Regeneron ($REGN) lost a bid to gain a key endorsement for using the biotech group's drug Arcalyst to combat gout flare-ups. An FDA advisory committee rejected the drug in an 11-0 vote against approval as experts harbored concerns about the lack of safety data and the limited time period of 16 weeks patients took the drug in studies.
The FDA will weigh the panel's advice in the agency's review of the drug, with a decision targeted by July 30, Reuters reported. Tarrytown, NY-based Regeneron is seeking approval of the injected therapy, which is already on the market for a rare genetic disorder, to treat a much larger population of patients during a period of time in which they are open to gout flare-ups. Yet members of the advisory committee were concerned that the drug could be used for longer periods, and there's a lack of data to show that the treatment is safe for prolonged usage.
Safety risks were highlighted in the FDA staff briefing documents on Regeneron's application last week, which noted that Arcalyst could boost the risk of infections and malignancies in patients while imparting only a modest benefit. And non-agency experts sounded equally unsatisfied with the body of evidence the company has provided to prove the safety and efficacy of the drug for gout patients.
"A lot of the patients where we envision using this drug are going to take it for longer than 16 weeks. It's hard for me to allow patients to be exposed [without longer safety data]," said panel member Dr. David Blumenthal, of Case Western Reserve University School of Medicine, as quoted by Reuters.
- check out the Reuters article
- see the PharmaTimes report
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