European Commission extends Evrysdi marketing authorization in EU for infants under two years of age

Roche announces that the European Commission has approved its Expanded marketing authorization for Evrysdi (risdiplam) in the European Union (EU) to infants clinically diagnosed with spinal muscular atrophy (SMA) types 1, 2 or 3 or infants born with 1 to 4 copies of SMN2 Included for less than two months. The marketing authorization extension is supported by interim data from the ongoing RAINBOWFISH trial of presymptomatic infants with SMA Type 1 from birth to six weeks.

in words Nicole GossettPresident and Chief Executive Officer SMAEurope, “The SMA community welcomes the European Commission’s decision to extend the use of Evrysdi from birth”. “PPreserving motor neurons from the earliest possible age and preventing their irreversible loss can have a major impact on a person’s future motor and functional abilities. We look forward to continuing our collaboration to improve diagnosis, including newborn screening, and ensure access to medicines for all SMA patients. “

As far as he is concerned, Levi GallawayChief Medical Officer and Head of Global Product Development Roche,show”With this expanded indication, we can treat infants shortly after birth Together with Evrysdi, give them the best chance of reaching the sitting, standing and walking milestones, just like healthy children. “

positive data

European Commission approval based on interim analysis rainbow fish (n=18), including 6 infants with 2 or 3 copies of the SMN2 gene who completed at least one year of study assessment. in, One year after Evrysdi treatment, 100% (6/6) could sit, 67% (4/6) could stand, and 50% (3/6) could walk independently. All infants survived at 12 months without the need for permanent ventilation.

Therefore, these interim results reflect that the safety profile of Evrysdi in asymptomatic infants is consistent with that observed in previous trials in patients with symptomatic SMA. Among the adverse reactions, the most common ones are fever, diarrhea, rash, upper respiratory tract infection (including nasopharyngitis, rhinitis), lower respiratory tract infection (including pneumonia, bronchitis), constipation, vomiting and cough.

Evrysdi was initially approved in Europe in March 2021 for the treatment of patients two months and older. The approval is based on clinical trial data from the pivotal SUNFISH and FIREFISH studies.

In addition, Roche is currently investigating Evrysdi in combination with the muscle growth-targeting anti-myostatin molecule in the phase II/III MANATEE trial in SMA.

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