Posted by Michael Wonder on 17 Dec 2018
PRIME designation granted by European Medicines Agency for Roche’s risdiplam for treatment of spinal muscular atrophy
17 December 2018 - Risdiplam has the potential to be the first oral medicine for the treatment of SMA.
Roche today announced that the EMA has granted PRIME (PRIority MEdicines) designation for the company’s investigational oral medicine risdiplam (RG7916) for the treatment of people with SMA.
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.
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.
