EU fast tracks Roche's risdiplam, rival to Spinraza
Roche has been granted European PRIME designation for the investigational oral medicine risdiplam (RG7916), for the treatment of people with SMA, a severe, inherited, progressive neuromuscular disease.
Like the US regulator’s Priority Review, the designation provides a pathway for accelerated evaluation by the agency. Roche is collaborating with the SMA Foundation and PTC Therapeutics to develop the therapy.
The decision is based on data from Part I of the pivotal FIREFISH and SUNFISH studies. Interim data from FIREFISH showed that infants with Type 1 SMA treated with risdiplam met developmental milestones, including sitting without support.
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