The U.S. Food and Drug Administration Grants Orphan Drug Designation to ACER-002 for the Potential Treatment of Vascular Ehlers-Danlos Syndrome
In January 2015, Acer Therapeutics Inc., a pharmaceutical company developing therapies for serious rare diseases with significant unmet medical need, was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) to celiprolol hydrochloride (ACER-002), a new chemical entity (NCE), for the treatment of vascular Ehlers-Danlos Syndrome (vEDS). The disease, also known as Ehlers-Danlos Syndrome type IV, is a rare, autosomal dominant, connective tissue disorder with no FDA-approved therapy for the disease.
In people with vEDS, mutations in the COL3A1 gene for type III procollagen lead to a deficit of collagen synthesis. This rare and lethal form of Ehlers-Danlos Syndrome (EDS) is associated with ruptured blood vessels, aneurysms, dissections, fistulas and spontaneous ruptures of the uterus and gastrointestinal structures. The median age of survival for people with vEDS is 51 years. There are approximately 2,000 people in the U.S. diagnosed with vEDS, though experts estimate as many as 5,000 patients may be affected.1
Orphan drug designation is provided to drugs and biologics that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases and disorders that affect fewer than 200,000 people in the U.S.
- Pepin MG, et al. Survival is affected by mutation type and molecular mechanism in vascular Ehlers–Danlos syndrome (EDS type IV) Genet Med. 16: 881-888.