Evrysdi (Risdiplam) Receives CADTH Reimbursement Recommendation for Some Patients With Spinal Muscular Atrophy
- The CADTH recommendation is a step in the right direction for the public reimbursement of Evrysdi; however, limits access to treatment for many patients with SMA
- Canadians now wait more than 450 days longer for access to new medicines than those in the US and Europe1
- Provincial jurisdictions will make the final decision on public reimbursement
MISSISSAUGA, ON, Aug. 30, 2021 /CNW/ - Hoffmann-La Roche Limited (Roche Canada) today announced that the Canadian Agency for Drugs and Technologies in Health (CADTH) Canadian Drug Expert Committee (CDEC) has issued its final recommendation for Evrysdi® (risdiplam) for treatment of spinal muscular atrophy (SMA) for public reimbursement, with conditions, outside Quebec. The recommendation states that Evrysdi should be reimbursed for SMA patients if certain conditions are met.2 SMA is a progressive neuromuscular disease that affects the nerve cells in the spinal cord and impacts the muscles used for activities such as breathing, eating, crawling and walking.3,4 Evrysdi works by helping the body make more of a protein called, "SMN protein". Evrysdi increases and sustains the amount of SMN protein in the body, which helps to treat SMA. It is the first medicine for SMA that can be taken at home and is administered once daily by mouth or feeding tube.5
The conditions, as outlined in the recommendation, for those patients with SMA are as follows:2
- Genetic documentation of 5q SMA homozygous gene deletion or compound heterozygote
- Patients who are symptomatic and either:
- Aged between 2 months and 7 months (inclusive) and genetic documentation of 2 or 3 copies of the SMN2 gene; OR
- Aged 8 months and up to 25 years who are non-ambulatory and genetic documentation of 2 or 3 copies of the SMN2 gene.
- Patient is not currently requiring permanent invasive ventilation.
- The maximum duration of initial authorization is 12 months.
While this recommendation enables access to Evrysdi for a large portion of SMA patients in Canada, in practice, the required clinical criteria limits access to Evrysdi for patients with SMA over the age of 25 or who are able to walk independently.
"These recommendations are such an important step forward for the SMA community," said Dr. Colleen O'Connell, Assistant Professor in Rehabilitation Medicine at Dalhousie University. "I know this represents hope for so many who live with this disease. Moving forward, we must continue to study how such treatments help our patients throughout their lives, and particularly by treating them before the muscles become paralyzed."
Roche's clinical program for Evrysdi is designed to represent a broad spectrum of people living with SMA. Through its clinical trials, Evrysdi has demonstrated improvements in motor function in both pediatric and adult patients, with various levels of disease severity, including Types 1, 2 and 3.5
"We are pleased to see Canadian regulatory bodies are recognizing the value of innovative medicines for this patient group," said Susi Vander Wyk, Executive Director, Cure SMA Canada. "There is clear justification in preserving important motor functions for the wide range of SMA patients such as walking in addition to improved swallowing and speaking. Rapid access to treatments for SMA are vital to Canadians living with the condition. We look forward to a future when all SMA patients are able to access treatment in a timely manner, so the effects of this condition are halted, and offer hope for the future. This is a positive step towards filling in gaps where the end goal is achieving access to treatment for all SMA patients."
"These recommendations will be disadvantageous for Canadian SMA patients, many who have no other treatment options," said Durhane Wong-Rieger, President and CEO, Canadian Organization for Rare Disorders (CORD). "In stark contrast, many other countries, as well as INESSS in Quebec have approved broader access in adult patients. From the perspective of CORD, as Canada moves toward implementing the National Rare Disease Drug Strategy, our hope is that these Health Technology Assessments in Canada are ready to contribute to innovative access programs for innovative therapies."
For decades, Roche has been conducting neuroscience research and clinical trials aimed at exploring the areas of the highest medical need, where there are no, or limited, treatment options available. As part of this, Roche acknowledges the healthcare budget constraints faced by governments across the country and increasing fiscal pressures. Roche is committed to being part of the solution in making medicines for rare conditions more accessible to patients.
About EVRYSDI (risdiplam)
EVRYSDI is a survival of motor neuron 2 (SMN2) pre-mRNA splicing modifier designed to treat SMA by increasing and sustaining the amount of a protein called SMN, or survival of motor neuron, throughout the central nervous system and in peripheral tissues. SMN protein is essential for movement and motor function. EVRYSDI is a strawberry-flavoured liquid taken once daily by mouth or feeding tube.5
About Spinal Muscular Atrophy (SMA)
SMA is a progressive neuromuscular condition that affects the nerve cells in the spinal cord and impacts the muscles used for activities such as breathing, eating, crawling and walking.6,7 It affects approximately one in 6,000 babies born, and about one in 40 people are genetic carriers.8 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 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.9
Roche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve people's lives. The combined strengths of pharmaceuticals and diagnostics, as well as growing capabilities in the area of data-driven medical insights, help Roche deliver truly personalized healthcare. Roche aims to improve patient access to medical innovations by working with stakeholders across the entire healthcare sector to provide the best care for each person.
Roche is the world's largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management. In recent years, Roche has invested in genomic profiling and real-world data partnerships, has become an industry-leading partner for medical insights, and has collaborated in artificial intelligence (AI) data-mining to fuel healthcare insights.
Roche Canada was founded in 1931, and employs more than 1,800 people across the country through its Pharmaceuticals division in Mississauga, Ontario as well as its Diagnostics and Diabetes Care divisions in Laval, Quebec.
Roche continues to search for better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. Globally, Roche has been recognized as one of the most sustainable companies in the Pharmaceuticals Industry by the Dow Jones Sustainability Indices (DJSI) for twelve consecutive years. Roche Canada is also actively involved in local communities through its charitable giving and partnerships with organizations and healthcare institutions that work together to improve the quality of life of Canadians.
For more information, please visit www.RocheCanada.com or follow us on Twitter @RocheCanada.
SOURCE Roche Canada
For further information: Media Contact: Jennifer Mota, Roche Canada, 437-219-7806, [email protected]
1 Fraser Institute, 'Timely Access to New Pharmaceuticals in Canada, the United States, and the European Union.' Available at: Timely Access to New Pharmaceuticals in Canada, the United States, and the European Union | Fraser Institute. Last accessed May 25, 2021
2 CDEC final recommendations, August 26, 2021. Available at: https://cadth.ca/sites/default/files/DRR/2021/SR0661%20Evrysdi%20Recommendation%20Final.pdf. Last accessed August 26, 2021
3 Cure SMA, "About SMA", Available at https://www.curesma.org/about-sma/, Last accessed May 25, 2021
4 Treat-NMD Neuromuscular Network, Disease Information- Spinal Muscular Atrophy, Available at https://treat-nmd.org/treat-nmd-diseases/spinal-muscular-atrophy/ Last accessed May 25, 2021
5 Evrysdi Product Monograph, April 14, 2021
6 Cure SMA, "About SMA", Available at https://www.curesma.org/about-sma/, Last accessed May 25, 2021
7 Treat-NMD Neuromuscular Network, Disease Information- Spinal Muscular Atrophy, Available at https://treat-nmd.org/treat-nmd-diseases/spinal-muscular-atrophy/ Last accessed May 25, 2021
8 Cure SMA Canada, "About SMA", Available at https://curesma.ca/about-spinal-muscular-atrophy/. Last accessed May 25, 2021
9 Anderton RS, Mastaglia FL. Advances and challenges in developing a therapy for spinal muscular atrophy. Expert Rev Neurother. 2015;15(8):895-908. doi: 10.1586/14737175.2015.1059757. PMID: 26200127.