SOUTH SAN FRANCISCO (dpa-AFX) - Swiss drug maker Roche Holding AG (RHHBY) announced Monday that the European Medicines Agency or EMA has granted PRIME (PRIority MEdicines) designation for the company's investigational oral medicine risdiplam (RG7916) for the treatment of people with Spinal Muscular Atrophy or 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 (SMN2) gene splicing modifier, has shown improvements in motor function in people with SMA Types 1, 2 and 3.
Roche leads the clinical development of risdiplam as part of a collaboration with the SMA Foundation and PTC Therapeutics.
PRIME designation for risdiplam is based on data from Part 1 of the pivotal studies FIREFISH (evaluating safety and determining dosage in infants with Type 1 SMA) and SUNFISH (in children and adults with Type 2 and 3 SMA) as well as a continuing medical need for alternative treatments and administration options for patients with SMA.
Risdiplam is currently being investigated in three global, multicentre clinical trials in all types of SMA.
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