Download ZOLGENSMA Prescription Form ›
Download Dosing and Infusion Guide ›
Your resource for understanding the ZOLGENSMA treatment journey
The materials below are here to help you and your care team provide the best experience for your patients throughout the ZOLGENSMA treatment journey:
Screening, Diagnosis, and Prescribing
ZOLGENSMA Prescription and Patient Consent Forms
Facts about ZOLGENSMA
Steps to Treatment
Pharmacy Quick Reference Guide
ZOLGENSMA Prescribing Information
Full Prescribing Information
AveXis Patient Support
The OneGene Program™, offered by AveXis, is a comprehensive, individualized support program for families with pediatric patients less than 2 years of age with SMA and their healthcare providers throughout the ZOLGENSMA treatment journey.
OneGene Program support includes:
- Answers to questions about ZOLGENSMA
- A dedicated, personalized support team focused on the need of each family
- Verification of insurance benefits
- Coordination of financial assistance programs for eligible patients
Contact the OneGene Program at 1-855-441-GENE (4363) Monday-Friday (8 AM to 8 PM ET) to learn more.
Important Safety Information
BOXED WARNING: Acute Serious Liver Injury
Acute serious liver injury and elevated aminotransferases can occur with ZOLGENSMA. Patients with pre-existing liver impairment may be at higher risk. Prior to infusion, assess liver function of all patients by clinical examination and laboratory testing (e.g., hepatic aminotransferases [aspartate aminotransferase (AST) and alanine aminotransferase (ALT)], total bilirubin, and prothrombin time). Administer a systemic corticosteroid to all patients before and after ZOLGENSMA infusion. Continue to monitor liver function for at least 3 months after infusion.
WARNINGS AND PRECAUTIONS
Transient decreases in platelet counts, some of which met the criteria for thrombocytopenia, were observed at different time points after ZOLGENSMA infusion. Monitor platelet counts before ZOLGENSMA infusion and on a regular basis for at least 3 months afterwards.
Transient increases in cardiac troponin-I levels were observed following ZOLGENSMA infusion. Monitor troponin-I before ZOLGENSMA infusion and on a regular basis for at least 3 months afterwards.
The most commonly observed adverse reactions (incidence ≥5%) in clinical studies were elevated aminotransferases and vomiting.
ZOLGENSMA is an adeno-associated virus 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 or the use in patients with advanced SMA (e.g., complete paralysis of limbs, permanent ventilator dependence) has not been evaluated with ZOLGENSMA.
Please click here for Full Prescribing Information.