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Zolgensma onasemnogene abeparvovec-xioi Kit — NDC 71894-0120-02 package photo
Label image from the product's FDA listing (DailyMed) — may show a different pack size or an older label revision.

Zolgensma onasemnogene abeparvovec-xioi Kit — NDC 71894-120-02 (Billing 71894-0120-02)

by Novartis Innovative Technologies Inc. · 1 KIT in 1 CARTON * 8.3 mL in 1 VIAL * 1 mL in 1 POUCH

This is a package of Zolgensma onasemnogene abeparvovec-xioi Kit from Novartis Innovative Technologies Inc., marketed since May 2019 and currently FDA-listed. It is this product's only package size.

NDC 71894-0120-02
🏷️ FDA NDC (as labeled) 71894-120-02 billing pads the product segment with a zero
Rx only Brand On market Non-controlled ⇄ Compare with another NDC
🗂️ FDA directory synced Oct 1, 2026 · this listing last changed Oct 1, 2026 · sources: openFDA · FDA label (DailyMed) · FDA Orange & Purple Book · First Databank · CMS NADAC, ASP, Medicare & Medicaid · RxNorm
📋 All sources & update times →

This page is curated, coded, and maintained by

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

NDC database record

One package, one record: these facts belong to NDC 71894-120-02 alone.

Record
FDA NDC Directory package listing · Human prescription drug
Code segments
71894 labeler · 120 product · 02 package
Package marketed since
May 24, 2019
Sample package
No — commercial package
Listing certified through
Dec 31, 2027
Barcode (UPC-A, from the NDC)
3 7189412002 1
FDA record last changed
Oct 1, 2026

Identity & classification

Regulatory identifiers FDA, NLM and CMS codes for this package

FDA NDC (as labeled) 71894-120-02
Product NDC 71894-120
11-digit billing NDC 71894012002
NCPDP billing unit EA — each (per item)
RxCUI 797544, 2170239, 2170241, 2170244, 2170245, 2170246, 2170247, 2170248,
Application # BLA125694
SPL Set ID 68cd4f06-70e1-40d8-bedb-609ec0afa471
DEA schedule Non-controlled
Marketing category BLA
Marketing status On market
FDA listing status Listed (active directory)
Marketing start 2019-05-24
Dosage form KIT
Biologic (Purple Book) 351(a)

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

GPI-14 74704050106410
GCN Seq No 079767
GCN 46324
HICL code 045760
Ingredient (HICL) Onasemnogene Abeparvovec-Xioi
HIC1 code Z
Therapeutic class — broad (HIC1) Body As A Whole
HIC2 code Z0
Therapeutic class — intermediate (HIC2) Biologicals (Continued 1)
HIC3 code Z0K
Therapeutic class — specific (HIC3) Gene Therapy Agents - Smn Protein Deficiency
AHFS code 26:12.00.00
AHFS class Gene Therapy
FDB label name ZOLGENSMA 2.6-3.0 KG KIT
FDB brand name Zolgensma
Legend status F — Federal legend — prescription drug or device
Quick answers
  • GSN (GCN sequence number): 079767
  • GCN: 46324
  • GPI-14 (Medi-Span): 74704050106410
  • HICL (First Databank): 045760
  • AHFS class code: 26:12.00.00
  • RxCUI (RxNorm): 797544
Why two NDCs? The FDA registers this code as 71894-120-02 — 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 → 71894-0120-02. 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 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.

Clinical

Label name ZOLGENSMA 2.6-3.0 KG KIT Ingredient Onasemnogene Abeparvovec-Xioi
📖 What it is MedlinePlus · NLM

Onasemnogene abeparvovec-xioi injection is used to treat spinal muscular atrophy (an inherited condition that reduces muscle strength and movement) in certain infants and children less than 2 years old. Onasemnogene abeparvovec-xioi is in a class of medications called gene therapy. It works by replacing a defective gene which helps preserve muscle and nerve function.

Read the full MedlinePlus article ↗
📗 Our plain-language guide HelloPharmacist
  • Zolgensma delivers a working copy of the gene your child was born without — the SMN1 gene — directly into their cells using a specially designed viral carrier. Without this gene, t...
  • What exactly does Zolgensma do for my child?
  • Yes, Zolgensma is given just once. The therapy is designed to deliver a lasting copy of the needed gene. Giving it a second time has not been studied, and it isn't recommended — in...
  • Is this really a one-time treatment? Will my child ever need it again?
📖 Read our full Onasemnogene Abeparvovec-Xioi guide →

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.

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.
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.

Packaging — all sizes for this product

Package NDCDescription Marketing startMarketing endStatus
71894-0120-02 You're viewing this Main listing 1 KIT in 1 CARTON * 8.3 mL in 1 VIAL * 1 mL in 1 POUCH 2019-05-24 — Active

Therapeutic equivalents

ProductLabelerPackNADAC/unitTEStatusPrice vs. this
Zolgensma 71894-0135-07 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0138-08 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0150-12 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0136-08 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0140-09 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0142-10 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0152-13 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0155-14 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0123-03 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0125-04 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0130-06 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0147-11 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0154-14 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0124-04 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0126-04 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0128-05 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0133-07 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0153-13 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0156-14 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0131-06 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0145-11 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0122-03 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0127-05 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0137-08 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0149-12 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0151-13 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0132-06 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0134-07 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0139-09 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0143-10 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0146-11 Novartis 1 kit — — FDA listed —
Zolgensmathis 71894-0120-02 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0144-10 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0121-03 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0129-05 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0141-09 Novartis 1 kit — — FDA listed —
Zolgensma 71894-0148-12 Novartis 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

🏛️
2019
First FDA approval
May 2019
📍
2026
Currently FDA-listed
7 years listed
🛡️
2031
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 May 2031. This may affect when biosimilars become widely available, but it is not a guaranteed launch date.
📅 FDA approved May 24, 2019 ⏳ ~4.6 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
Itvismaonasemnogene abeparvovec-brve Zolgensmaonasemnogene abeparvovec-xioi
🔒 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
2019 2021 2023 2025 2027 2029 2031
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 dateMay 24, 2031
Common questions
Is there a biosimilar for ZOLGENSMA 2.6-3.0 KG KIT?
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.

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.

Manufacturer & labeler

LabelerNovartis Innovative Technologies Inc.
FDA applicationBLA125694 (BLA)
Labeler code71894
First marketedMay 2019
Product typeHuman Prescription Drug
Portfolio38 products on file

More NDCs from Novartis Innovative Technologies Inc. labeler code 71894

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

Full prescribing information FDA SPL

The complete FDA label for this product — the official prescribing information, verbatim, section by section. Very long sections are excerpted here and marked; the full text is on DailyMed (linked in the sources below). Jump with a chip, search within the label, or expand everything.
🚨 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 🧬 Clinical Pharmacology 🧬 Mechanism of Action 📦 How Supplied / Storage and Handling 📋 Description 💬 Information for Patients 🧬 Pharmacokinetics 🧬 Pharmacodynamics 🔬 Clinical Studies 🧪 Nonclinical Toxicology 📄 Carcinogenesis, Mutagenesis, Impairment of Fertility 📄 Package Label / Principal Display Panel
🚨 Boxed Warning 214 words ▾

WARNING: SERIOUS LIVER INJURY and ACUTE LIVER FAILURE Cases of acute liver failure with fatal outcomes have been reported. Acute serious liver injury and elevated aminotransferases can also occur with ZOLGENSMA [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 of all patients by clinical examination and laboratory testing. Administer systemic corticosteroid to all patients before and after ZOLGENSMA infusion. Continue to monitor liver function for at least 3 months after infusion, and at other times as clinically indicated [see Dosage and Administration ( 2.1 , 2.3 )].

WARNING: SERIOUS LIVER INJURY and ACUTE LIVER FAILURE See full prescribing information for complete boxed warning. Cases of acute liver failure with fatal outcomes have been reported. Acute serious liver injury and elevated aminotransferases can also occur with ZOLGENSMA.

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

Continue to monitor liver function for at least 3 months after infusion, and at other times as clinically indicated. ( 2.1 , 2.3 )

🎯 Indications and Usage 181 words ▾

1 INDICATIONS AND USAGE ZOLGENSMA is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Limitations of Use The safety and effectiveness of repeat administration of ZOLGENSMA have not been evaluated [see Adverse Reactions ( 6.2 )] . The use of ZOLGENSMA in patients with advanced SMA (e.g., complete paralysis of limbs, permanent ventilator-dependence) has not been evaluated [see Clinical Studies ( 14 )] .

ZOLGENSMA is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 ( SMN1) gene. ( 1 ) Limitations of Use The safety and effectiveness of repeat administration of ZOLGENSMA have not been evaluated. ( 1 , 6.2 ) The use of ZOLGENSMA in patients with advanced SMA (e.g., complete paralysis of limbs, permanent ventilator dependence) has not been evaluated.

( 1 , 14 )

⏱️ Dosage and Administration ~3 min read ▾

2 DOSAGE AND ADMINISTRATION For single-dose intravenous infusion only. For single-dose intravenous infusion only ( 2 ). The recommended dosage of ZOLGENSMA is 1.1 × 10 14 vector genomes (vg) per kg of body weight.

( 2.1 ) Administer ZOLGENSMA as an intravenous infusion over 60 minutes. ( 2.1 , 2.3 ) Postpone ZOLGENSMA in patients with infections until the infection has resolved and the patient is clinically stable. ( 2.1 , 5.2 ) Starting one day prior to ZOLGENSMA infusion, administer systemic corticosteroids equivalent to oral prednisolone at 1 mg/kg of body weight per day for a total of 30 days.

At the end of the 30-day period, check liver function by clinical examination and by laboratory testing. For patients with unremarkable findings, taper the corticosteroid dose gradually over the next 28 days. If liver function abnormalities persist, continue systemic corticosteroids (equivalent to oral prednisolone at 1 mg/kg/day) until findings become unremarkable, and then taper the corticosteroid dose gradually over the next 28 days or longer if needed.

Do not stop systemic corticosteroids abruptly. ( 2.1 ) If liver function abnormalities continue to persist ≥ 2 × ULN after the 30-day period of systemic corticosteroids, promptly consult a pediatric gastroenterologist or hepatologist. ( 2.1 )

2.1Dose and Administration The recommended dose of ZOLGENSMA is 1.1 × 10 14 vector genomes per kilogram (vg/kg) of body weight. Table 1: Dosing Patient weight range (kg) Dose volume a (mL) a Dose volume is calculated using the upper limit of the patient weight range for pediatric patients less than 2 years of age between 2.6 kg and 21.0 kg. 2.6 – 3.0 16.5 3.1 – 3.5 19.3 3.6 – 4.0 22.0 4.1 – 4.5 24.8 4.6 – 5.0 27.5 5.1 – 5.5 30.3 5.6 – 6.0 33.0 6.1 – 6.5 35.8 6.6 – 7.0 38.5 7.1 – 7.5 41.3 7.6 – 8.0 44.0 8.1 – 8.5 46.8 8.6 – 9.0 49.5 9.1 – 9.5 52.3 9.6 – 10.0 55.0 10.1 – 10.5 57.8 10.6 – 11.0 60.5 11.1 – 11.5 63.3 11.6 – 12.0 66.0 12.1 – 12.5 68.8 12.6 – 13.0 71.5 13.1 – 13.5 74.3 13.6 – 14.0 77.0 14.1 – 14.5 79.8 14.6 – 15.0 82.5 15.1 – 15.5 85.3 15.6 – 16.0 88.0 16.1 – 16.5 90.8 16.6 – 17.0 93.5 17.1 – 17.5 96.3 17.6 – 18.0 99.0 18.1 – 18.5 101.8 18.6 – 19.0 104.5 19.1 – 19.5 107.3 19.6 – 20.0 110.0 20.1 – 20.5 112.8 20.6 – 21.0 115.5 Prior to ZOLGENSMA infusion: Due to the increased risk of serious systemic immune response, administer ZOLGENSMA to patients who are clinically stable in their overall baseline health status (e.g., hydration and nutritional status, absence of infection) prior to infusion.

Postpone ZOLGENSMA in patients with infections until the infection has resolved and the patient is clinically stable. Clinical signs or symptoms of infection should not be evident at the time of ZOLGENSMA infusion [see Warnings and Precautions ( 5.2 , 5.4 ), Patient Counseling Information ( 17 )]. Assess liver function [see Boxed Warning, Dosage and Administration ( 2.3 ), Warnings and Precautions ( 5.1 ), Use in Specific Populations ( 8.6 )].

Obtain creatinine and complete blood count (including hemoglobin and platelet count) [see Dosage and Administration ( 2.3 ), Warnings and Precautions ( 5.3 , 5.4 )] . Perform baseline testing for the presence of anti-AAV9 antibodies [see Dosage and Administration ( 2.3 ), Adverse Reactions ( 6.2 )]. One day prior to ZOLGENSMA infusion, begin administration of systemic corticosteroids equivalent to oral prednisolone at 1 mg per kg of body weight per day (mg/kg/day) for a total of 30 days.

Administer ZOLGENSMA as a single-dose intravenous infusion through a venous catheter. Follow the steps below for infusion: 1. Place a primary catheter into a vein (generally a peripheral vein in the arm or leg).

Insertion of a back-up catheter is recommended. 2. Program syringe pump for saline priming, or prime tubing manually with saline.

3. Administer ZOLGENSMA as a slow infusion over 60 minutes. DO NOT INFUSE AS AN INTRAVENOUS PUSH OR BOLUS.

4. Flush line with saline following completion of infusion. Monitor liver func… [Excerpted — this section continues on DailyMed.]

💊 Dosage Forms and Strengths 161 words ▾

3 DOSAGE FORMS AND STRENGTHS ZOLGENSMA is a suspension for intravenous infusion. ZOLGENSMA is provided in a kit containing 2 to 14 vials. Vials are provided in 2 fill volumes: 5.5 mL or 8.3 mL.

ZOLGENSMA has a nominal concentration of 2.0 × 10 13 vg/mL, and each vial contains an extractable volume of not less than either 5.5 mL or 8.3 mL. The intravenous dosage is determined by patient body weight, with a recommended dose of 1.1 × 10 14 vg/kg for pediatric patients. ZOLGENSMA is a suspension for intravenous infusion, supplied as single-use vials.

( 3 ) ZOLGENSMA is provided in a kit containing 2 to 14 vials, as a combination of 2 vial fill volumes (either 5.5 mL or 8.3 mL). All vials have a nominal concentration of 2.0 × 10 13 vector genomes (vg) per mL. Each vial of ZOLGENSMA contains an extractable volume of not less than either 5.5 mL or 8.3 mL.

( 3 )

⛔ Contraindications 7 words ▾

4 CONTRAINDICATIONS None. None. ( 4 )

⚠️ Warnings and Cautions ~3 min read ▾

5 WARNINGS AND PRECAUTIONS Systemic Immune Response: Administer ZOLGENSMA to patients who are clinically stable in their overall baseline health status (e.g., hydration and nutritional status, absence of infection) prior to infusion. ( 5.2 ) Thrombocytopenia: Monitor platelet counts before ZOLGENSMA infusion, and at least weekly for the first month and then every other week for the second and third month or until platelet counts return to baseline. ( 2.3 , 5.3 ) Thrombotic Microangiopathy (TMA): Prompt attention to signs and symptoms of TMA is advised, as TMA can result in life-threatening or fatal outcomes.

If clinical signs, symptoms and/or laboratory findings occur, consult a pediatric hematologist and/or pediatric nephrologist immediately to manage as clinically indicated. ( 5.4 ) Elevated Troponin I: Increases in cardiac troponin I levels have occurred following ZOLGENSMA infusion. Consider cardiac evaluation after ZOLGENSMA infusion and consult a cardiologist as needed.

( 5.5 ) AAV Vector Integration and Risk of Tumorigenicity: There is a theoretical risk of tumorigenicity due to integration of AAV vector DNA into the genome. Report cases of tumors in patients who received ZOLGENSMA, to Novartis Gene Therapies, Inc. ( 5.6 , 17 ) Infusion-Related Reactions: Monitor patients during and after ZOLGENSMA infusion.

Interrupt ZOLGENSMA infusion if infusion-related reaction occurs and administer supportive treatment as appropriate. Infusion of ZOLGENSMA may be resumed based on clinical assessment. ( 5.7 )

5.1Acute Serious Liver Injury, Acute Liver Failure or Elevated Aminotransferases Acute serious liver injury, acute liver failure and elevated aminotransferases can occur with ZOLGENSMA. Hepatotoxicity (which may be immune-mediated), generally manifested as elevated ALT and/or AST levels. Acute serious liver injury and acute liver failure, including fatal cases, have been reported with ZOLGENSMA use [see Adverse Reactions ( 6 )] .

In order to mitigate potential aminotransferase elevations, administer systemic corticosteroid to all patients before and after ZOLGENSMA infusion. Immune-mediated hepatotoxicity may require adjustment of the corticosteroid treatment regimen, including longer duration, increased dose, or prolongation of the corticosteroid taper [see Dosage and Administration ( 2.1 )] . Patients with preexisting liver impairment or acute hepatic viral infection may be at higher risk of acute serious liver injury/acute liver failure.

Patients with ALT, AST, or total bilirubin levels (except due to neonatal jaundice) > 2 × ULN have not been studied in clinical trials with ZOLGENSMA. Carefully consider the risks and benefits of ZOLGENSMA therapy in patients with preexisting liver impairment. Although in the clinical trials and in postmarketing experience, asymptomatic aminotransferase elevations were very commonly reported [see Adverse Reactions ( 6.1 )] , in the managed access program and in the postmarketing setting, cases of acute serious liver injury and acute liver failure, including a few cases with fatal outcomes, have been reported.

Some patients have experienced elevations in ALT and AST > 20 × ULN, prolonged prothrombin time and have been symptomatic (e.g., vomiting, jaundice), which required the use of corticosteroids, sometimes with prolonged duration and/or a higher dose. If acute serious liver injury or acute liver failure is suspected, promptly consult a pediatric gastroenterologist or hepatologist. Prior to ZOLGENSMA infusion, assess liver function by clinical examination and laboratory testing (hepatic aminotransferases [AST and ALT], total bilirubin level, albumin, prothrombin time, PTT, and INR).

Continue to monitor liver function (AST, ALT, total bilirubin, prothrombin time, INR) for at least 3 months after ZOLGENSMA infusion, and at other times as clinically indicated. Promptly assess and closely monitor patients with worsening liver function test results and/or signs or symptoms of acute illness (e.g… [Excerpted — this section continues on DailyMed.]

🤒 Adverse Reactions ~3 min read ▾

6 ADVERSE REACTIONS The most common adverse reactions (incidence ≥ 5%) were elevated aminotransferases and vomiting. The most common adverse reactions (incidence ≥ 5%) were elevated aminotransferases and vomiting. ( 6 ) To report SUSPECTED ADVERSE REACTIONS, contact Novartis Gene Therapies at 1-833-828-3947 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 product and may not reflect the rates observed in practice. The safety data described in this section reflect exposure to ZOLGENSMA in five clinical studies enrolling a total of 68 patients. This includes four prospective open-label clinical trials [NCT03306277 (Study 1), NCT02122952 (Study 2), NCT03505099, NCT04851873 (Study 3)], and one observational long-term follow-up study [NCT03421977].

The patient population in NCT03306277, NCT03505099, and NCT02122952 ranged in age from 0.3 months to 7.9 months at the time of infusion (median age, 3.3 months), with weight range from 3.0 kg to 8.4 kg (median weight, 5.5 kg) [see Clinical Studies ( 14 )] . In an open-label, post-authorization clinical study (Study 3, NCT04851873), safety of ZOLGENSMA was evaluated in 24 children, aged between 1.5 to 9.1 years (median age, 4.9 years), with weight range from ≥ 8.5 kg to ≤ 21 kg (median weight, 15.8 kg). Only one of the 24 patients was under the age of 2 years at the time of ZOLGENSMA administration.

Patients in Study 3 had 2 to 4 copies of SMN2 . Before treatment with ZOLGENSMA, 21 patients discontinued their previous treatment with nusinersen or risdiplam. The types of adverse reactions observed in Study 3 were consistent with those of Studies 1 and 2.

Liver enzyme increases in Study 3 occurred at a higher frequency compared with the previous 4 studies. AST or ALT elevations > 2 × ULN were observed in the majority of patients (23 out of 24 patients), including 21 patients with ALT elevations > 3 × ULN and 5 patients with ALT elevations > 20 × ULN. These patients were clinically asymptomatic and there were no elevations of bilirubin.

The AST and ALT elevations were managed with the use of corticosteroids, typically with prolonged duration and/or given at a higher dose [see Warnings and Precautions ( 5.1 )] . Transient decreases in platelet counts, which met the criteria for thrombocytopenia were observed in 20 out of 24 patients. Four patients had platelet counts below 50,000 per µL [see Warnings and Precautions ( 5.3 )] .

The most frequent adverse reactions (incidence ≥ 5%) and increases in alanine aminotransferase in the 4 studies (data cut-off date: September 27, 2018) are summarized in Table 2 . Table 2: Adverse Reactions and ALT Increases* Following Treatment With ZOLGENSMA Patients n = 44 (3.0-8.4 kg) Adverse reactions n (%) Abbreviations: ULN, upper limit of normal; ALT, alanine aminotransferase. *Laboratory finding. Elevated aminotransferases 12 (27%) ALT > 3 X ULN 7 (16%) ALT > 20 X ULN 4 (9%) Vomiting 3 (7%) One death occurred in a patient, who received ZOLGENSMA at the age of 5 months (6 kg), in a completed non-United States clinical trial (NCT03461289).

The patient initially presented with respiratory insufficiency 12 days after ZOLGENSMA infusion and was found to have RSV and parainfluenza in respiratory secretions. The patient had episodes of serious hypotension, followed by seizures, and was found to have leukoencephalopathy (brain white matter defects) approximately 30 days after ZOLGENSMA infusion. The patient died after withdrawal of life support 52 days after ZOLGENSMA infusion.

6.2Postmarketing Experience The following adverse reactions have been identified during post-approval use of ZOLGENSMA. Because these reactions are reported voluntarily, it is not always possible to reliably estimate their frequency or establish a causal relat… [Excerpted — this section continues on DailyMed.]

🔄 Drug Interactions 105 words ▾

7 DRUG INTERACTIONS Where feasible, adjust a patient’s vaccination schedule to accommodate concomitant corticosteroid administration prior to and following ZOLGENSMA infusion [see Dosage and Administration ( 2.1 )] . Certain vaccines, such as measles, mumps, and rubella (MMR) and varicella, are contraindicated for patients on a substantially immunosuppressive steroid dose (i.e., ≥ 2 weeks of daily receipt of 20 mg or 2 mg/kg body weight of prednisone or equivalent). Seasonal RSV prophylaxis is recommended (General Best Practice Guidelines for Immunization [ www.cdc.gov/vaccines/hcp/acip-recs/general-recs/downloads/general-recs.pdf ], eds2017).

Where feasible, adjust a patient’s vaccination schedule to accommodate concomitant corticosteroid administration prior to and following ZOLGENSMA infusion. ( 7 )

👥 Use in Specific Populations ~2 min read ▾

8 USE IN SPECIFIC POPULATIONS Pediatric Use: Use of ZOLGENSMA in premature neonates before reaching full term gestational age is not recommended because concomitant treatment with corticosteroids may adversely affect neurological development. Delay ZOLGENSMA infusion until full-term gestational age is reached. ( 8.4 )

8.1Pregnancy Risk Summary There are no available data regarding ZOLGENSMA use in pregnant women. It is not known whether ZOLGENSMA has the potential to be transferred to the fetus in humans. Animal reproduction studies in mice have shown no risk of fetal abnormalities nor transfer to mouse fetus with intravenous (IV) administration of onasemnogene abeparvovec, the active ingredient found in ZOLGENSMA, on gestational day 6 at dose levels equivalent to the clinical IV dose of ZOLGENSMA [see Carcinogenesis, Mutagenesis, Impairment of Fertility ( 13.1 )] , see Animal Data.

In the United States general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. Data Animal Data In an embryo-fetal development study in mice, pregnant animals received an IV dose of either 1.1 x 10 13 vg/kg or 1.1 x 10 14 vg/kg of onasemnogene abeparvovec on gestational day 6, which resulted in no evidence of maternal toxicity, embryo-fetal toxicity, teratogenicity, or reduced viability. Onasemnogene abeparvovec DNA was detected in the placenta at low levels but was not detected in any fetal tissue at gestational day 18.

The no observed adverse effect level for maternal and developmental toxicity is 1.1 x 10 14 vg/kg, which is the recommended clinical IV dose of ZOLGENSMA.

8.2Lactation Risk Summary There is no information available on the presence of ZOLGENSMA in human milk, the effects on the breastfed infant, or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for ZOLGENSMA and any potential adverse effects on the breastfed child from ZOLGENSMA or from the underlying maternal condition. There is no information on whether breastfeeding should be restricted in mothers who may be seropositive for anti-AAV9 antibodies.

8.4Pediatric Use The safety of ZOLGENSMA was studied in pediatric patients who received ZOLGENSMA infusion at age 0.3 to 7.9 months (weight range, 3.0 kg to 8.4 kg). Safety was also studied in Study 3 (post-authorization study) in patients weighing 9.5 kg to 20.2 kg [see Adverse Reactions ( 6 )] . The efficacy of ZOLGENSMA was studied in pediatric patients who received ZOLGENSMA infusion at age 0.5 to 7.9 months (weight range, 3.6 kg to 8.4 kg) [see Clinical Studies ( 14 )] .

Administration of ZOLGENSMA to premature neonates before reaching full-term gestational age is not recommended, because concomitant treatment with corticosteroids may adversely affect neurological development. Delay ZOLGENSMA infusion until the corresponding full-term gestational age is reached.

8.6Hepatic Impairment ZOLGENSMA therapy should be carefully considered in patients with liver impairment. Cases of acute serious liver injury and acute liver failure have been reported with ZOLGENSMA in patients with preexisting liver abnormalities. In clinical trials, elevation of aminotransferases was observed in patients following ZOLGENSMA infusion [see Warnings and Precautions ( 5.1 )] .

🤰 Pregnancy 218 words ▾

8.1Pregnancy Risk Summary There are no available data regarding ZOLGENSMA use in pregnant women. It is not known whether ZOLGENSMA has the potential to be transferred to the fetus in humans. Animal reproduction studies in mice have shown no risk of fetal abnormalities nor transfer to mouse fetus with intravenous (IV) administration of onasemnogene abeparvovec, the active ingredient found in ZOLGENSMA, on gestational day 6 at dose levels equivalent to the clinical IV dose of ZOLGENSMA [see Carcinogenesis, Mutagenesis, Impairment of Fertility ( 13.1 )] , see Animal Data.

In the United States general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. Data Animal Data In an embryo-fetal development study in mice, pregnant animals received an IV dose of either 1.1 x 10 13 vg/kg or 1.1 x 10 14 vg/kg of onasemnogene abeparvovec on gestational day 6, which resulted in no evidence of maternal toxicity, embryo-fetal toxicity, teratogenicity, or reduced viability. Onasemnogene abeparvovec DNA was detected in the placenta at low levels but was not detected in any fetal tissue at gestational day 18.

The no observed adverse effect level for maternal and developmental toxicity is 1.1 x 10 14 vg/kg, which is the recommended clinical IV dose of ZOLGENSMA.

🧒 Pediatric Use 121 words ▾

8.4Pediatric Use The safety of ZOLGENSMA was studied in pediatric patients who received ZOLGENSMA infusion at age 0.3 to 7.9 months (weight range, 3.0 kg to 8.4 kg). Safety was also studied in Study 3 (post-authorization study) in patients weighing 9.5 kg to 20.2 kg [see Adverse Reactions ( 6 )] . The efficacy of ZOLGENSMA was studied in pediatric patients who received ZOLGENSMA infusion at age 0.5 to 7.9 months (weight range, 3.6 kg to 8.4 kg) [see Clinical Studies ( 14 )] .

Administration of ZOLGENSMA to premature neonates before reaching full-term gestational age is not recommended, because concomitant treatment with corticosteroids may adversely affect neurological development. Delay ZOLGENSMA infusion until the corresponding full-term gestational age is reached.

🧬 Clinical Pharmacology ~2 min read ▾

12 CLINICAL PHARMACOLOGY

12.1Mechanism of Action ZOLGENSMA is a recombinant AAV9-based gene therapy designed to deliver a copy of the gene encoding the human SMN protein. SMA is caused by a bi-allelic mutation in the SMN1 gene, which results in insufficient SMN protein expression. Intravenous administration of ZOLGENSMA that results in cell transduction and expression of the SMN protein has been observed in two human case studies [see Clinical Pharmacology ( 12.3 )] .

12.2Pharmacodynamics There are no clinically relevant pharmacodynamics data for ZOLGENSMA.

12.3Pharmacokinetics Vector shedding after infusion with ZOLGENSMA was investigated at multiple time points during Study 2. Samples of saliva, urine and stool were collected the day after infusion, weekly through Day 30, and then monthly through Month 12 and every 3 months thereafter. Samples from 5 patients were used for ZOLGENSMA vector DNA shedding analysis through the Month 18 visit.

Vector DNA was shed in saliva, urine and stool after infusion of ZOLGENSMA, with much higher concentrations of vector DNA found in stool than in saliva or urine. The vector DNA concentration in saliva was low on Day 1 after infusion and declined to undetectable levels within 3 weeks. In urine, the vector DNA concentration was very low on Day 1 after infusion and declined to undetectable levels within 1 to 2 weeks.

In stool, the vector DNA concentration was much higher than in saliva or urine for 1 to 2 weeks after infusion and declined to undetectable levels by 1 to 2 months after infusion. Biodistribution was evaluated in two patients who died 5.7 months and 1.7 months, respectively, after infusion of ZOLGENSMA at the dose of 1.1 x 10 14 vg/kg. Both cases showed that the highest levels of vector DNA were found in the liver.

Vector DNA was also detected in the spleen, heart, pancreas, inguinal lymph node, skeletal muscles, peripheral nerves, kidney, lung, intestines, gonads, spinal cord, brain, and thymus. Immunostaining for SMN protein showed generalized SMN expression in spinal motor neurons, neuronal and glial cells of the brain, and in the heart, liver, skeletal muscles, and other tissues evaluated.

12.6Immunogenicity In ZOLGENSMA clinical trials, patients were required to have baseline anti-AAV9 antibody titers of ≤ 1:50, measured using an enzyme-linked immunosorbent assay (ELISA). Evidence of prior exposure to AAV9 was uncommon. The safety and efficacy of ZOLGENSMA in patients with anti-AAV9 antibody titers above 1:50 have not been evaluated.

Perform baseline testing for the presence of anti AAV9 antibodies prior to ZOLGENSMA infusion. Retesting may be performed if anti-AAV9 antibody titers are reported as > 1:50 [see Dosage and Administration ( 2.1 , 2.3 )] . Following ZOLGENSMA infusion, increases from baseline in anti-AAV9 antibody titers occurred in all patients.

In Study 2, anti-AAV9 antibody titers reached at least 1:102,400 in every patient, and titers exceeded 1:819,200 in most patients. Re administration of ZOLGENSMA in the presence of high anti-AAV9 antibody titer has not been evaluated.

🧬 Mechanism of Action 72 words ▾

12.1Mechanism of Action ZOLGENSMA is a recombinant AAV9-based gene therapy designed to deliver a copy of the gene encoding the human SMN protein. SMA is caused by a bi-allelic mutation in the SMN1 gene, which results in insufficient SMN protein expression. Intravenous administration of ZOLGENSMA that results in cell transduction and expression of the SMN protein has been observed in two human case studies [see Clinical Pharmacology ( 12.3 )] .

📦 How Supplied / Storage and Handling ~2 min read ▾

16 HOW SUPPLIED/STORAGE AND HANDLING

16.1How Supplied ZOLGENSMA is shipped frozen (≤ -60°C [-76°F]) in 10 mL vials with 2 fill volumes (either 5.5 mL or 8.3 mL). ZOLGENSMA is provided as a customized kit to meet dosing requirements for each patient [see Dosage and Administration ( 2.1 )] , with each kit containing: Two (2) to fourteen (14) vials of ZOLGENSMA (see below) One alcohol wipe per vial Kit sizes and National Drug Codes (NDC) are provided in Table 3 . Table 3: ZOLGENSMA Kit Sizes Patient weight (kg) 5.5 mL vial a 8.3 mL vial b Total vials per kit NDC number a Vial nominal concentration is 2.0 × 10 13 vg/mL and contains an extractable volume of not less than 5.5 mL. b Vial nominal concentration is 2.0 × 10 13 vg/mL and contains an extractable volume of not less than 8.3 mL.

2.6– 3.0 0 2 2 71894-120-02 3.1 – 3.5 2 1 3 71894-121-03 3.6 – 4.0 1 2 3 71894-122-03 4.1 – 4.5 0 3 3 71894-123-03 4.6 – 5.0 2 2 4 71894-124-04 5.1 – 5.5 1 3 4 71894-125-04 5.6 – 6.0 0 4 4 71894-126-04 6.1 – 6.5 2 3 5 71894-127-05 6.6 – 7.0 1 4 5 71894-128-05 7.1 – 7.5 0 5 5 71894-129-05 7.6 – 8.0 2 4 6 71894-130-06 8.1 – 8.5 1 5 6 71894-131-06 8.6 – 9.0 0 6 6 71894-132-06 9.1 – 9.5 2 5 7 71894-133-07 9.6 – 10.0 1 6 7 71894-134-07 10.1 – 10.5 0 7 7 71894-135-07 10.6 – 11.0 2 6 8 71894-136-08 11.1 – 11.5 1 7 8 71894-137-08 11.6 – 12.0 0 8 8 71894-138-08 12.1 – 12.5 2 7 9 71894-139-09 12.6 – 13.0 1 8 9 71894-140-09 13.1 – 13.5 0 9 9 71894-141-09 13.6 – 14.0 2 8 10 71894-142-10 14.1 – 14.5 1 9 10 71894-143-10 14.6 – 15.0 0 10 10 71894-144-10 15.1 – 15.5 2 9 11 71894-145-11 15.6 – 16.0 1 10 11 71894-146-11 16.1 – 16.5 0 11 11 71894-147-11 16.6 – 17.0 2 10 12 71894-148-12 17.1 – 17.5 1 11 12 71894-149-12 17.6 – 18.0 0 12 12 71894-150-12 18.1 – 18.5 2 11 13 71894-151-13 18.6 – 19.0 1 12 13 71894-152-13 19.1 – 19.5 0 13 13 71894-153-13 19.6 – 20.0 2 12 14 71894-154-14 20.1 – 20.5 1 13 14 71894-155-14 20.6 – 21.0 0 14 14 71894-156-14

16.2Storage and Handling Product is shipped and delivered frozen (≤ -60°C [-76°F]) in clear vials. Upon receipt, immediately place the kit in a refrigerator at 2°C to 8°C (36°F to 46°F). ZOLGENSMA is stable for 14 days from receipt when stored at 2°C to 8°C (36°F to 46°F). DO NOT REFREEZE . Must use within 14 days of receipt.

📋 Description 104 words ▾

11 DESCRIPTION ZOLGENSMA is a suspension of an adeno-associated viral vector-based gene therapy for intravenous infusion. It is a recombinant self-complementary AAV9 containing a transgene encoding the human survival motor neuron (SMN) protein, under the control of a cytomegalovirus enhancer/chicken-β-actin hybrid promoter. ZOLGENSMA has a nominal concentration of 2.0 × 10 13 vg/mL.

Each vial contains an extractable volume of not less than either 5.5 mL or 8.3 mL and the excipients 20 mM Tris (pH 8.0), 1 mM magnesium chloride (MgCl 2 ), 200 mM sodium chloride (NaCl) and 0.005% poloxamer 188. ZOLGENSMA is packaged as a sterile suspension and contains no preservative.

💬 Information for Patients ~3 min read ▾

17 PATIENT COUNSELING INFORMATION Acute Serious Liver Injury, Acute Liver Failure or Elevated Aminotransferases Inform caregivers that ZOLGENSMA could increase liver enzyme levels and cause acute serious liver injury or acute liver failure, and death. Inform caregivers that patients will receive an oral corticosteroid medication before and after infusion with ZOLGENSMA, and will undergo regular blood tests to monitor liver function. Advise caregivers to contact their 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 decrease in alertness [see Warnings and Precautions ( 5.1 )] .

Vaccination Before and After Infusion With ZOLGENSMA Advise caregivers to consult with their healthcare provider to determine if adjustments to the patient’s vaccination schedule are necessary during corticosteroid use. Inform caregivers that where feasible, the vaccination schedule should be adjusted appropriately to accommodate treatment with corticosteroid. Prophylaxis against influenza and RSV is recommended and vaccination status should be up-to-date prior to ZOLGENSMA administration.

Please consult your healthcare provider [see Drug Interactions ( 7 )] . Systemic Immune Response Caregivers should be aware that an infection (e.g., cold, flu, gastroenteritis, otitis media, bronchiolitis, etc.) before or after ZOLGENSMA infusion could lead to more serious complications. Caregivers and close contacts of patients should follow infection prevention practices (e.g., hand hygiene, coughing/sneezing etiquette, limiting potential contacts).

Advise caregivers of the signs of a possible infection, such as coughing, wheezing, sneezing, runny nose, sore throat, or fever. Caregivers should contact their healthcare provider immediately if the patient experiences any symptoms suggestive of infection before or after ZOLGENSMA infusion [see Warnings and Precautions ( 5.2 )] . Thrombocytopenia Inform caregivers that ZOLGENSMA could decrease blood platelet count and increase the risk of bruising or bleeding.

Inform caregivers that thrombocytopenia has been reported to generally occur within the first two weeks after ZOLGENSMA infusion. Advise caregivers to seek medical attention if the patient experiences unexpected bruising or bleeding [see Warnings and Precautions ( 5.3 )] . Thrombotic Microangiopathy Inform caregivers that ZOLGENSMA could decrease blood platelet and red blood cell counts, cause acute kidney injury, and increase the risk of bruising or bleeding, which may be indicative of TMA.

Inform caregivers that TMA has been reported to generally occur within the first two weeks after ZOLGENSMA infusion. Advise caregivers to seek immediate medical attention if the patient experiences unexpected bruising or bleeding, seizures, or decreased urine output [see Warnings and Precautions ( 5.4 )] . AAV Vector Integration and Risk of Tumorigenicity Inform caregivers that there is a theoretical risk of tumorigenicity with AAV therapies such as ZOLGENSMA.

Advise caregivers to contact their healthcare provider and Novartis Gene Therapies, Inc. (1-833-828-3947) if the patient who received ZOLGENSMA develops a tumor [see Warnings and Precautions ( 5.6 )] . Vector Shedding Temporary vector shedding of ZOLGENSMA occurs primarily through body waste.

Advise caregivers on the proper handling of patient feces; recommended procedures include sealing disposable diapers in disposable trash bags and then discarding into regular trash. Provide instructions to caregivers and family members regarding proper hand hygiene when coming into direct contact with patient body waste. These precautions should be followed for one month after ZOLGENSMA infusion.

Infusion-Related Reactions Inform caregivers that infusion-related reactions may occur during and after ZOLGENSMA infusion. Advise caregivers to seek immediate medical evaluation if signs and sympto… [Excerpted — this section continues on DailyMed.]

🧬 Pharmacokinetics ~1 min read ▾

12.3Pharmacokinetics Vector shedding after infusion with ZOLGENSMA was investigated at multiple time points during Study 2. Samples of saliva, urine and stool were collected the day after infusion, weekly through Day 30, and then monthly through Month 12 and every 3 months thereafter. Samples from 5 patients were used for ZOLGENSMA vector DNA shedding analysis through the Month 18 visit.

Vector DNA was shed in saliva, urine and stool after infusion of ZOLGENSMA, with much higher concentrations of vector DNA found in stool than in saliva or urine. The vector DNA concentration in saliva was low on Day 1 after infusion and declined to undetectable levels within 3 weeks. In urine, the vector DNA concentration was very low on Day 1 after infusion and declined to undetectable levels within 1 to 2 weeks.

In stool, the vector DNA concentration was much higher than in saliva or urine for 1 to 2 weeks after infusion and declined to undetectable levels by 1 to 2 months after infusion. Biodistribution was evaluated in two patients who died 5.7 months and 1.7 months, respectively, after infusion of ZOLGENSMA at the dose of 1.1 x 10 14 vg/kg. Both cases showed that the highest levels of vector DNA were found in the liver.

Vector DNA was also detected in the spleen, heart, pancreas, inguinal lymph node, skeletal muscles, peripheral nerves, kidney, lung, intestines, gonads, spinal cord, brain, and thymus. Immunostaining for SMN protein showed generalized SMN expression in spinal motor neurons, neuronal and glial cells of the brain, and in the heart, liver, skeletal muscles, and other tissues evaluated.

🧬 Pharmacodynamics 11 words ▾

12.2Pharmacodynamics There are no clinically relevant pharmacodynamics data for ZOLGENSMA.

🔬 Clinical Studies ~3 min read ▾

14 CLINICAL STUDIES The efficacy of ZOLGENSMA in pediatric patients less than 2 years of age with SMA with bi-allelic mutations in the SMN1 gene was evaluated in an open-label, single-arm clinical trial (Study 1, NCT03306277) and an open-label, single-arm, ascending-dose clinical trial (Study 2, NCT02122952). Patients experienced onset of clinical symptoms consistent with SMA before 6 months of age. All patients had genetically confirmed bi-allelic SMN1 gene deletions, 2 copies of the SMN2 gene, and absence of the c.859G>C modification in exon 7 of SMN2 gene (which predicts a milder phenotype).

All patients had baseline anti-AAV9 antibody titers of ≤ 1:50, measured by ELISA. In both trials, ZOLGENSMA was delivered as a single-dose intravenous infusion. Efficacy was established on the basis of survival, and achievement of developmental motor milestones, such as sitting without support.

Survival was defined as time from birth to either death or permanent ventilation. Permanent ventilation was defined as requiring invasive ventilation (tracheostomy), or respiratory assistance for 16 or more hours per day (including noninvasive ventilatory support) continuously for 14 or more days in the absence of an acute reversible illness, excluding perioperative ventilation. Efficacy was also supported by assessments of ventilator use, nutritional support and scores on the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND).

CHOP-INTEND is an assessment of motor skills in patients with infantile-onset SMA. Study 1 enrolled 21 patients (10 male and 11 female) with infantile-onset SMA. Before treatment with ZOLGENSMA, none of the 21 patients required noninvasive ventilator (NIV) support, and all patients could exclusively feed orally (i.e., no need for non-oral nutrition).

The mean CHOP-INTEND score at baseline was 31.0 (range, 18 to 47). All the patients received 1.1 × 10 14 vg/kg of ZOLGENSMA. The mean age of the 21 patients at the time of treatment was 3.9 months (range, 0.5 to 5.9 months).

As of the March 2019 data cutoff, 19 patients were alive without permanent ventilation (i.e., event-free survival) and were continuing in the trial, while one patient died at age 7.8 months due to disease progression, and one patient withdrew from the study at age 11.9 months. The 19 surviving patients who were continuing in the trial ranged in age from 9.4 to 18.5 months. By the data cutoff, 13 of the 19 patients continuing in the trial reached 14 months of age without permanent ventilation, one of the study’s co-primary efficacy endpoints.

In addition to survival, assessment of the other co-primary efficacy endpoint found that 10 of the 21 patients (47.6%) achieved the ability to sit without support for ≥ 30 seconds between 9.2 and 16.9 months of age (mean age was 12.1 months). Based on the natural history of the disease, patients who met the study entry criteria would not be expected to attain the ability to sit without support, and only approximately 25% of these patients would be expected to survive (i.e., being alive without permanent ventilation) beyond 14 months of age.

In addition, 16 of the 19 patients had not required daily NIV use. Comparison of the results of Study 1 to available natural history data of patients with infantile-onset SMA provides primary evidence of the effectiveness of ZOLGENSMA. Study 2 enrolled 15 patients (6 male and 9 female) with infantile-onset SMA, 3 in a low-dose cohort and 12 in a high-dose cohort.

At the time of treatment, the mean age of patients in the low-dose cohort was 6.3 months (range, 5.9 to 7.2 months), and 3.4 months (range, 0.9 to 7.9 months) in the high-dose cohort. The dosage received by patients in the low-dose cohort was approximately one-third of the dosage received by patients in the high-dose cohort. However, the precise dosages of ZOLGENSMA received by patients are unclear due to a change in the method of measuring ZOLGENSMA concentration, and to decreases in t… [Excerpted — this section continues on DailyMed.]

🧪 Nonclinical Toxicology ~2 min read ▾

13 NONCLINICAL TOXICOLOGY

13.1Carcinogenesis, Mutagenesis, Impairment of Fertility No animal studies have been performed to evaluate the effects of ZOLGENSMA on carcinogenesis or mutagenesis. In fertility and early embryonic development studies conducted in mice administered onasemnogene abeparvovec, the active ingredient found in ZOLGENSMA, intravenously (IV) at dose levels of 1.1 × 10 13 or 1.1 × 10 14 vector genomes (vg)/kg body weight, onasemnogene abeparvovec DNA was detected in gonadal tissues of both males and females. No adverse effects on male or female fertility, fecundity, or mating indices were observed.

No evidence of germ cell transduction or germline transmission was reported [see Use in Specific Populations ( 8.3 )] .

13.2Animal Toxicology and/or Pharmacology In toxicology studies conducted in neonatal mice, dose-dependent cardiac and hepatic toxicities were observed following intravenous (IV) administration of onasemnogene abeparvovec. Onasemnogene abeparvovec related findings in the myocardium, at doses of 7.9 × 10 13 vg/kg and higher, included slight to mild mononuclear cell inflammation accompanied by edema, slight to mild fibrosis, and scattered myocardial cell degeneration/regeneration. Additional cardiac findings at dose levels of 1.5 × 10 14 vg/kg and higher included minimal to moderate atrial thrombosis and slight to marked atrial dilation.

Liver findings included hepatocellular hypertrophy, Kupffer cell activation, perinuclear vacuolation, and scattered hepatocellular necrosis. Target organ toxicity in the heart and liver was associated with mortality in mice at dose levels of 2.4 × 10 14 vg/kg and above, approximately 2.2-fold higher than the recommended clinical dose level. In a 6-month toxicology study conducted in juvenile non-human primates, single IV administration of onasemnogene abeparvovec at the recommended clinical dose level of 1.1 × 10 14 vg/kg, with or without corticosteroid treatment, resulted in microscopic findings in the dorsal root ganglia (DRG), trigeminal ganglia (TG), spinal cord, brainstem, and liver.

At 6 weeks post-administration, microscopic findings included minimal to slight mononuclear cell inflammation and neuronal degeneration in the DRG and TG; axonal degeneration and gliosis in the spinal cord; mixed cell inflammation, gliosis, and axonal degeneration in the brainstem; and oval cell hyperplasia in the liver. These microscopic findings were still present at 6 months with decreased severity and incidence.

📄 Carcinogenesis, Mutagenesis, Impairment of Fertility 108 words ▾

13.1Carcinogenesis, Mutagenesis, Impairment of Fertility No animal studies have been performed to evaluate the effects of ZOLGENSMA on carcinogenesis or mutagenesis. In fertility and early embryonic development studies conducted in mice administered onasemnogene abeparvovec, the active ingredient found in ZOLGENSMA, intravenously (IV) at dose levels of 1.1 × 10 13 or 1.1 × 10 14 vector genomes (vg)/kg body weight, onasemnogene abeparvovec DNA was detected in gonadal tissues of both males and females. No adverse effects on male or female fertility, fecundity, or mating indices were observed.

No evidence of germ cell transduction or germline transmission was reported [see Use in Specific Populations ( 8.3 )] .

📄 Package Label / Principal Display Panel ~3 min read ▾

PRINCIPAL DISPLAY PANEL NDC 71894-110-01 onasemnogene abeparvovec-xioi ZOLGENSMA ® Rx ONLY Suspension for intravenous infusion. See enclosed prescribing information for dosage and directions for use. Manufactured by Novartis Gene Therapies, Inc.

Bannockburn, IL 60015. US License No: 2250 5.5 mL NOVARTIS PRINCIPAL DISPLAY PANEL NDC 71894-110-01 onasemnogene abeparvovec-xioi ZOLGENSMA® Rx ONLY Suspension for intravenous infusion. See enclosed prescribing information for dosage and directions for use.

Manufactured by Novartis Gene Therapies, Inc. Bannockburn, IL 60015. US License No: 2250 5.5 mL NOVARTIS

PRINCIPAL DISPLAY PANEL NDC 71894-115-01 onasemnogene abeparvovec-xioi ZOLGENSMA ® Rx ONLY Suspension for intravenous infusion. See enclosed prescribing information for dosage and directions for use. Manufactured by Novartis Gene Therapies, Inc.

Bannockburn, IL 60015. US License No: 2250 8.3 mL NOVARTIS PRINCIPAL DISPLAY PANEL NDC 71894-115-01 onasemnogene abeparvovec-xioi ZOLGENSMA® Rx ONLY Suspension for intravenous infusion. See enclosed prescribing information for dosage and directions for use.

Manufactured by Novartis Gene Therapies, Inc. Bannockburn, IL 60015. US License No: 2250 8.3 mL NOVARTIS

PRINCIPAL DISPLAY PANEL onasemnogene abeparvovec-xioi ZOLGENSMA ® Rx ONLY Suspension for intravenous infusion. NOVARTIS Manufactured by Novartis Gene Therapies, Inc. Bannockburn, IL 60015 US License No: 2250 ©2021 Novartis Gene Therapies, Inc.

PRINCIPAL DISPLAY PANEL onasemnogene abeparvovec-xioi ZOLGENSMA® Rx ONLY Suspension for intravenous infusion. NOVARTIS Manufactured by Novartis Gene Therapies, Inc. Bannockburn, IL 60015.

US License No: 2250 US License No: 2250 ©2021 Novartis Gene Therapies, Inc.

PRINCIPAL DISPLAY PANEL - NDC: 71894-120-02 - 2.6 - 3.0 kg Kit Variable Label 2.6 - 3.0 kg Kit Variable Label

PRINCIPAL DISPLAY PANEL - NDC: 71894-121-03 - 3.1 - 3.5 kg Kit Variable Label 3.1 - 3.5 kg Kit Variable Label

PRINCIPAL DISPLAY PANEL - NDC: 71894-122-03 - 3.6 - 4.0 kg Kit Variable Label 3.6 - 4.0 kg Kit Variable Label

PRINCIPAL DISPLAY PANEL - NDC: 71894-123-03 - 4.1 - 4.5 kg Kit Variable Label 4.1 - 4.5 kg Kit Variable Label

PRINCIPAL DISPLAY PANEL - NDC: 71894-124-04 - 4.6 - 5.0 kg Kit Variable Label 4.6 - 5.0 kg Kit Variable Label

PRINCIPAL DISPLAY PANEL - NDC: 71894-125-04 - 5.1 - 5.5 kg Kit Variable Label 5.1 - 5.5 kg Kit Variable Label

PRINCIPAL DISPLAY PANEL - NDC: 71894-126-04 - 5.6 - 6.0 kg Kit Variable Label 5.6 - 6.0 kg Kit Variable Label

PRINCIPAL DISPLAY PANEL - NDC: 71894-127-05 - 6.1 - 6.5 kg Kit Variable Label 6.1 - 6.5 kg Kit Variable Label

PRINCIPAL DISPLAY PANEL - NDC: 71894-128-05 - 6.6 - 7.0 kg Kit Variable Label 6.6 - 7.0 kg Kit Variable Label

PRINCIPAL DISPLAY PANEL - NDC: 71894-129-05 - 7.1 - 7.5 kg Kit Variable Label 7.1 - 7.5 kg Kit Variable Label

PRINCIPAL DISPLAY PANEL - NDC: 71894-130-06 - 7.6 - 8.0 kg Kit Variable Label 7.6 - 8.0 kg Kit Variable Label

PRINCIPAL DISPLAY PANEL - NDC: 71894-131-06 - 8.1 - 8.5 kg Kit Variable Label 8.1 - 8.5 kg Kit Variable Label

PRINCIPAL DISPLAY PANEL - NDC: 71894-132-06 - 8.6 – 9.0 kg Kit Variable Label 8.6 – 9.0 kg Kit Variable Label

PRINCIPAL DISPLAY PANEL - NDC: 71894-133-07 – 9.1 – 9.5 kg Kit Variable Label 9.1 – 9.5 kg Kit Variable Label

PRINCIPAL DISPLAY PANEL - NDC: 71894-134-07 – 9.6 – 10.0 kg Kit Variable Label 9.6 – 10.0 kg Kit Variable Label

PRINCIPAL DISPLAY PANEL - NDC: 71894-135-07 – 10.1 – 10.5 kg Kit Variable Label 10.1 – 10.5 kg Kit Variable Label

PRINCIPAL DISPLAY PANEL - NDC: 71894-136-08 – 10.6 – 11.0 kg Kit Variable Label 10.6 – 11.0 kg Kit Variable Label

PRINCIPAL DISPLAY PANEL - NDC: 71894-137-08 – 11.1 – 11.5 kg Kit Variable Label 11.1 – 11.5 kg Kit Variable Label

PRINCIPAL DISPLAY PANEL - NDC: 71894-138-08 – 11.6 – 12.0 kg Kit Variable Label 11.6 – 12.0 kg Kit Variable Label

PRINCIPAL DISPLAY PANEL - NDC: 71894-139-09 – 12.1 – 12.5 kg Kit Variable Label 12.1 – 12.5 kg Kit Variable Label

PRINCIPAL DISPLAY PANEL - NDC… [Excerpted — this section continues on DailyMed.]

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

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 — Not published for this NDC Most self-administered / retail products have no J-code — that is normal.
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 form printed on the packaging and shown on DailyMed is the one the FDA registered. Insurance claims use a fixed 11-digit 5-4-2 format, so the short segment is padded with a leading zero and the dashes are dropped. The Identity section at the top of this page lists each form of this code.
Is this package still being marketed?
Yes, per the latest FDA NDC Directory data on this page: this package is listed as actively marketed, with no marketing end date reported by Novartis Innovative Technologies Inc.. Listing status can change — the directory data on this page refreshes weekly.
Who lists this product with the FDA?
Novartis Innovative Technologies 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.
← 71889-0350-08 · Kapa'ihi, 71894-0121-03 · Novartis →
For educational and professional reference only — not medical advice. Pricing reflects published NADAC and CMS ASP (free public data) and may differ from your acquisition cost; always verify before billing or dispensing.