By Angela Maas

The FDA in August put out a statement addressing “data accuracy issues” with Zolgensma (onasemnogene abeparvovec-xioi), a new gene therapy to treat spinal muscular atrophy in people less than two years old who have bi-allelic mutations in the survival motor neuron 1 gene.

The FDA approved the drug from AveXis, Inc., which was acquired by Novartis AG last year, on May 24. On June 28, AveXis notified the FDA that there was “a data manipulation issue that impacts the accuracy of certain data from product testing performed in animals submitted in the biologics license application (BLA) and reviewed by the FDA.”

The FDA said Novartis learned about the issue March 14, but rather than informing the agency, which was assessing Zolgensma’s BLA at the time, the company conducted its own internal investigation, reporting its findings to the FDA after the investigation was completed. Still, the agency said that it “remains confident that Zolgensma should remain on the market.”

The one-time therapy is the most expensive drug in the world, with a price tag of $2.1 million.

In response, Novartis issued a press release Aug. 6 that read, in part, “we maintain that the totality of the evidence demonstrating the product’s effectiveness and its safety profile continue to provide compelling evidence supporting an overall favorable benefit-risk profile.”

Novartis recently said that Zolgensma has coverage plans from payers representing 40% of commercial lives, and high-profile news reports name Aetna Inc. and Anthem, Inc. among insurers expanding their policies to cover more patients. So will the current situation have any impact on pickup among payers and providers?

Based on the FDA’s statement, “I expect there will be no more than a minor and temporary blip in pickup of Zolgensma among providers and payers,” says Elan Rubinstein, Pharm.D., principal at EB Rubinstein Associates.