Another novel SMA option steps closer to approval in the USA

26 November 2019

Roche (ROG: SIX) subsidiary Genentech's New Drug Application for risdiplam has been accepted for Priority Review by the US regulator in spinal muscular atrophy (SMA).

Risdiplam is an investigational SMN-2 splicing modifier, which is designed to increase and sustain SMN protein levels in the body. A Food and Drug Administration decision on approval is due by May 24, 2020.

Roche has already been granted PRIME designation, the European equivalent, for the candidate.

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