Biogen Inc. received marketing authorization approval from the European Commission for Spinraza, a drug to treat 5q spinal muscular atrophy, a genetic neuromuscular disorder.
Spinal muscular atrophy is characterized by loss of motor neurons in the spinal cord and lower brain stem, resulting in severe and progressive muscular atrophy and weakness.
The drug is the first approved treatment in the EU for spinal muscular atrophy. It was reviewed under the European Medicines Agency's accelerated assessment program, intended to expedite access to patients with unmet medical needs.
Spinraza's approval is primarily based on data from two trials, both of which demonstrated the clinically meaningful efficacy and favorable benefit-risk profile of the drug.
The timing of the drug's availability in the EU will vary by country, per local reimbursement and access pathways.
Biogen has also submitted regulatory filings in Japan, Canada, Australia, Switzerland and Brazil and plans to initiate additional filings in other countries in 2017.