MHLW Panel Backs Orphan Status for SMA Gene Therapy, CAR-Ts

August 30, 2018
A key health ministry panel on August 29 supported orphan drug designation for four products including Novartis Pharma’s gene therapy, AVXS-101, for the treatment of spinal muscular atrophy (SMA), and two CAR-T therapies for types of lymphoma. Novartis acquired AVXS-101...read more