FDA Declines to Approve Acer Therapeutics' Treatment for Rare Genetic Disorder

By Reuters Staff

June 26, 2019

(Reuters) - Acer Therapeutics Inc said on Tuesday its treatment for a severe, rare genetic disorder that can cause blood vessels to fatally rupture was declined approval from the U.S. Food and Drug Administration.

The orphan drug, Edsivo (celiprolol), aims to treat vascular Ehlers-Danlos syndrome (vEDS) - the most severe type of a hereditary disorder of the connective tissue.

FDA, in its complete response letter, has asked Acer to conduct further trials to determine the efficiency of Edsivo in treating patients with vEDS, Acer said.

"We expect to respond to the FDA in the third quarter of this year," Acer Chief Executive Officer Chris Schelling said.

Edsivo's marketing application was based on data from an analysis of a 2010 European study involving 53 patients.

The small group size, however, raised questions among experts about the adequacy of the trial results.

Although, some analysts have been bullish on the drug's prospects, citing patient follow-up data recently published in a medical journal showed that those treated with the drug over several years lived longer.