Sen. Ron Johnson, R-Wis., said he is opening an investigation into the FDA’s rejection of treatments for rare diseases, including ataluren, a drug used by some patients with Duchenne muscular dystrophy.
Mr. Johnson, who chairs the Senate Permanent Subcommittee on Investigations, announced the inquiry at a news conference March 11. He said the FDA should allow patients access to high-risk treatments with clear disclosures rather than remove those options altogether, according to a report from Spectrum News 1.
Ataluren’s manufacturer, PTC Therapeutics, withdrew its application in February after the FDA indicated it would be denied. Patients and families said the loss of access to the medication has led to worsening symptoms and reduced mobility.
Mr. Johnson said the investigation will begin by collecting complete response letters issued by the FDA to better understand the agency’s rationale for rejecting certain therapies.
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