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FDA Grants Evrysdi® Priority Review Based on Results From Treating Pre-Symptomatic Infants With Spinal Muscular Atrophy
PTC Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted priority review of a supplemental new drug application (sNDA) for Evrysdi® (risdiplam) to expand the indication to include pre-symptomatic infants under 2 months old with spinal muscular atrophy (SMA). If approved, Evrysdi would be the first medicine administered at-home for pre-symptomatic babies with SMA.
The results demonstrating that almost all of the pre-symptomatic infants achieved motor milestones comparable to healthy infants is tremendous,” said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. “The granting of the Priority Review for Evrysdi recognizes this and the significant need to treat babies with SMA as early as possible. We are proud that such a transformative treatment for patients living with SMA came from our splicing platform.
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