Edsivo filed with FDA for vascular Ehlers-Danlos syndrome.- Acer Therapeutics.

Acer Therapeutics announced that the FDA has accepted for review Acer�s New Drug Application (NDA) for Edsivo (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation. The FDA also granted a priority review of the NDA and assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 25, 2019. The application is based on a randomized controlled clinical study of celiprolol. Edsivo received FDA Orphan Drug Designation for the potential treatment of vEDS in 2015.

The previously completed European study, published on October 30, 2010, in The Lancet, was stopped early having achieved statistical significance in its primary endpoints, with arterial dissection or rupture affecting 5 (20%) celiprolol patients and 14 (50%) subjects in the non-treated control group. The combined primary and secondary endpoints of intestinal or uterine rupture affected 6 (24%) celiprolol patients and 17 (61%) subjects in the non-treated control group. The study was conducted in 53 patients, who were randomly assigned either a twice daily treatment of celiprolol or no treatment. Mean duration of follow-up was 47 months prior to trial halt.