When Novartis’ Zolgensma data manipulation problem first went public last August, an angry FDA said it would consider civil or criminal penalties. But it looks like neither of those is going to happen.
In a statement shared with FiercePharma, an FDA spokesperson said the agency has decided it will not punish Novartis’ gene therapy unit AveXis for doctoring Zolgensma mice testing data in its drug application package. The news was first reported by Biopharma Dive.
Specifically, the FDA deemed a Zolgensma-triggered inspection at an AveXis facility as “voluntary action indicated” despite the Form 483 enforcement letter issued last August. That means the agency figures the company has at least reached a minimally acceptable state of compliance and that no enforcement action will be taken.
The FDA reached that decision after reviewing “the information and records of the inspection, the evidence collected, and the firm’s responses as well as the corrective actions to the inspectional observations,” the spokesperson said.
“Based on its review of the information available, FDA continues to find Zolgensma to be safe and effective for its intended use,” the spokesperson added.
According to the FDA, AveXis told the agency about the data fabrication issue last June, and the agency conducted an inspection between July 24 and August 2, which ended with the Form 483. That document showed the initial allegation was raised within the company on March 14, 2019 but AveXis didn’t open a non-conformance report until May.
During the two-month gap, Novartis had confirmed the allegation internally but decided to investigate further before telling the FDA. That move effectively put off its report to the agency until after it greenlighted Zolgensma on May 24.
This delay apparently hit a nerve at the FDA, which at the time said it would “use its full authorities to take action, if appropriate, which may include civil or criminal penalties.”
But now, after assessing the information from the inspection and re-evaluating Zolgensma's biologics license application, the FDA has decided no regulatory action is necessary.
At least the agency was clear from the beginning that the concern was only limited to a small portion of animal testing data and that it didn’t affect human clinical trials. Novartis has also said the problematic assay was never used for the release of commercial product.
In its response to the Form 483, AveXis pinned the data falsification on two of its former top scientists, Brian and Allan Kaspar, and accused them of not cooperating with the internal investigation. The Kaspar brothers have denied any wrongdoing.
Facing increased criticism over its delays in alerting the FDA, the Swiss drugmaker pledged to inform the agency within five business days of any future data integrity issue if a drug filing is involved.
As a one-time gene therapy—versus chronic treatment—Zolgensma comes with the potential of a cure for spinal muscular atrophy. Its strong efficacy as shown in clinical studies has drawn great interest, helping Novartis collect $361 million in 2019 sales.
Since the FDA nod, Japanese regulators have approved Zolgensma as Novartis aims to wrap up reimbursement discussions in the first half of 2020. In Europe, the EMA’s Committee for Medicinal Products for Human Use has also endorsed its approval.