The U.S. Food and Drug Administration (FDA) stated on Tuesday that some data from early testing of Novartis’ over $2 million gene therapy Zolgensma was manipulated; however, the company believes the treatment ought to remain available on the market.
The FDA mentioned it’s carefully assessing the situation.
The manipulated information was used to illustrate comparability between an early model of Zolgensma and the later model of the therapy, which was manufactured using a unique procedure.
The FDA doesn’t believe that the manipulation impacts the safety or testing surrounding the model of the drug, which cures spinal muscular atrophy, the leading genetic reason for death in infants.
Novartis’ Avexis unit informed the FDA of the information manipulation on June 28, the regulator stated.
Novartis acquired the remedy’s maker in 2018, and the drugmaker was aware of the manipulation as early as March – over two months before the therapy’s approval, the FDA stated.
The regulator plans to take action against the corporate along with possible civil or legal penalties.
In a press release on Tuesday, Novartis mentioned it’s “fully assured” in the safety, high quality and efficacy of Zolgensma.
The difficulty will not be expected to impact the timing of its ongoing Zolgensma administrative filings and development plans, the drugmaker added.
The disease typically results in paralysis, respiratory problem, and death within months for infants born with the most severe Type I type. SMA affects one in every 10,000 live births, with 50% to 70% having Type I condition.
The FDA mentioned its concerns had been currently limited to a small portion of product testing information included in the advertising and marketing application for the remedy.