Risdiplam (Evrysdi®) treatment allows presymptomatic babies with spinal muscular atrophy (SMA) to maintain their ability to swallow, according to new data from the RAINBOWFISH study presented at the World Muscle Society 2021 Virtual Congress.
“These new data for Evrysdi may help extend the potential benefits of this medicine to the youngest SMA patients,” said Levi Garraway, MD, PhD, the chief medical officer and head of global product development at Roche, in a press release.
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RAINBOWFISH is an ongoing open-label, single-arm, multicenter phase 2 clinical trial that aims to investigate the safety, efficacy, pharmacokinetics, and pharmacodynamics of risdiplam in asymptomatic infants with genetically diagnosed SMA who are aged 0 to 6 weeks. All participants receive oral risdiplam once a day for 2 years. This is followed by an open-label extension phase of at least 3 years and a follow-up. The total treatment duration is at least 5 years.
The primary endpoint is the percentage of participants who are sitting without support for at least 5 seconds, according to the study. Secondary outcome measures include the percentage of participants developing SMA that’s clinically manifested, the time to permanent ventilation and/or death, and the percentage of participants who are alive without permanent ventilation. They also include the percentage of participants who achieve the attainment level of the motor milestones at 12 and 24 months including head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing, and walking, and the percentage of participants who can swallow and feed orally.
Four out of 5 patients treated with risdiplam for at least 1 year achieved standing and walking independently, according to the data presented at the conference. Moreover, all 5 babies maintained the ability to swallow and could exclusively feed by mouth after 1 year of treatment.
The trial is still recruiting participants at sites across the world and is expected to be completed on January 21, 2029.
Risdiplam is a splicing modifier of the SMN2 gene, which is designed to increase the production of functional SMN protein from this gene. It has already been approved by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of adults and children aged 2 months and older with SMA.
Roche presents new data at World Muscle Society (WMS) 2021 highlighting new advances for people living with rare neuromuscular disorders. News release. Roche; September 24, 2021.
A study of risdiplam in infants with genetically diagnosed and presymptomatic spinal muscular atrophy (Rainbowfish). US National Library of Medicine. Last updated September 10, 2021. Accessed October 1, 2021.