EU fast tracks Roche's risdiplam, rival to Spinraza

17 December 2018

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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