Federal officials say the pharmaceutical giant, Novartis, intentionally manipulated data collected during animal trials of Zolgensma, a new type of gene therapy for children, but has decided to let it stay on the market.
The U.S. Food and Drug Administration (FDA) released a statement this week indicating it will allow Zolgensma gene therapy products, intended to treat spinal muscular atrophy in young children, to remain on the market after new evidence indicates the manufacturer intentionally manipulated and withheld inaccurate data readings during its approval process.
According to the statement, Novartis only informed the FDA once the spinal muscular atrophy (SMA) gene therapy product was approved for market use.
Zolgensma is a proprietary gene therapy approved by the FDA in May of this year. It is designed to treat pediatric patients less than two years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.
The product is designed to address the genetic root cause of SMA by producing a copy of the human SMN gene to halt the progression of the disease through a one-time intravenous protein treatment.
Zolgensma is the first therapeutic treatment approved to treat the rare and fatal SMA disease. SMA is a severe neuromuscular disease that causes extreme muscle weakness and paralysis caused by a genetic defect in the SMN1 gene. It is diagnosed in approximately one out of every 10,000 live births and is the leading genetic cause of infant mortality. Generally, if left untreated in children up to 24 months, the disease will require the child to receive permanent ventilation support.
On June 28, AveXis and its parent company Novartis, disclosed to the FDA and other health authorities that some of the testing data collected during animal trials was inaccurate, and potentially manipulated before being submitted to the FDA during the approval process.
According to the FDA, AveXis was aware of the inaccurate data readings during the time of the approval process and allegedly manipulated the test results before submitting them to the FDA to avoid a delay in approving the treatment for commercial use.
AveXis stated that the data in question involves a small portion of the overall submission to the FDA and does not jeopardize the safety, efficacy or quality of Zolgensma use on humans.
FDA officials have announced Zolgensma will be permitted to remain on the market, indicating the importance of the treatment to provide potentially life-saving benefits for fragile children outweighs the risk.
However, officials have announced they are investigating and assessing the data manipulation during product testing to determine the implications for the FDA’s scientific review. At this time, the investigation is still ongoing and the agency stated a full evaluation of the events is required to see if further actions including civil and criminal penalties should be taken.
AveXis Biotechnology Company was acquired by Novartis in 2018. The SMA disease treatments are currently the most expensive medication in the world, costing over $2 million per treatment.
Although the FDA is continuing to allow Novartis and sub-company AveXis to remain marketing and selling Zolgensma, the company witnessed a 2.8% drop in stock market shares Tuesday, while competing biotech companies Biogen and Ionis pharmaceuticals, who make revival treatments for SMA seen stock market shares jump 2.1% and 3.3%