CHMP Adopts Positive Opinion for Evrysdi for Spinal Muscular Atrophy

PTC Therapeutics announced 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 and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 with one to four SMN2 copies. The CHMP review was completed under the accelerated assessment pathway, which is offered to medicines deemed to be of major interest for public health and therapeutic innovation.

"CHMP opinion marks another important advancement in ensuring Evrysdi is available to SMA patients around the globe," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "Given its proven efficacy and strong safety profile, coupled with the convenience of an at-home administration, we expect Evrysdi to become the treatment of choice for SMA patients and their families."

The CHMP recommendation is based on data from the FIREFISH study in infants aged 2 to 7 months with symptomatic Type 1 SMA and the SUNFISH study in children and young adults with Type 2 or 3 SMA. The two pivotal studies were designed to represent a broad spectrum of people living with SMA, and SUNFISH is the first and only placebo-controlled trial to include adults with Types 2 and 3 SMA. 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 is designed to treat SMA by increasing and sustaining the production of the survival motor neuron (SMN) protein. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and movement. More than 2,500 patients have now been treated with Evrysdi in clinical trials, compassionate use programs and real-world settings, with patients ranging from birth to over 70 years old including those previously treated with other SMA therapies.

Evrysdi has been approved in seven countries thus far including the U.S., Chile, Brazil, Ukraine, South Korea, Georgia and Russia. Evrysdi is under review in a further 30 countries including Japan and China.

Evrysdi is based on PTC science and is commercialized in the United States by Genentech, a member of the Roche Group. Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.

Spinal muscular atrophy (SMA) is a severe, progressive neuromuscular disease that can be fatal. It affects approximately 1 in 10,000 babies and when untreated is the leading genetic cause of infant mortality. SMA is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of SMN protein. This protein is found throughout the body and is essential to the function of nerves that control muscles and movement. Without it, nerve cells cannot function correctly, leading to progressive muscle weakness over time. Depending on the type of SMA, an individual's physical strength and their ability to walk, eat or breathe can be significantly diminished or lost.

Evrysdi™ is a survival motor neuron 2 (SMN2)-directed RNA splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency. Evrysdi™ is designed to distribute evenly to all parts of the body, including the central nervous system (CNS). Evrysdi™ is administered daily at home in liquid form by mouth or feeding tube. The U.S. Food and Drug Administration recently approved Evrysdi™ (risdiplam) for the treatment of spinal muscular atrophy for adults and children 2 months and older. Evrysdi™ (risdiplam) is marketed in the United States by Genentech, a member of the Roche Group.