However, committee members who did not support approval said they were troubled by the lack of evidence on durability of effect and the limited safety data.
Committee member Dr. Darren McGuire, of the University of Texas Southwestern Medical Center, said he was encouraged by the data but did not think they were "sufficiently persuasive for a single trial to bring approval, even factoring in the orphan nature of the disease.
"These are vulnerable patients and they have lots of disability, and they present clinical challenges that we all struggle with when we treat them. That vulnerability makes us want to rush new drugs to market," but care must be taken that "the drugs we’re rushing to market are going to do more good than harm." Dr. McGuire suggested that the sponsor, Chelsea Therapeutics International, conduct another randomized trial lasting 3 to 6 months.
"I think I can forgive almost an entire absence of safety data if I’m very convinced of the efficacy data," said committee member Dr. Mori Krantz, of Denver Health Medical Center, who abstained. "But I just felt with the 1-week duration for a chronic condition ... I was contorting myself and doing a backbend. I think this drug has promise, but at this point I was just uncomfortable without further data."
This coverage is provided courtesy of "The Pink Sheet." Elsevier Global Medical News and "The Pink Sheet" are published by Elsevier.