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FDA Grants Evrysdi® Priority Review Based on Results From Treating Pre-Symptomatic Infants with Spinal Muscular Atrophy
- RAINBOWFISH study interim results showed that after treatment with Evrysdi pre-symptomatic infants with spinal muscular atrophy achieved same motor
About this update from Ptc Therapeutics, Inc.
[{"type":"text","content":"- RAINBOWFISH study interim results showed that after treatment with Evrysdi pre-symptomatic infants with spinal muscular atrophy achieved same motor milestones as healthy infants -\n\n\nSOUTH PLAINFIELD, N.J., Jan. 25, 2022 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today 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.\n\n \n \n \n \n \n \n\n \n\"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.\"\nIncluded in the sNDA submission was interim data from the RAINBOWFISH study, which showed 80 percent of pre-symptomatic infants with SMA treated with Evrysdi for at least 12 months achieved motor milestones such as sitting without support, rolling, crawling, standing unaided, and walking independently.\nEvrysdi is designed to treat SMA by increasing and sustaining the production of the SMN protein, which is found throughout the body and is critical for maintaining healthy motor neurons and movement. Evrysdi was based on PTC's splicing platform. Evrysdi is marketed by Roche and in the United States by Genentech, a member of the Roche Group. Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.\nAbout Spinal Muscular Atrophy (SMA)Spinal muscular atrophy (SMA) is a severe, progressive neuromuscular disease that can be fatal. It affects approximately 1 in 10,000 babies and when untreated is the leading genetic cause of infant mortality. SMA is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of SMN protein. This protein is found throughout the body and is essential to the function o...