FDA Gives JR-171 Orphan Drug Status for Rare Genetic Disease

The FDA granted Orphan Drug status to JCR Pharmaceuticals’ JR-171, an investigational treatment for mucopolysaccharidosis type 1 (MPS I), an extremely rare genetic disease linked to the lack of an enzyme needed to break down sugar molecules.

JR-171 is a recombinant protein that is being evaluated in an ongoing phase 1/2 study in Japan that will also enroll volunteers in Brazil and U.S.

JCR estimates that MPS I, which causes a wide range of debilitating symptoms, affects approximately 3,600 individuals worldwide.