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Genentech: FDA Grants Priority Review For Risdiplam - Quick Facts

Genentech, a member of the Roche Group (RHHBY.PK), announced the U.S. FDA has accepted the New Drug Application and granted priority review for risdiplam, an investigational survival motor neuron-2 splicing modifier for spinal muscular atrophy. Previously, the FDA granted Orphan Drug Designation for risdiplam in 2017, followed by Fast Track Designation. The final FDA decision is expected by May 24, 2020.

If approved, risdiplam would be the first at-home administered medicine for people living with spinal muscular atrophy. Genentech leads the clinical development of risdiplam as part of a collaboration with the SMA Foundation and PTC Therapeutics, Inc. (PTCT).

The FDA's acceptance of the NDA triggers a $15 million milestone payment to PTC by Roche.

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