended for individuals with: Preexisting liver problems or liver infection because of the high risk of rapid serious liver injury and rapid liver failure Recent vaccination (within 4 weeks of ELEVIDYS treatment) Current or recent infections (within 4 weeks of ELEVIDYS treatment) Who should not take ELEVIDYS?

You/Your child should not receive E…

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