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Roche's Evrysdi approved by European Commission as first home treatment for spinal muscular atrophy

Christian Fernsby |
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.

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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.

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.

Roche is working closely with reimbursement and assessment bodies in European countries to enable broad and rapid access to patients in need. Evrysdi will be accessible to patients in Germany in the coming days and in France from early April through the cohort Temporary Authorization for Use. Reimbursement dossiers have been submitted in many countries in anticipation of the decision by the European Commission to minimise any delay in patient access.


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