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Roche's neuromuscular disease drug gets EU priority review

Roche Holding AG said the European Medicines Agency granted priority medicines designation to risdiplam for treating spinal muscular atrophy, a neuromuscular disease.

Spinal muscular atrophy, or SMA, is a rare, genetic disease affecting the area of the nervous system that controls voluntary muscle movement. The EMA grants priority medicines designation for therapies that improve on the effect of current treatments or medicines that benefit patients without treatment options.

The designation is based on data from the Firefish and Sunfish clinical studies. The Firefish study evaluated risdiplam in infants with type 1 SMA, while Sunfish studied the therapy in children and adults with type 2 and 3 SMA.

The types of SMA are based on the age of onset and highest physical milestones reached. Type 1 is the most severe and most common form of the disease and is usually diagnosed within an infant's first months, while types 2 and 3 are usually diagnosed after 6 months and 18 months, respectively.

Switzerland's Roche is developing risdiplam in collaboration with South Plainfield, N.J.-based PTC Therapeutics Inc. and the Spinal Muscular Atrophy Foundation.

Risdiplam, which is orally administered, is expected to compete with Biogen Inc.'s SMA drug Spinraza, which is injected directly into the spine.