IONS - Europe Approves Biogen's Tofersen For Adult Patients With Rare Type Of Neurodegenerative Disorder | Benzinga
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.
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