ZOLGENSMA (Onasemnogene Abeparvovec-xioi) Injection

ZOLGENSMA (onasemnogene abeparvovec-xioi), from Novartis Gene Therapies, is a one-time 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 SMN1 gene.

ZOLGENSMA uses an AAV9 vector to deliver a functional copy of the human SMN gene to motor neuron cells, addressing the root genetic cause of SMA and helping to sustain motor neuron survival and function with a single infusion.

• Boxed warning: acute serious liver injury and elevated liver enzymes; liver function must be monitored before and for months after infusion, with corticosteroid prophylaxis as directed.
• Thrombotic microangiopathy has been reported, sometimes serious; monitor platelet counts and clinical signs.
• Requires anti-AAV9 antibody titer testing before treatment, as pre-existing antibodies may reduce efficacy or increase risk.
• Vaccinations should be adjusted/deferred per corticosteroid administration; discuss the full vaccination schedule with the treating physician.

What documents are required to import ZOLGENSMA to India?

ZOLGENSMA may be imported in the patient’s name only, by patients or government hospitals. The following documentation is required:

• A valid prescription from a registered pediatric neurologist
• Genetic test report confirming bi-allelic SMN1 mutations and SMA diagnosis
• Patient ID/birth proof confirming age under 2 years (issued by the Government of India)

How is the order confirmed?

The order is confirmed only after receipt and verification of a valid prescription and genetic test report, given the one-time, high-value nature of this therapy.

Is ZOLGENSMA available in India?

ZOLGENSMA is not commercially marketed in India. It can be legally accessed through the Named Patient Program (NPP).

Indian Pharma Network (IPN) can source ZOLGENSMA from its authorized distribution network and facilitate supply to patients, doctors, and hospitals under applicable legal provisions, including the specialized cold-chain logistics this gene therapy requires.

For ZOLGENSMA cost in India and availability, contact IPN at Phone: +91-9310090915 or email info@indianpharmanetwork.in.

Global Approval Status:

• United States: Approved by the FDA — May 2019.

What is ZOLGENSMA used for?

ZOLGENSMA is a one-time gene therapy for spinal muscular atrophy in children under 2 with bi-allelic SMN1 mutations.

Who manufactures ZOLGENSMA?

ZOLGENSMA is developed by Novartis Gene Therapies.

How is ZOLGENSMA administered?

As a single intravenous infusion, administered once in a hospital setting.

How much does ZOLGENSMA cost in India?

As a one-time gene therapy, cost is substantial — contact IPN with the patient’s genetic report for a detailed quote.