y for thesecond and third month until troponin-I level returns to baseline.(2.3, 5.3)
ADVERSE REACTIONS
The most common adverse reactions (incidence ≥ 5%) were elevatedaminotransferases and vomiting. (6)
To report SUSPECTED ADVERSE REACTIONS, contact AveXis at 1833-828-3947 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
USE IN SPECIFIC POPULATIONS
Pediatric use: Use of ZOLGENSMA in premature neonates before reachingfull term gestational age is not recommended because concomitant treatmentwith corticosteroids may adversely affect neurological development. DelayZOLGENSMA infusion until full-term gestational age is reached. (8.4)
See 17 for PATIENT COUNSELING INFORMATION
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FULL PRESCRIBING INFORMATION: CONTENTS*WARNING: ACUTE SERIOUS LIVER INJURY
1 INDICATIONS AND USAGE
2 DOSAGE AND ADMINISTRATION
2.1 Dose and Administration
2.2 Preparation
2.3 Laboratory Testing and Monitoring to Assess Safety
3 DOSAGE FORMS AND STRENGTHS
4 CONTRAINDICATIONS
5 WARNINGS AND PRECAUTIONS
5.1 Acute Serious Liver Injury and Elevated Aminotransferases
5.2 Thrombocytopenia
5.3 Elevated Troponin-I
6 ADVERSE REACTIONS
6.1 Clinical Trials Experience
6.2 Immunogenicity
7 DRUG INTERACTIONS
8 USE IN SPECIFIC POPULATIONS
8.1 Pregnancy
8.2 Lactation
8.4 Pediatric Use
8.6 Hepatic Impairment
11 DESCRIPTION
12 CLINICAL PHARMACOLOGY
12.1 Mechanism of Action
12.2 Pharmacodynamics
12.3 Pharmacokinetics
13 NONCLINICAL TOXICOLOGY
13.1 Carcinogenesis, Mutagenesis, Impairment of Fertility
13.2 Animal Toxicology and/or Pharmacology
14 CLINICAL STUDIES
16 HOW SUPPLIED/STORAGE AND HANDLING
16.1 How Supplied
16.2 Storage and Handling
17 PATIENT COUNSELING INFORMATION
*Sections or subsections omitted from the full prescribing informationare not listed.
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FULL PRESCRIBING INFORMATION
WARNING: ACUTE SERIOUS LIVER INJURY
• Acute serious liver injury and elevated aminotransferases can occur with
ZOLGENSMA. (5.1)
• Patients with pre-existing liver impairment may be at higher risk. (8.6)
• Prior to infusion, assess liver function of all patients by clinical examination andlaboratory testing (e.g., hepatic aminotransferases [aspartate aminotransferase
(AST) and alanine aminotransferase (ALT)], total bilirubin, and prothrombin time).
Administer systemic corticosteroid to all patients before and after ZOLGENSMAinfusion. Continue to monitor liver function for at least 3 months after infusion(2.1) (2.3).
1 INDICATIONS AND USAGE
ZOLGENSMA (onasemnogene abeparvovec-xioi) is an adeno-associated virus vector-basedgene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinalmuscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.
Limitation of Use
• The safety and effectiveness of repeat administration of ZOLGENSMA have not been
eva luated [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 eva luated [see Clinical
Studies (14)].
2 DOSAGE AND ADMINISTRATION
For single-dose intravenous infusion only.
2.1 D
