Repligen's RG2833 granted orphan drug designation for treatment of Friedreich's ataxia

Repligen Corporation (Nasdaq: RGEN) announced today that the Office of Orphan Products Development of the Food and Drug Administration (FDA) has granted orphan drug designation to RG2833, a selective histone deacetylase 3 (HDAC-3) inhibitor for the treatment of Friedreich's ataxia.  Orphan drug designation qualifies Repligen to receive seven years of marketing exclusivity in the United States if the company is the first to obtain marketing approval for RG2833 for the treatment of Friedreich's ataxia.  This designation may also qualify Repligen to benefit from certain tax credits and a waiver of the company's obligation to pay the FDA application user fees for this product as required by the Prescription Drug User Fee Act. The U.S. Orphan Drug Act provides incentives for companies developing and marketing therapies for rare diseases, defined as those affecting fewer than 200,000 Americans.  There are approximately 15,000 people worldwide with Friedreich's ataxia.

"We are very pleased to receive Orphan Drug Designation for our Friedreich's ataxia program," stated Walter C. Herlihy, President and Chief Executive Officer of Repligen.  "RG2833 is the first compound that targets activation of the defective gene responsible for Friedreich's ataxia.  If this approach is successful, it has the potential to significantly impact patients' lives."

Earlier this month, we filed an Investigational New Drug Application with the FDA for a Phase 1 study of RG2833 to evaluate the pharmacokinetic and safety profile of RG2833 in up to 40 healthy volunteers.  This study will also evaluate the pharmacodynamic response of various biomarkers in peripheral blood to RG2833.  RG2833 is a new chemical entity, which is the subject of a composition of matter patent application, which if allowed, will remain in force until 2029 prior to any patent term extensions.  RG2833 has been developed in collaboration with scientists from The Scripps Research Institute and a broad network of international scientific thought leaders.  Repligen's research efforts have been partially funded with grants from the Muscular Dystrophy Association, the Friedreich's Ataxia Research Alliance, GoFAR and the National Ataxia Foundation.  We are also evaluating HDAC-3 inhibitors in animal models of Huntington's disease and cognition.