Roche's spinal muscular atrophy drug Evrysdi gets EU nod for pre-symptomatic babies

29 Aug 2023
firstwordpharma.com
Drug ApprovalClinical StudyClinical Result
Roche said Tuesday that the European Commission expanded approval of Evrysdi (risdiplam) to include use in infants under two months old with spinal muscular atrophy (SMA). The SMN2 splicing modifier was first authorised in the EU in 2021 for patients two months of age and older.
"With this label extension, we can treat babies soon after birth with Evrysdi, allowing them the greatest chance to achieve the milestones of sitting, standing and walking, similar to healthy children," commented Levi Garraway, Roche's head of global product development. The Swiss drugmaker is leading the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC TherapeuticsPTC Therapeutics.
Specifically, Evrysdi's new indication in the EU is for use in patients with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies from birth to below two months. The approval is based on interim data from the RAINBOWFISH trial in pre-symptomatic babies from birth to six weeks with Type 1 SMA. Results showed that all six babies with 2 or 3 copies of the SMN2 gene who completed at least one year of study assessments were able to sit after one year of treatment with Evrysdi, 67% could stand and 50% could walk independently.
Evrysdi, which generated sales growth of 25% in the second quarter to CHF 342 million ($388 million), is also approved in the US in babies under two months old, having initially been cleared in 2020 for all types of SMA in adult and paediatric patients two months and older. Roche is currently investigating Evrysdi in combination with an anti-myostatin molecule targeting muscle growth in the Phase II/III trial MANATEE for the treatment of SMA.
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