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.
Reference:
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.
The U.S. Food and Drug Administration Grants Orphan Drug Designation to ACER-001 for the Potential Treatment of Maple Syrup Urine Disease
In August 2014, 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 sodium phenylbutyrate (ACER-001) for the treatment of Maple Syrup Urine Disease (MSUD). Approximately 1,000 patients suffer from MSUD in the U.S. and 3,000 patients are affected worldwide. There is no FDA-approved therapy for MSUD, and the condition is sub-optimally managed via a BCAA-restricted diet alone. Despite this diet, patients still experience poor neurological outcomes and social impairment.
Orphan drug designation, which qualifies Acer for various development incentives under the Orphan Drug Act, 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.
Acer Therapeutics Licenses Exclusive Worldwide Rights from Baylor College of Medicine for Development of Sodium Phenylbutyrate for Maple Syrup Urine Disease
In April 2014, Acer Therapeutics Inc. entered into an exclusive worldwide license agreement with Baylor College of Medicine to develop and commercialize formulations and prodrugs of sodium phenylbutyrate for the treatment of Maple Syrup Urine Disease (MSUD). MSUD is a rare, devastating genetic disease which prevents the proper metabolism of three essential, branched-chain amino acids (BCAAs) – leucine, isoleucine and valine. There is no FDA-approved therapy for MSUD, and the condition is sub-optimally managed via a BCAA-restricted diet alone. Despite this diet, patients still experience poor neurological outcomes and social impairment.
“The research data generated by the team at Baylor College of Medicine supports phenylbutyrate, when used in conjunction with a BCAA-restricted diet, as a promising therapy to lowering levels of BCAAs – and in particular leucine – for patients affected by MSUD,” said Chris Schelling, COO and Founder of Acer Therapeutics. “We are looking forward to advancing our proprietary taste-masked, immediate-release formulation of sodium phenylbutyrate, ACER-001, through clinical studies and potentially bring the first therapeutic option to patients with this serious disease.”
