Roche SMA treatment risdiplam granted PRIME designation by EMA

Roche announced that the European Medicines Agency has granted PRIME designation for the company’s investigational oral medicine risdiplam for the treatment of people with SMA. PRIME designation is granted by the EMA to support data generation and development plans for promising medicines, providing a pathway for accelerated evaluation by the agency, and thus potentially enabling them to reach patients earlier. Risdiplam, an orally administered, survival motor neuron-2 gene splicing modifier, has shown improvements in motor function in people with SMA Types 1, 2 and 3. An increasing body of clinical evidence suggests that SMA is a multisystem disorder, and the loss of SMN protein may affect many tissues and cells beyond the central nervous system. Risdiplam is systemically distributed and designed to durably increase SMN protein levels in the central nervous system and throughout the body.

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