Biogen & Ionis: SPINRAZA Meets Primary Endpoint In Phase 3 Study - Quick Facts

(RTTNews) - Biogen (BIIB) and Ionis Pharmaceuticals (IONS) announced that SPINRAZATM (nusinersen), an investigational treatment for spinal muscular atrophy, met the primary endpoint at the interim analysis of CHERISH, the Phase 3 study evaluating SPINRAZA in later-onset spinal muscular atrophy. The companies said, with the positive interim analysis, the CHERISH study will be stopped and participants will be able to transition into the SHINE open-label extension study to receive SPINRAZA.

The U.S. FDA recently accepted the company's New Drug Application for SPINRAZA and communicated they plan to act early on the NDA under an expedited review. The European Medicines Agency recently validated Biogen's Marketing Authorization Application in the EU. The EMA's Committee for Medicinal Products for Human Use granted Accelerated Assessment status and the FDA granted Priority Review to SPINRAZA. Biogen is preparing for the potential launch of SPINRAZA in the U.S. possibly as early as the end of 2016 or the first quarter of 2017.

SPINRAZA is an investigational, potentially disease-modifying therapy for the treatment of SMA that was discovered and developed by Ionis Pharmaceuticals. Biogen exercised its option to worldwide rights to SPINRAZA in August 2016.