The European Commission (EC) has granted conditional approval to Zolgensma (onasemnogene abeparvovec) for treating patients with spinal muscular atrophy (SMA) and a clinical diagnosis of type 1 SMA, or for those with up to three copies of the SMN2 gene. The approval will cover infants…

The U.S. Food and Drug Administration has decided not to fine or otherwise sanction Novartis for manipulated data included in the application that led to Zolgensma‘s approval to treat spinal muscular atrophy (SMA) in children up to age 2. The FDA was not informed until roughly a month after it announced…

Young children given a single injection of Zolgensma into the spinal canal continue to show gains in motor function and safety, new interim data from the STRONG trial in spinal muscular atrophy (SMA) type 2 patients up to age 5 show. These findings from the open-label Phase 1/2 clinical…

The European Medicine Agency’s Committee for Medicinal Products for Human Use (CHMP), has recommended conditional approval of Zolgensma (onasemnogene abeparvovec) for the treatment of patients with spinal muscular atrophy (SMA) and a clinical diagnosis of type 1, or for those with SMA carrying up to three…

A single infusion of Zolgensma (onasemnogene abeparvovec-xioi) continues to lead to meaningful and sustained benefits in children with spinal muscular atrophy (SMA) treated in infancy, according to data from several clinical trials. Zolgensma, formerly known as AVXS-101, is a gene therapy originally developed by AveXis, now…

The Japanese Ministry of Health, Labour and Welfare approved Zolgensma (onasemnogene abeparvovec-xioi) for the treatment of spinal muscular atrophy (SMA) patients under the age of two, Novartis announced. The approval includes toddlers and newborns who are pre-symptomatic at diagnosis, meaning those who have not yet shown symptoms.

Just 27 days after Nicole Almeida gave birth to her son, Matteo, the baby received a one-time infusion of what one day would be known as Zolgensma. That made him the youngest person ever to receive the world’s most expensive medication. Since that day in August 2015,…

Novartis plans to give up to 100 doses annually of Zolgensma, its costly gene therapy for spinal muscular atrophy (SMA), free-of-charge to eligible young children outside the U.S. with this progressive, neuromuscular disease. These treatments come under a global managed access program (MAP) the company runs for people with…

The parents of two babies treated in July with Zolgensma, the first gene therapy approved for spinal muscular atrophy or any neuromuscular disease, are confident they made the right choice for their girls. Both families also said they would make the same decision today, with minor adjustments.

With about 100 spinal muscular atrophy (SMA) patients already treated with Zolgensma (onasemnogene abeparvovec-xioi) since the gene therapy’s May approval in the United States, and expected growth in newborn screening, the groundbreaking treatment that is also the world’s most expensive is off to a robust start, Novartis…