Roche’s Evrysdi approved by European Commission as first and only at home treatment for spinal muscular atrophy - Seite 2

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 today’s decision by the European Commission to minimise any delay in patient access.

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.

About FIREFISH
In FIREFISH, 29% (12/41; p<0.0001 compared to natural history) of infants treated with Evrysdi for 12 months were able to sit without support for at least five seconds, as assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition. This is a key motor milestone never achieved in the natural history of Type 1 SMA. In addition, 93% of infants were alive and 85% were event-free (alive with no permanent ventilation). Furthermore, 5% (2/41) of infants were able to stand with support, as measured by the Hammersmith Infant Neurological Examination, and 83% were able to feed orally. Ninety per cent (37/41) had a CHOP-INTEND* score increase of at least 4 points, with 56% (23/41) achieving a score above 40; the median increase was 20 points. The median age at enrolment was 5.3 months.