Roche announced that the European Commission (EC) has approved Evrysdi (risdiplam) for the treatment of 5q spinal muscular atrophy (SMA) in patients two months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies. SMA is a leading genetic cause of death in infants and 5q SMA is the most common form of the disease. The condition causes muscle weakness and progressive loss of movement and significant unmet need remains, particularly in adults living with this condition.

“Today’s approval of Evrysdi, the first and only SMA treatment with proven efficacy that can be taken at home, potentially transforms treatment options for a broad range of people with SMA living in the EU. By avoiding the need for in-hospital administration, Evrysdi can reduce the treatment burden on those living with SMA, their caregivers and healthcare systems. We thank the SMA community for their partnership, the trust they have placed in us and their unyielding commitment to achieve this significant milestone,” said Levi Garraway, Roche’s Chief Medical Officer and Head of Global Product Development.

The approval is based on data from two clinical studies, designed to represent a broad spectrum of people living with SMA: FIREFISH in symptomatic Type 1 infants aged 2 to 7 months and SUNFISH in symptomatic Type 2 and 3 children and adults aged 2 to 25 years. SUNFISH is the first and only placebo-controlled trial to include adults with Types 2 and 3 SMA. Evrysdi demonstrated a favourable efficacy and safety profile, with the safety profile established across both trials.

The decision from the European Commission follows a positive recommendation from the Committee for Medicinal Products for Human Use (CHMP) in February 2021. The review was completed under the accelerated assessment pathway for medicines, which is offered to medicines deemed to be of major interest for public health and therapeutic innovation. This approval is applicable to all 27 European Union member states, as well as Iceland, Norway, and Liechtenstein. Evrysdi was granted PRIME designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation in 2019. Maintenance of Orphan Drug Designation was recently confirmed by the Committee for Orphan Medicinal Products based on the assumption of Evrysdi’s significant benefit over existing treatments. Evrysdi has been approved in 38 countries and submitted in a further 33 countries.

Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.