Raptor granted FDA orphan drug designation for cysteamine

Cysteamine is currently approved by the FDA and European Medicines Agency to treat nephropathic cystinosis, a rare lysosomal storage disease.

Preclinical results suggest that cysteamine has neuroprotective effects that could potentially help treat Huntington’s disease (HD). Raptor’s clinical development subsidiary, Bennu Pharmaceuticals, plans to evaluate cysteamine in patients with HD.

Christopher Starr, CEO of Raptor, said: “We are delighted that the FDA has granted orphan drug designation for cysteamine in HD. Our team at Raptor is well-versed in developing and gaining regulatory approval of new therapies for orphan drug disorders. We hope to contribute some of this orphan drug expertise and know-how to facilitate Bennu’s clinical program for HD.”