Zolgensma Recommended for European Commission Conditional Approval

• By Laurie Watanabe
• Mar 31, 2020

Zolgensma, the one-time gene therapy to treat spinal muscular atrophy (SMA), has been recommended for conditional approval by the European Commission (EC).

The approval would apply to treating infants and children with Type 1 SMA or SMA Type 2 with up to three copies of the SMN2 gene.

A March 27 news announcement from AveXis, Zolgensma’s manufacturer, said, “The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending conditional marketing authorization of Zolgensma (onasemnogene abeparvovec) for the treatment of patients with 5q spinal muscular atrophy with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1; or for patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene.”

Zolgensma is administered to each patient just once, via intravenous infusion. It was approved in May 2019 by the U.S. Food & Drug Administration for children with SMA who are younger than 2.

SMA is the leading genetic cause of death worldwide for infants.