Acer Therapeutics announced it has received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding its New Drug Application (NDA) for Edsivo (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS).
The CRL states that it will be necessary to conduct an “adequate and well-controlled trial to determine whether celiprolol reduces the risk of clinical events in patients with vEDS”. Acer plans to respond to the FDA in the third quarter of this year to discuss the FDA’s decision.
The NDA was based on data from an analysis of a 2010 European study involving 53 patients. The small group size, however, raised questions among the review panel about the adequacy of the trial results.
In April long-term data from a cohort of COL3A1-positive vEDS patients was published in the Journal of the American College of Cardiology (JACC). That included up to 17 years of safety data in the population with survival curve analysis showing that patients who did not receive celiprolol had much worse outcomes than those receiving the drug. They also cited a relative decrease in hospitalization rates for acute arterial events during that period, when a majority of patients were on celiprolol.
About Ehlers-Danlos syndrome
EDS is a group of hereditary diseases of the connective tissue. vEDS is the most severe subtype. Patients with vEDS suffer from life-threatening arterial dissections and ruptures, in addition to intestinal and uterine ruptures. The average mortality is 51 years of age. A study commissioned by the company identified 4,169 vEDS patients in the U.S. from a database of about 190 million unique patients.
About Edsivo (celiprolol)
Celiprolol is a β-blocker (β1-andrenoceptor antagonist with partial β2 agonist activity) originally developed to treat hypertension. Celiprolol is believed to provide clinical benefit for people with vEDS by promoting normal collagen synthesis in the blood vessels, and by shifting the pressure load away from the vessels most prone to dissection and rupture.