FDA grants orphan drug designation for Friedreich’s ataxia treatment

WALTHAM, Mass. The Food and Drug Administration has granted special designation to encourage the development of a drug made by Repligen for treating a rare degenerative disease, Repligen said Monday.

Repligen said the FDA’s Office of Orphan Products Development granted orphan drug designation to the drug RG2833, a treatment for Friedreich’s ataxia. The FDA grants orphan drug designation to treatments for rare diseases and grants incentives such as a seven-year market exclusivity period, compared with the usual five years granted to new drugs.

Friedreich’s ataxia is a genetic illness affecting around 15,000 people worldwide that results from a gene defect causing insufficient production of the protein frataxin, leading to degeneration of nerves controlling muscle movements in the arms and legs and nerve tissue in the spinal cord. Symptoms of the disease typically emerge between the ages of 5 and 15 years and lead to severe disability, incapacitation and loss of life in early adulthood.

“We are very pleased to receive orphan drug designation for our Friedreich’s ataxia program,” Repligen CEO Walter Herlihy said. “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.”