Andrea Lobo,  —

Sanfilippo B enzyme replacement therapy wins orphan drug status

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to JR-446, JCR Pharmaceuticals’ brain-penetrating experimental enzyme replacement therapy (ERT) for Sanfilippo syndrome type B, the company has announced. The designation is meant to accelerate the clinical development and review of investigational therapies with the potential…

Japan agency clears JR-441 trial for Sanfilippo syndrome type A

JCR Pharmaceuticals said it received permission from the Pharmaceuticals and Medical Devices Agency (PMDA), a regulatory agency in Japan, to start a Phase 1 clinical trial of JR-441, an experimental enzyme replacement therapy for Sanfilippo syndrome type A. The company didn’t provide details. “We are pleased that the…

JR-441 enzyme replacement therapy named orphan drug by FDA

An experimental enzyme replacement therapy (ERT), called JR-441, for Sanfilippo syndrome type A has been designated an orphan drug by the U.S. Food and Drug Administration, the treatment’s developer, JCR Pharmaceuticals, has announced. The designation is given to therapies being developed for conditions affecting fewer than 200,000 people…