News

Sanfilippo B therapy JR-446 gets orphan drug status in Europe

The European Commission has granted orphan drug status to JR-446, a brain-penetrating enzyme replacement therapy (ERT) for Sanfilippo syndrome type B. The designation, announced in a press release from developer JCR Pharmaceuticals, is aimed at accelerating the clinical development and review of investigational therapies that have the…

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…

UX111 offers cognitive, motor skill gains in Sanfilippo syndrome type A

Ultragenyx Pharmaceutical’s experimental gene therapy UX111 slows or halts the brain damage that affects communication and cognitive and motor skills in children with Sanfilippo syndrome type A, according to new trial data. While younger children receiving the one-time therapy saw gains in these functional domains compared with untreated age-matched…