On Thursday, the European Commission granted marketing authorization under exceptional circumstances and maintained orphan designation for Biogen Inc’s (NASDAQ:BIIB) Qalsody (tofersen) for amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 gene (SOD1-ALS). Qalsody is the first treatment approved in the European Union to target a genetic cause of ALS, also known as motor neuron disease (MND). SOD1-ALS is an ultra-rare genetic form of ALS estimated to affect less than 1,000 people in Europe. Qalsody is Biogen’s third rare disease therapy to be approved in the EU. Related: ...