More than 2 months after the FDA approved an expensive new gene therapy drug for a rare disease in children, the agency said it has major questions about the accuracy of some of the testing data.
More than two months after the Food and Drug Administration approved an expensive new gene therapy drug for a rare disease in children, the agency said it has major questions about the accuracy of some of the testing data. The FDA said Tuesday that the drug maker — AveXis, which is owned by Novartis — was aware of problems with the data and didn’t inform the agency until after the drug was approved.in May to treat spinal muscular atrophy in children 2 and under.
Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said Tuesday in a statement that AveXis manipulated some of the early data from testing in animals. That data was submitted as part of the drug’s application for approval. “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,” Marks said. “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.”
The FDA stopped short of pulling approval for the drug, saying that the agency “remains confident that Zolgensma should remain on the market,” and that all testing done in humans showed that the drug was safe and effective. The agency hasn't ruled out possible criminal or civil action against AveXis, according to the FDA statement.
“The idea that Novartis would go for approval of the most expensive drug in the history of the world and do it with data it knew to be inaccurate is an outrage and it could put patients in jeopardy,” David Mitchell, a cancer patient and founder of the advocacy group Patients For Affordable Drugs said via email.
Singapore Latest News, Singapore Headlines
Similar News:You can also read news stories similar to this one that we have collected from other news sources.
FDA says some data testing Novartis' $2 million gene therapy was manipulatedThe U.S. Food and Drug Administration said on Tuesday that some data from early ...
Read more »
U.S. FDA says some data testing Novartis' $2 million gene therapy was manipulatedThe U.S. Food and Drug Administration said on Tuesday that some data from early ...
Read more »
Jeff Bezos sells Amazon stock worth $2.8BAmazon boss Jeff Bezos offloads $990M worth of shares, taking the total value of shares sold last week to $2.8B
Read more »
Meghan Markle's Old Los Angeles Home for Sale for Nearly $2 MillionMeghan Markle's old house in L.A. is up for grabs.
Read more »
North Korean cryptocurrency heists net estimated $2 billion: UN reportNorth Korea has earned as much as $2 billion dollars through large-scale cyber attacks to help fund its weapons programs, a United Nations panel alleges in a new report.
Read more »
North Korea took $2 billion in cyber attacks to fund weapons program: U.N. reportNorth Korea has generated an estimated $2 billion for its weapons of mass destru...
Read more »