Repligen Corporation has been granted orphan drug designation by the US Food and Drug Administration (FDA) for RG2833, a selective histone deacetylase 3 (HDAC-3) inhibitor to treat Friedreich’s ataxia.
The designation may provide seven years of marketing exclusivity to Repligen if it receives first marketing approval, qualifying Repligen for certain tax credits and a waiver of application fees payable to the FDA.
RG2833 is the first compound that targets activation of the defective gene that causes Friedreich’s ataxia, an inherited neurodegenerative disease.
The company filed an investigational new drug application with the FDA during early May to initiate a Phase I study of RG2833 to evaluate the pharmacokinetic and safety profile in 40 healthy subjects.
RG2833 has been developed by the Scripps Research Institute scientists and a network of international scientific experts.
The research activities of Repligen have been partially funded with grants from the Friedreich’s Ataxia Research Alliance, GoFAR, the Muscular Dystrophy Association and the National Ataxia Foundation.
An estimated 15,000 people suffer from Friedreich’s ataxia worldwide.