NEW YORK – The European Medicines Agency's Committee for Medicinal Products for Human Use on Friday said that marketing authorization for Genentech's Evrysdi (risdiplam) should be extended to newborns and babies younger than 2 months old who have a genetically confirmed diagnosis of spinal muscular atrophy or one to four copies of the survival motor neuron (SMN) 2 gene.
CHMP issued the positive opinion after reviewing interim data from the RAINBOWFISH trial in which the majority of pre-symptomatic babies on Evrysdi could stand or walk within a year of treatment, the typical time frame for such activities for healthy babies.
"Treating babies with spinal muscular atrophy early helps them to carry out daily activities such as sitting, standing, and walking," Levi Garraway, Genentech's chief medical officer and head of global product development, said in a statement. "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 spinal muscular atrophy."
Spinal muscular atrophy is a progressive neuromuscular disease that affects 1 in 10,000 babies and can be fatal. It is caused by mutations in the SMN1 gene, and the age of disease onset and its severity can be impacted by how many copies of the SMN2 gene a child has. Abnormalities in these genes can cause a deficiency in the SMN protein, which is critical for normal functioning of motor neurons. Evrysdi, an SMN2 splicing modifier, is designed to treat spinal muscular atrophy caused by these genetic abnormalities.
Evrysdi is currently approved in the EU for spinal muscular atrophy patients who are at least 2 months old. If the European Commission approves the expanded indication for the drug based on CHMP's recommendation, Evrysdi will become available for patients starting at birth.
Genentech, the pharmaceutical division of Roche, expects the EC to make a decision about this expanded indication later this year. The firm is also studying Evrysdi with an anti-myostatin molecule targeting muscle growth in the Phase II/III MANATEE trial in spinal muscular atrophy patients.