Rare Disease Drug Boom Bolstered By FDA Orphan Drug Grant Program

The FDA on Oct. 21 awarded 15 grants totaling more than $14 million to various research organizations — and one drugmaker — to spur development of new treatments for rare diseases, the latest signal the agency wants to prime the orphan drug pipeline. Boston-based start-up Alkeus Pharmaceuticals was the only drugmaker to receive a grant from the agency, getting $167,000 to fund a Phase I study of ALK001 to treat the genetic eye disorder known as Stargardt disease.
Washington Drug Letter