Getting Started

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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
Required for all patients who are prescribed ZOLGENSMA, this form serves as the official prescription for ZOLGENSMA. It’s divided into 5 sections: Patient Information, Insurance Information, Prescriber and Institution Information, Laboratory Test Documentation, and Prescription Information.

ZOLGENSMA Prescription and Patient Consent FormsZOLGENSMA Prescription and Patient Consent Forms

Laboratory Tests
Certain laboratory tests may be necessary to determine if your patient is right for ZOLGENSMA. Please download the form for more information about laboratory tests performed prior to treatment.
Laboratory TestsLaboratory Tests
Caregiver Resources

Facts about ZOLGENSMA

Facts about ZOLGENSMA
This informational sheet about ZOLGENSMA is designed for Caregivers and explains what ZOLGENSMA is, how it is given, and what support is available to their family.
Facts about ZOLGENSMAFacts about ZOLGENSMA

Steps to Treatment

Steps to Treatment
This guide will take Caregivers through the steps to treatment, including tests that may be needed, the support their family will receive, and continued care after infusion.
Steps to TreatmentSteps to Treatment
Dosing Information

ZOLGENSMA Treatment Guide

ZOLGENSMA Treatment Guide
The 5 steps to a one-time-only ZOLGENSMA infusion are outlined in this guide. Steps include baseline testing, storage and handling, premedication, preparation and infusion, and postmedication and monitoring.ZOLGENSMA Treatment GuideZOLGENSMA Treatment Guide

Pharmacy Quick Reference Guide

Pharmacy Quick Reference Guide
Designed with the pharmacy in mind, this guide provides a quick summary of how ZOLGENSMA is supplied, stored, and dosed.
Pharmacy Quick Reference GuidePharmacy Quick Reference Guide


Dosing and Infusion Video

Walk through the 5 steps of ZOLGENSMA infusion. See how to properly prepare, dose, and infuse ZOLGENSMA—including corticosteroid treatments and monitoring patients after infusion.

ZOLGENSMA Prescribing Information

Full Prescribing Information

Full Prescribing Information
Download the Full Prescribing Information to review the efficacy, safety, and dosing of ZOLGENSMA.
Full Prescribing InformationFull Prescribing Information
AveXis Patient Support

The OneGene Program

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 InformationISI Tray Icon

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.

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

Thrombocytopenia
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.

Elevated Troponin-I
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.

ADVERSE REACTIONS

The most commonly observed adverse reactions (incidence ≥5%) in clinical studies were elevated aminotransferases and vomiting.

Indication

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.