Roche has announced that the US Food and Drug Administration (FDA) has approved a priority review of a supplemental new drug application (sNDA) for the use of Evrysdi in treating pre-symptomatic babies diagnosed with spinal muscular atrophy (SMA) under two months of age.
SMA is chronic, progressive neuromuscular disease, affecting an estimated one in 10,000 babies as a leading genetic cause of infant mortality. The disease is caused by a mutation of the survival motor neuron 1 (SMN1) gene which causes a deficiency of survival motor neuron (SMN) protein – an essential protein for a functional nervous system and muscle movement. Without SMN, nerve cells are dysfunctional and lead to increased muscle weakness over time. Depending on the patient’s form of SMA, physical strength, walking, eating or breathing can be impacted significantly or is lost altogether.
The FDA assessed the interim data submission from the RAINBOWFISH study, which demonstrated that the majority of pre-symptomatic babies who were administered Evrysdi achieved key milestones including sitting, walking, standing and a maintained ability to swallow having undergone 12 months of treatment.
Evrysdi (risdiplam) is a drug designed to treat SMA through an increased and sustained production of the SMN protein – a vital protein for maintaining functioning motor neurons and movement – found throughout the body found in the central nervous system and peripheral tissues.
Presently, the existing FDA label and recommendation for use of Evrysdi’s is for the treatment of SMA in adults, children and for babies aged two months and older. The drug is currently approved
in 70 countries and submitted in a further 31, with more than 4,500 patients treated so far.
“Treating very young babies with Evrysdi before SMA symptoms arise may help them to achieve milestones such as standing and walking within time frames typical of healthy infants,” commented Levi Garraway, Roche’s chief medical officer and head of global product development. “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.”
The initial interim data gathered from the RAINBOWFISH study – presented at the World Muscle Society (WMS) Virtual Congress 2021 – provided findings that demonstrated all of the babies (5/5) involved in the interim efficacy analysis maintained the ability to swallow.
The study also showed that all of the babies were able to feed exclusively orally following 12 months of treatment, while 80% treated with Evrysdi for at least 12 months met key milestones such as standing and walking independently according to guidelines sets by the World Health Organization (WHO) windows for healthy children.
Results from the latest RAINBOWFISH evaluation will be presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in March 2022.