Zolgensma Manufacturer: FDA Approval Is “Just the Beginning” of SMA Treatment

• By Laurie Watanabe
• May 29, 2019

Last week’s U.S. Food & Drug Administration approval of Zolgensma, a drug that treats young children who have spinal muscular atrophy (SMA), is “just the beginning,” according to the drug’s manufacturer.

Zolgensma, manufactured by AveXis, a Novartis company, made mainstream news headlines because of the drug’s high cost: $2.125 million per dose.

Nevertheless, news of FDA approval was celebrated by the SMA community, which has been raising funds for years to support research aimed at creating a treatment for SMA.

In a May 24 bulletin to stakeholders, AveXis said Zolgensma has been approved for children under age 2 who have SMA. It’s a one-time, intravenous dose that takes about 60 minutes to administer.

AveXis also recommends that children taking Zolgensma receive an oral corticosteroid the day before getting the treatment and continue taking corticosteroids for at least 30 days afterward “to help manage elevated liver enzyme levels.”

Children who take Zolgensma should have liver enzyme levels monitored via blood tests for at least three months after receiving the drug, AveXis said. Physicians may gradually lower corticosteroid dosages based on blood test results.

AveXis said there are five ongoing Zolgensma clinical studies ongoing, with two more studies planned. As of May 2019, the company said 130 children with SMA have taken Zolgensma.

Due to the drug’s high cost, AveXis said that when a physician prescribes Zolgensma, “highly trained people from the OneGene Program, a dedicated resource from AveXis, can work closely with the patient’s insurance company to help obtain coverage.” The OneGene Program is also available to answer questions and provide guidance for families “during treatment and beyond.”

Cure SMA and AveXis are cohosting a June 12 Webinar on Zolgensma. To register, click here.