The US Food and Drug Administration (FDA) has issued a response to ARCA biopharma for its new drug application (NDA) seeking approval for Gencaro, declaring that the new chronic heart failure treatment cannot be approved in its current form.
The FDA said the company needs to conduct an additional clinical efficacy trial of Gencaro in patients with heart failure.
The company has also been instructed to conduct additional clinical pharmacology studies to address drug interaction and pharmacokinetic issues, and to conduct additional non-clinical studies to further characterise Gencaro metabolites.
The FDA has recommended that the best clinical study, which serves as the pivotal Phase III trial for the application, does not adequately demonstrate efficacy of Gencaro in reducing all-cause mortality in patients with heart failure.
In addition, the FDA letter raises concerns over the statistical significance of some of the pharmacogenetic data relied upon by the company to assert that individual patient response to Gencaro may be predicted by genotype.
ARCA founder and chief science and medical officer Michael Bristow said the company is reviewing the letter and plans to discuss it with the FDA as soon as possible.
“We appreciate the FDA’s review of the NDA and will continue to work with the FDA to resolve the issues raised in the letter,” Bristow said.
ARCA has declared that a comparative effectiveness analysis has demonstrated that Gencaro is as effective in reducing all-cause mortality as other ß-blocker therapies, when comparable patient populations are analysed.