Ehlers-Danlos Syndrome (EDS) is a group of hereditary disorders of connective tissue. vEDS is the most severe subtype where patients suffer from life-threatening arterial dissections and ruptures, as well as intestinal and uterine ruptures. The median age of death in the U.S. is 51 years.1 An Acer-commissioned patient-finder study phenotypically identified 4,169 vEDS patients in the U.S. from an analysis of a commercially available patient claims database with data of approximately 190 million unique patient lives. Based on that information, Acer estimates the prevalence of phenotypically-defined vEDS in the U.S. could be greater than 1 in 45,000. Currently, there are no FDA-approved therapies for vEDS.
We are developing EDSIVO™, a new chemical entity (NCE), for the treatment of COL3A1+ vEDS patients. An EDSIVO™ NDA was originally submitted based on data obtained from the BBEST trial1 and accepted for filing in October 2018 with priority review. Following FDA review, we received a Complete Response Letter (CRL) stating 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 subsequently appealed the FDA decision. While FDA denied the appeal, it described possible paths forward for Acer to explore. Following our Type B meeting with FDA in the second quarter of 2021, we decided to conduct a clinical trial for EDSIVO™. More information on our planned pivotal clinical trial can be found here.
There can be no assurance that the resulting data from the trial would be adequate to support approval of an NDA, although having a SPA agreement in place would indicate concurrence by FDA with the adequacy and acceptability of specific critical elements of overall protocol design (e.g., conduct, entry criteria, dose selection, endpoints, and planned analyses) for a study intended to support a future marketing application.
EDSIVO™ is an investigational drug and is not currently FDA approved for any indication. There can be no assurance that the funding to conduct and complete the clinical trial will be obtained, that the clinical trial will be successful, or that the resubmission of the original New Drug Application will be approved.
- Ong KT, et al. Effect of celiprolol on prevention of cardiovascular events in vascular Ehlers-Danlos syndrome: a prospective randomised, open, blinded-endpoints trial. Lancet. 2010;376(9751):1476-1484