AveXis Receives FDA Approval of Zolgensma, a Gene Therapy, for Spinal Muscular Atrophy for Patients Under Two Years of Age

AveXis, Inc., a Novartis company, today announced that they have received FDA approval for Zolgensma (formerly AVXS-101), a gene therapy that replaces the survival motor neuron 1 (SMN1) gene, which is missing or mutated in individuals with SMA.

This first approval is for intravenous (IV) delivery of gene therapy. The FDA has approved Zolgensma for IV delivery in patients with all forms and types of SMA who are under two years of age at the time of dosing.

In addition, AveXis is currently testing intrathecal (IT) delivery of Zolgensma in clinical trials. IT delivery would allow older patients to receive the therapy. Once these clinical trials are complete, AveXis will determine whether the data supports a separate BLA filing for IT delivery.

“This is an important day for the SMA community. Clinical data shows that a one-time infusion of Zolgensma can change the course of SMA,” said Kenneth Hobby, President of Cure SMA. “We look forward to working with our partners from AveXis and Novartis and with healthcare professionals across the US, to ensure rapid access to Zolgensma according to this FDA approval. We also look forward to the additional data coming soon from ongoing clinical trials which could broaden the approval to also cover older SMA patients.”

Cure SMA and AveXis to Co-Host Webinar

Cure SMA and AveXis will host a joint webinar on the launch of Zolgensma. This webinar will include information about the approval, dosing sites, future IT clinical trials, and more. The webinar will also include a Q&A session.

The date and time of the webinar will be announced shortly.

Cure SMA Supports Multiple Gene Therapy Approaches

Beginning in 2010, Cure SMA made a series of grants to Nationwide Children’s Hospital to study gene therapy, also called gene transfer. Spinal muscular atrophy (SMA) is caused by a mutation in the survival motor neuron 1 gene (SMN1). Because of this mutation, the individual does not produce enough survival motor neuron (SMN) protein.

Gene transfer may increase SMN levels by using a virus, called a vector, to deliver the SMN1 gene to affected cells. Adeno-associated virus serotype 9 (AAV9) has the unique ability to cross the blood brain barrier and the Blood-Cerebrospinal Fluid Barrier (CSF).

Gene therapy can be delivered in two ways: an injection into a vein, known as IV delivery, and injection directly into the CSF, a process known as IT delivery. IV delivery is now FDA-approved for patients with all forms and types of SMA who are under two years of age at the time of dosing.

Clinical trials testing IT delivery are ongoing. IT delivery of gene therapy has shown promise for reducing the amount of drug required for older patients. This could eventually make the treatment accessible to a wider population.

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