U.S. regulators want to know why Novartis waited until after the Swiss drugmaker’s $2.1 million gene therapy was approved to disclose a problem with testing data.
The Food and Drug Administration said earlier this week that the manipulated data that involved testing was done with animals, not patients. The agency said they remain confident that the drug, called Zolgensma, should stay on the market despite this revelation. They are investigating the matter and may consider criminal or civil penalties if appropriate.
Approved in May for children under two, Zolgensma is the most expensive drug treatment ever. It’s a one-time therapy for spinal muscular atrophy, a rare inherited condition which destroys a baby’s muscle control.
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About 400 babies are born in the U.S. each year with spinal muscular atrophy. It is a top genetic cause of death in infants. Zolgensma works by supplying a healthy copy of the faulty gene that causes the condition.
According to a statement from the FDA, AveXis Inc. (the Novartis AG subsidiary that manufactures Zolgensma) informed the agency five weeks after approval about a “data manipulation issue.” They said this error resulted in inaccurate information about testing in animals. The company was aware of the problem before the FDA approval of Zolgensma. The FDA indicated that the erroneous data is a small subset of the testing information that they evaluated. According to an inspection note released by the agency, there were inconsistencies in some of its survival data results with mice.
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Ned Sharpless, acting FDA Commissioner, said in a tweet that “the agency will use its full authorities to take action.”
Novaritis said in a statement earlier this week that after AveXis became aware of alleged data manipulation in one animal testing procedure, they immediately began investigating. Once it had “interim conclusions,” it shared them with the FDA. The drugmaker added:
“The data in question were a small portion of our overall submission and are limited to an older process no longer in use.”1
Novartis did not explain why it failed to notify the FDA before its approval of Zolgensma. Novartis stated that the animal test in question isn’t used in producing the therapy for patients and that Zolgensma is safe and effective.
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FDA staff inspected the company’s Irvine, California, manufacturing plant in recent weeks, then issued a report finding several insufficiencies, including not sufficiently following quality control procedures.
Considering this is the most expensive drug treatment ever, and it is used to treat infants, it seems reasonable to demand that there is absolutely no tolerance for error in any stage of the drug’s development.
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