PTC Therapeutics Inc. (NASDAQ: PTCT) has announced that the US FDA has given its supplemental new drug application for Evrysdi (risdiplam) priority review to expand the indication to incorporate pre-symptomatic infants below 2 months with spinal muscular dystrophy. Evrysdi will be the first at-home drug for pre-symptomatic infants with SMA if authorized.
Study findings showed that pre-symptomatic infants attained motor milestones
Study results demonstrated that all the pre-symptomatic babies attained motor milestones relative to healthy babies, which is a massive achievement.
CEO Stuart W. Peltz stated, “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.”
Interim findings from the RAINBOWFISH study were included in the sNDA submission, showing that 80% of pre-symptomatic babies with SMA that were given Evrysdi for around one year attained motor milestones like sitting without assistance, crawling, rolling, standing unassisted, and walking freely.
Evrysdi is a treatment for SMA that works by enhancing and maintaining the SMN protein production, which is found all over the body and is essential for retaining healthy motor neurons and mobility. The company’s splicing platform served as the foundation for Evrysdi. Roche and Genentech, a Roche Group company, are responsible for marketing Evrysdi in the United States. In addition, as part of the partnership with the SMA Foundation and PTC Therapeutics, Roche is leading Evrysdi’s clinical development.
Spinal Muscular atrophy affects one in 10,000 babies
Spinal muscular atrophy (SMA) is a fatal neuromuscular illness that progresses rapidly. It affects about 1 in 10,000 newborns, and it is the largest hereditary risk factor of infant mortality when left untreated. SMA is brought by a mutation in the survival motor neuron 1 (SMN1) gene, resulting in SMN protein shortage. This protein can be found all over the body and is required to function neurons that regulate muscles and movement. Notably, nerve cells cannot function properly without it, resulting in gradual muscular weakening over time.