Rare Daily Staff

Novartis CEO Vasant Narasimhan sought to assure analysts and address questions about the company’s recently approved gene therapy Zolgensma for the rare neuromuscular disease spinal muscular atrophy following a statement from the FDA that the company’s application for marketing approval included manipulated data.

The FDA said AveXis, which Novartis acquired in 2018 for $8.7 billion, notified the agency at the end of June about the issue following the approval of Zolgensma, its gene therapy to correct the underlying cause of SMA. The FDA said it is currently assessing the situation, but that it “remains confident” Zolgensma should remain on the market.

The agency said it is only concerned with a small portion of the product testing data that was contained in the marketing application. This data was used by the manufacturer to support the development of its production process for the product. Nevertheless, it said that AveXis became aware of the data manipulation that created inaccuracies in its application prior to the approval of Zolgensma and that it failed to inform the agency until after the product won approval. The FDA said it will use its full authority to act, which may include civil or criminal penalties.

“Ensuring truthful, complete and accurate data in product applications is a critical component of industry’s responsibility as they work to demonstrate the safety, purity, and potency of biological products,” Peter Marks, director of FDA’s Center for Biologics Evaluation and Research, said in a public statement. “The submission of such truthful, complete and accurate data is also critical for the FDA to be able to protect the public health, and the law requires it.”

On the call with analysts, Novartis’ Narasimhan explained the delay as a result of the company wanting to complete an internal investigation to determine what happened prior to notifying the agency. He said he’s committed to rebuilding trust.

“I would like to emphasize we did not delay any of the above due to the timing of the Zolgensma BLA,” said Narasimhan on his call with analysts. “We moved quickly to conduct the relevant investigations and to be science-based and patient driven. In our approach, we determined that the potential data issue in question impacted a very small amount of data and did not impact the safety, efficacy or product quality of Zolgensma.”

He said the company is reaching out to key opinion leaders and the patient advocacy community to address concerns they may have and to help ensure they have the information they need to communicate with patients.

When one analyst asked whether this represented a systemic cultural issue at AveXis, Narasimhan said he did not believe so. “I think this was an isolated incident of certain individuals,” he said.

SMA is a rare genetic disease caused by a mutation in the SMN1 gene, which encodes the protein critical for the maintenance and function of specialized nerve cells called motor neurons. If there is not enough functional SMN protein, then the motor neurons die, leading to debilitating and often fatal muscle weakness. Infantile-onset SMA is the most severe and most common subtype of SMA. Children with this condition have problems holding their head up, swallowing and breathing. These symptoms may be present at birth or may present by the age of six months.

Photo: Vasant Narasimhan CEO of Novartis

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