Medicine Compliance Aid Stability
Lactation Safety Information
EdsivoVascular Ehlers-Danlos syndrome (vEDS), associated with collagen mutation
Development and Regulatory status
Dec 18: No information on Acer website to indicate plans for licensing celiprolol for vEDS in Europe. Corporate presentation indicates company is focussed on US launch. If approved, Edsivo will be the only FDA-approved therapy to treat vEDS patients .
Dec 18: Has orphan drug status in US .
Dec 18: FDA grants priority review with a Prescription Drug User Fee Act (PDUFA) target action date of 25 Jun 19 .
Dec 18: FDA has accepted for review EDSIVO™ for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation .
Dec 16: Acer Therapeutics announces the signing of an agreement with the Greater Paris University Hospital, granting the company exclusive rights to access and use data from a randomized controlled clinical study of celiprolol. Acer Therapeutics will use this pivotal clinical data to support a New Drug Application (NDA) regulatory filing for its lead product, VASEBRA™ (celiprolol), for the treatment of vascular Ehlers-Danlos Syndrome (vEDS) .
Trial or other data
Dec 18: Phase IV study, which has been published in the Lancet in 2010, was stopped early for treatment benefit. The primary endpoints were reached by five (20%) in the celiprolol group and by 14 (50%) controls (hazard ratio [HR] 0·36; 95% CI 0·15-0·88; p=0·040). Adverse events were severe fatigue in one patient after starting 100 mg celiprolol and mild fatigue in two patients related to dose uptitration .
Apr 11: PIV study (NCT00190411) recruited 54 patients aged 15 to 65 years old in France and Belgium. Patients were randomised to either celiprolol (50 to 400 mg BID) or no treatment. Patients who are not randomized enter a longitudinal survey of events. Primary outcome is rate of major cardiovascular events in the treated group during a 5 years follow-up. The hypothesis is a 50% reduction in the occurrence of cardiovascular events under treatment, assessed by a blinded, independent events committee .
2010: Prospective randomized open trial completed in 2010 (NCT00190411) .