FDA grants priority review of the sNDA for spinal muscular atrophy treatment in pre-symptomatic babies under 2 months of age

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The US Food and Drug Administration (FDA) has granted priority evaluation of a supplementary new drug application (sNDA) for the use of Evrysdi (risdiplam) to treat pre-symptomatic newborns with spinal muscular atrophy under the age of two months (SMA). The sNDA filing includes interim data from the RAINBOWFISH trial, which reveals that after 12 months of treatment, the majority of pre-symptomatic newborns treated with Evrysdi accomplished important milestones like sitting, standing, walking, and maintaining their ability to swallow.

“Treating very young babies with Evrysdi before SMA symptoms arise may help them to achieve milestones such as standing and walking within timeframes typical of healthy infants. Extending treatment access for the youngest members of the SMA community is crucial and we look forward to working with the FDA on this application,”

said Levi Garraway, M.D., Ph. D., Roche’s Chief Medical Officer and Head of Global Product Development. 

Evrysdi is a drug that works to cure SMA by boosting and maintaining the production of the survival motor neuron (SMN) protein in the CNS and peripheral tissues. The SMN protein is distributed all over the body and is essential for the health of motor neurons and movement. The FDA has approved Evrysdi for the treatment of SMA in adults, children, and newborns aged two months and up. If authorized, Evrysdi would be the first drug for pre-symptomatic babies with SMA to be given at home.

After 12 months of treatment, all (5/5) of the newborns included in the intermediate efficacy analysis were able to feed exclusively orally, according to preliminary data from the RAINBOWFISH trial, which was presented at the World Muscle Society (WMS) Virtual Congress 2021. Eighty percent (4/5) of children treated with Evrysdi for at least 12 months were able to stand and walk independently within WHO guidelines for healthy children. After 12 months of Evrysdi therapy, all of the patients (n=5) achieved HINE-2* motor milestones in head control, sitting upright, rolling, and crawling.

During the interim safety analysis period (n=12), no treatment-related major adverse events were observed in any of the neonates treated with Evrysdi. Four treatment-emergent adverse events were recorded, all of which were resolved or were on their way to being addressed with continued Evrysdi treatment. Nasal congestion (33 percent), cough (25 percent), teething (25 percent), vomiting (25 percent), eczema (17 percent), abdominal discomfort (17 percent), diarrhea (17 percent), gastroenteritis (17 percent), a papule (17 percent), and pyrexia (17 percent) were the most prevalent adverse effects (AEs) (17 percent ). The AEs were caused by the babies’ age rather than the underlying SMA. RAINBOWFISH is now recruiting participants.

The latest results from the RAINBOWFISH study will be presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in March 2022.  As part of a collaboration with the SMA Foundation and PTC Therapeutics, Roche is leading the clinical development of Evrysdi.

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