FDA lets off Novartis after gene therapy data row

Novartis is off the hook following the FDA’s review of data manipulation in the filing of its hugely expensive spinal muscular atrophy gene therapy Zolgensma.

To recap, Novartis was facing potential civil or criminal penalties because of manipulation of data used to support the filing of the $2.1 billion gene therapy against the muscle wasting disease.

Novartis has blamed executives from Avexis, the drug’s original developer, for the incident involving non-clinical data said to have occurred before it bought the biotech for $8.7 billion in 2018.

The executives were fired as the scandal blew up last year and the Swiss pharma went into fire-fighting mode to try and save its reputation.

A furious FDA had threatened to throw the book at Novartis but it now appears that the regulator has other more pressing matters such as the unfolding coronavirus crisis to deal with.

According to several press reports the FDA has decided that the corrective actions taken by Novartis are sufficient and no further action is to be taken.

Paul Richards, acting chief of CBER’s consumer affairs branch, told Regulatory Focus: “FDA has completed its review of the inspection, the evidence collected, and the firm’s responses and corrective actions to the inspectional observations, and the agency has classified the inspection as Voluntary Action Indicated (VAI).”

VAI classification means “objectional conditions were found and documented” but “do not meet the threshold for regulatory action,” according to Richards.

Richards added that the FDA “takes data very seriously and expects data submitted in support of a marketing application to be reliable and accurate.”

The FDA approved Zolgensma in May last year, a month before Novartis notified it about the data manipulation issues.

Last week the European Medicines Agency’s CHMP scientific committee adopted a positive opinion to Zolgensma, paving the way for a European marketing authorisation in the coming months.

The European Commission usually rubber stamps decisions from the CHMP within a few months.

Zolgensma is the world's most expensive drug, costing $2.1 million for a single shot that addresses the underlying defect that causes SMA.