Roche receives positive CHMP opinion for Evrysdi, the first and only at home spinal muscular atrophy (SMA) treatment with proven efficacy in adults, children and infants two months and older

• In two pivotal clinical studies, Evrysdi showed event-free survival and motor milestone improvements never previously achieved in the natural history of the disease
• More than 2,500 patients now treated with Evrysdi in clinical trial, compassionate use and real-world settings
• Evrysdi is approved in seven countries, submitted in 30 more
  
  

Basel, 26 February 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Evrysdi (risdiplam) for the treatment of 5q spinal muscular atrophy (SMA) in patients 2 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. SMA causes progressive muscle weakness and atrophy, and significant unmet need remains, particularly in adults living with this condition.

The CHMP recommendation was completed under the accelerated assessment pathway, which is offered to medicines deemed to be of major interest for public health and therapeutic innovation. A final decision regarding approval is expected from the European Commission in the next two months and will be applicable to all 27 European Union member states, as well as Iceland, Norway, and Liechtenstein. Evrysdi was approved by the U.S. Food and Drug Administration (FDA) in August 2020.

“Our close partnership with the SMA community has enabled the development of the first ‘at-home’ treatment for SMA in infants, children and adults with varying levels of disease severity, the majority of whom remain untreated,” said Levi Garraway, M.D., Ph. D., Roche’s Chief Medical Officer and Head of Global Product Development. “If approved, Evrysdi would represent a much-needed therapeutic option and we expect it to become the treatment of choice for people living with SMA and their families."

The CHMP recommendation 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.

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. A liquid medicine, Evrysdi is administered daily at home by mouth or feeding tube.