Repligen Corporation 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.”