Questioning both the integrity of its late-stage data as well as its ability to demonstrate efficacy, the FDA has rejected ARCA Biopharma's heart failure drug Gencaro. The agency is demanding more clinical studies on the drug's efficacy as well as a review of pharmacokinetic issues before it will approve the therapy. And in short order investors trounced the developer's stock, shearing away 58 percent of its value.
Quoting the FDA's full response letter, Broomfield, CO-based ARCA said the agency raised concerns about the integrity of its clinical data after reviewing audits of certain sites. An independent audit of more sites as well as other remedies may be required to assure the FDA of the quality of the data. And ARCA said that their Phase III trial did "not adequately demonstrate efficacy of Gencaro in reducing all-cause mortality in patients with heart failure."
ARCA also noted, though, that the FDA had acknowledged that it had not reviewed several substantial amendments to its application before issuing the letter. And those amendments, it adds, could reduce the additional work it needs to do.
"The company is reviewing the complete response letter and plans to discuss it with FDA as soon as possible," said Michael R. Bristow, founder and chief science and medical officer ARCA.
- read the ARCA release
- check out the report from Reuters