Biogen, Ionis Get FDA Approval For SMA Drug

(RTTNews) - Biogen Inc. (BIIB) and Ionis Pharmaceuticals Inc. (IONS) said the U.S. Food and Drug Administration has approved Spinraza to treat spinal muscular atrophy or SMA in pediatric and adult patients.

Spinraza, or nusinersen, is the first FDA-approved medicine for SMA. The FDA approval under priority review was received within three months of regulatory filing.

SMA is a leading genetic cause of death in infants and toddlers that is marked by progressive, debilitating muscle weakness. It is characterized by loss of motor neurons in the spinal cord and lower brain stem, resulting in severe and progressive muscular atrophy and weakness.

Spinraza was discovered and developed by Ionis Pharmaceuticals and Biogen, and is licensed to Biogen, who is responsible for future development, manufacturing and commercialization of the drug.

Biogen and Ionis conducted a clinical development program that moved Spinraza from its first dose in humans in 2011 to its first regulatory approval in five years.

Based on the FDA's approval of Spinraza, Ionis will receive a $60 million milestone payment and also receive $90 million in additional milestone payments based on regulatory approvals in Europe and Japan. Further, Ionis is eligible to receive tiered royalties on sales of Spinraza up to a percentage in the mid-teens.

Biogen noted that the overall findings of the studies support the effectiveness of Spinraza across the range of SMA patients, and appear to support the early initiation of treatment.

Biogen plans to make Spinraza available for shipment in the U.S. to healthcare providers in approximately one week. The company expects possible variation in time to treatment as institutions and treatment centers learn about the drug.