CHMP favors extending Evrysdi to newborns with SMA in the EU
Final European Commission decision on oral risdiplam likely this year
The Committee for Medicinal Products for Human Use (CHMP) favors European Union (EU) approval of Evrysdi (risdiplam) being extended to cover babies with spinal muscular atrophy (SMA) younger than 2 months of age.
The recommendation now will be reviewed by the European Commission, which is expected to issue a final decision later this year. The commission isn’t required to abide by a CHMP opinion, but it usually does.
“This CHMP recommendation is an important step towards treating babies from birth with an oral formulation, and is a testament to Evrysdi’s impact on preserving precious muscle function and improving the daily lives of people with SMA,” Levi Garraway, MD, PhD, chief medical officer and head of global product development at Roche, which markets Evrysdi through its subsidiary Genentech, said in a company press release.
RAINBOWFISH trial supporting use of risdiplam in newborns with SMA
CHMP specifically recommended that Evrysdi be approved for babies with genetically confirmed SMA types 1, 2, or 3, who have one to four copies of the SMN2 gene. This would effectively cover all babies with SMA who might benefit from the treatment, since people with type 4 disease and five or more copies generally don’t develop symptoms until adulthood. Evrysdi currently is approved in the EU to treat SMA patients starting at 2 months old.
“Treating babies with SMA early helps them to carry out daily activities such as sitting, standing, and walking,” Garraway said.
SMA is caused by mutations in the gene SMN1, which provides instructions for making a protein called survival motor neuron (SMN). The “backup” gene SMN2 also contains instructions for making SMN protein, but due to a quirk in the gene’s sequence, it cannot make nearly as much SMN protein as SMN1. Evrysdi works by modulating the activity of SMN2 to allow more SMN protein to be produced from this backup gene.
CHMP’s recommendation was based on data from the Phase 2 RAINBOWFISH clinical trial (NCT03779334), an ongoing study that’s evaluating Evrysdi in young children with SMA who start on treatment as newborns. Interim study data have shown that, after a year on the therapy, these babies were reaching motor milestones like being able to crawl, stand, and walk.
The safety profile of Evrysdi in these babies so far has been consistent with findings in older patients. The most commonly reported safety issues in RAINBOWFISH to date include cough, vomiting, constipation, diarrhea, fever, rash, and infections in the lungs and/or nose.
Based on data from RAINBOWFISH, Evrysdi was approved in the U.S. for babies with SMA younger than 2 months old last year.