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Zolgensma: FDA: Drugmaker knew about 'manipulated data' before approval of the $2.1 million drug
Source: USA Today
FDA: Drugmaker knew about 'manipulated data' before approval of the $2.1 million drug
Ken Alltucker, USA TODAY Published 6:10 p.m. ET Aug. 6, 2019
Novartis knew about "data manipulation" in early testing of its groundbreaking gene therapy drug but did not tell the Food and Drug Administration until after the $2.1 million therapy hit the market, the agency said Tuesday.
The drugmaker knew about Zolgensma's inaccurate data on March 14 but did not tell the FDA until June 28, more than one month after the agency approved the drug, the agency said.
The FDA recommends the gene therapy, used to treat spinal muscular atrophy, remain on the market as the agency continues to review data, according to Peter Marks, director of the agency's Center for Biologics Evaluation and Research.
But officials warned that AveXis, the gene therapy subsidiary of Swiss giant Novartis, could face penalties.
-snip-
Ken Alltucker, USA TODAY Published 6:10 p.m. ET Aug. 6, 2019
Novartis knew about "data manipulation" in early testing of its groundbreaking gene therapy drug but did not tell the Food and Drug Administration until after the $2.1 million therapy hit the market, the agency said Tuesday.
The drugmaker knew about Zolgensma's inaccurate data on March 14 but did not tell the FDA until June 28, more than one month after the agency approved the drug, the agency said.
The FDA recommends the gene therapy, used to treat spinal muscular atrophy, remain on the market as the agency continues to review data, according to Peter Marks, director of the agency's Center for Biologics Evaluation and Research.
But officials warned that AveXis, the gene therapy subsidiary of Swiss giant Novartis, could face penalties.
-snip-
Read more: https://www.usatoday.com/story/news/health/2019/08/06/novartis-used-faulty-data-worlds-most-expensive-drug-zolgensma/1934856001/
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Source: U.S. Food & Drug Administration
Statement on data accuracy issues with recently approved gene therapy
For Immediate Release: August 06, 2019
Statement From: Director - Center for Biologics Evaluation and Research (CBER)
Dr. Peter Marks M.D. PhD.
As a public health agency, we believe that it is critical to facilitate the development of innovative safe and effective medical products, like the cellular and gene therapy products that have shown enormous potential to treat previously untreatable diseases. As part of the approval process for biological products, the agency reviews extensive information submitted by manufacturers, including details of how the product is made in a reproducible manner, how it has been tested in animals, and how it has been investigated in human clinical trials to demonstrate its safety and efficacy.
It is the manufacturer’s responsibility to submit complete and accurate information in marketing applications for evaluation by the FDA. If we become aware of a concern with data submitted to the agency as part of our review of a product application, it is in the best interest of patients, their caregivers, and the public that we disclose such information, to the extent permitted by law.
On May 24, the FDA approved Zolgensma, a gene therapy product intended to treat children less than two years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 gene — the most severe form of SMA. SMA is a leading genetic cause of infant mortality. Subsequently, on June 28, following the FDA’s approval of the product, the agency was informed by AveXis Inc., the product’s manufacturer, about a data manipulation issue that impacts the accuracy of certain data from product testing performed in animals submitted in the biologics license application (BLA) and reviewed by the FDA.
The FDA is carefully assessing this situation and remains confident that Zolgensma should remain on the market. Out of the large amount of submitted information reviewed by the agency, our concerns at this time are limited to only a small portion of the product testing data that was contained in the marketing application. This product testing data was used by the manufacturer to support the development of its production process for the product. These data do not change the agency’s positive assessment of the information from the human clinical trials that were conducted as part of the development program. The totality of the evidence demonstrating the product’s effectiveness and its safety profile continues to provide compelling evidence supporting an overall favorable benefit-risk profile. However, the integrity of the product testing data used in the development of the product’s manufacturing process is still a matter that we are continuing to evaluate and take very seriously.
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. 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. We are carefully assessing the issue of the manipulation of the product testing data used in the production process and are conducting a thorough assessment of the information from a recently completed inspection. In part, this will allow us to determine the implications for the FDA’s scientific review of the information in the BLA and will allow us to amend our publicly posted BLA reviews, as appropriate.
-snip-
For Immediate Release: August 06, 2019
Statement From: Director - Center for Biologics Evaluation and Research (CBER)
Dr. Peter Marks M.D. PhD.
As a public health agency, we believe that it is critical to facilitate the development of innovative safe and effective medical products, like the cellular and gene therapy products that have shown enormous potential to treat previously untreatable diseases. As part of the approval process for biological products, the agency reviews extensive information submitted by manufacturers, including details of how the product is made in a reproducible manner, how it has been tested in animals, and how it has been investigated in human clinical trials to demonstrate its safety and efficacy.
It is the manufacturer’s responsibility to submit complete and accurate information in marketing applications for evaluation by the FDA. If we become aware of a concern with data submitted to the agency as part of our review of a product application, it is in the best interest of patients, their caregivers, and the public that we disclose such information, to the extent permitted by law.
On May 24, the FDA approved Zolgensma, a gene therapy product intended to treat children less than two years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 gene — the most severe form of SMA. SMA is a leading genetic cause of infant mortality. Subsequently, on June 28, following the FDA’s approval of the product, the agency was informed by AveXis Inc., the product’s manufacturer, about a data manipulation issue that impacts the accuracy of certain data from product testing performed in animals submitted in the biologics license application (BLA) and reviewed by the FDA.
The FDA is carefully assessing this situation and remains confident that Zolgensma should remain on the market. Out of the large amount of submitted information reviewed by the agency, our concerns at this time are limited to only a small portion of the product testing data that was contained in the marketing application. This product testing data was used by the manufacturer to support the development of its production process for the product. These data do not change the agency’s positive assessment of the information from the human clinical trials that were conducted as part of the development program. The totality of the evidence demonstrating the product’s effectiveness and its safety profile continues to provide compelling evidence supporting an overall favorable benefit-risk profile. However, the integrity of the product testing data used in the development of the product’s manufacturing process is still a matter that we are continuing to evaluate and take very seriously.
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. 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. We are carefully assessing the issue of the manipulation of the product testing data used in the production process and are conducting a thorough assessment of the information from a recently completed inspection. In part, this will allow us to determine the implications for the FDA’s scientific review of the information in the BLA and will allow us to amend our publicly posted BLA reviews, as appropriate.
-snip-
Read more: https://www.fda.gov/news-events/press-announcements/statement-data-accuracy-issues-recently-approved-gene-therapy