EMA Panel Backs Zolgensma Gene Therapy for SMA

Megan Brooks


March 27, 2020

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended granting conditional marketing authorization for onasemnogene abeparvovec (Zolgensma, AveXis Inc), a gene therapy for spinal muscular atrophy (SMA).

Zolgensma is indicated for patients with 5q SMA with a bi-allelic mutation in the survival motor neuron 1 (SMN1) gene and a clinical diagnosis of SMA type 1; or patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene.

SMA is a rare and often fatal genetic disease that causes muscle weakness and progressive loss of movement. Approximately 550 to 600 infants are born with SMA in Europe each year.

Zolgensma is an adeno-associated virus vector-based gene therapy that addresses the genetic root cause of SMA by replacing the defective or missing SMN1 gene to halt disease progression.

"A one-time intravenous administration of Zolgensma supplies a fully functioning copy of the human SMN1 gene enabling the body to produce enough SMN protein. This is expected to improve their muscle function, movement and survival of children with the disease," the EMA said in a statement.

The US Food and Drug Administration approved Zolgensma for SMA last year, as reported by Medscape Medical News.

The positive CHMP opinion for Zolgensma "marks a critical step closer...to bringing the only gene therapy for SMA to Europe, helping to address the devastating impact the disease has on patients and their families," Dave Lennon, PhD, president of AveXis, said in a news release.

The CHMP positive opinion is based on the completed phase 3 STR1VE-US and phase 1 START trials that demonstrated the efficacy and safety of a one-time intravenous infusion of Zolgensma in symptomatic SMA type 1 patients younger than age 6 months at dosing.

"Zolgensma demonstrated prolonged event-free survival; rapid motor function improvement, often within 1 month of dosing; and sustained milestone achievement, including the ability to sit without support, a milestone never achieved in untreated type 1 patients," the company said.

The EMA positive opinion is also based on interim results from the ongoing SPR1NT trial, a phase 3, open-label, single-arm study in presymptomatic patients (< 6 weeks at age of dosing). In this study, Zolgensma was associated with "rapid, age appropriate major milestone gain, reinforcing the critical importance of early intervention in SMA patients," the company said.

AveXis said it expects approval by the European Commission in about 2 months and will offer a "Day One" access program to EU governments and reimbursement agencies to enable immediate access.

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