Roche Evrysdi gets CHMP recommendation for babies under two months old with spinal muscular atrophy
Evrysdi is currently approved in the EU for the treatment of patients aged two months or older,
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Basel: Roche has announced that the EU Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the extension of the Evrysdi (risdiplam) European Union (EU) marketing authorisation, which would include infants with genetically confirmed diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies, including from birth to below two months. The recommendation is based on an interim analysis from the ongoing RAINBOWFISH trial in pre-symptomatic babies with Type 1 SMA from birth to six weeks. In SMA, early treatment is critical to counteract ongoing and irreversible loss of motor neurons.3,4,5 A final decision regarding the approval is expected from the European Commission later this year.
“Treating babies with SMA early helps them to carry out daily activities such as sitting, standing, and walking,” said Levi Garraway, M.D., Ph. D., Chief Medical Officer and Head of Global Product Development. “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.”
The CHMP decision is based on the RAINBOWFISH interim analysis (n=18), which included 6 babies with 2 or 3 copies of the SMN2 gene who completed at least one year of study assessments. Of these, 100% (6/6) were able to sit after one year of treatment with Evrysdi, 67% (4/6) could stand and 50% (3/6) could walk independently. All infants were alive at 12 months without permanent ventilation.
The RAINBOWFISH data show that the safety profile of Evrysdi in pre-symptomatic babies is consistent with the safety profile seen in previous trials with symptomatic SMA patients.
Evrysdi is currently approved in the EU for the treatment of patients aged two months or older.
"Roche is currently investigating Evrysdi in combination with an anti-myostatin molecule targeting muscle growth in the Ph II/III trial MANATEE for the treatment of SMA." the company stated.
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