MISSISSAUGA, ON,?April 15, 2021?/CNW/ - Hoffmann-La Roche Limited (Roche Canada) today announced that Health Canada has granted EVRYSDI^??(risdiplam) market authorization for the treatment of spinal muscular atrophy (SMA) in patients two months of age or older.ⁱⁱⁱ 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.ⁱⁱⁱ "The Health Canada approval of EVRYSDI is a welcome addition in our ability to treat SMA. It is an efficacious treatment that significantly increases survival motor neuron (SMN) protein in SMA patients," said Dr.?Hugh McMillan, Pediatric Neurologist at the Children's Hospital of?Eastern Ontario. "We now have an additional and convenient treatment option with EVRYSDI, as it's the first at-home and oral option for patients." The Health Canada market authorization was based on data from two pivotal clinical trials designed to represent a spectrum of people living with SMA: FIREFISH in symptomatic infants aged two?to seven months and SUNFISH in children and adults aged two to 25 years.ⁱⁱⁱ 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.ⁱ?ⁱⁱ?It affects approximately one in 6,000 babies born, and about one in 40 people are genetic carriers.^iv??SMA is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of the survival motor neuron (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.^v "Cure SMA enthusiastically welcomes the arrival of EVRYSDI to the Canadian market. Despite the treatments for SMA currently available, a number of Canadians remain without treatment and a large percentage do not have access to existing ones due to their age, state of health or due to the complexity of administering treatment in a hospital setting," said?Amy Loignon, Co-President, Cure SMA Quebec. "This situation is especially exacerbated by COVID-19 and the burden caused by it. We are pleased to see this first oral treatment for SMA, giving these patients hope that their condition may stabilize and even improve." About the Health Canada Approval The Health Canada market authorization of EVRYSDI is based on data from two pivotal clinical trials designed to represent a spectrum of people living with SMA: FIREFISH in symptomatic infants aged two to seven months; and SUNFISH in children and adults aged two to 25 years.ⁱⁱⁱ

• FIREFISH?is an open-label, two-part study investigating the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of EVRYSDI in symptomatic Type 1 SMA patients (all patients had genetically confirmed disease with two copies of the SMN2 gene).ⁱⁱⁱ
  • Part 1 of FIREFISH was designed as the dose-finding part of the study. The confirmatory Part 2 of the FIREFISH study assessed the efficacy of EVRYSDI at the therapeutic dose selected based on the results from Part 1.ⁱⁱⁱ
  • Patients from Part 1 did not take part in Part 2. In Parts 1 and 2, the key efficacy endpoint was the ability to sit without support for at least five seconds, as measured by Item 22 of the Bayley Scales of Infant and Toddler Development ? Third Edition (BSID-III) gross motor scale, after 12 months treatment with EVRYSDI.ⁱⁱⁱ
    
    
• SUNFISH?is a two-part, multicenter trial to investigate the efficacy, safety, PK and PD of EVRYSDI in SMA Type 2 or Type 3 patients between two to 25 years of age.
  • Part 1 was the exploratory dose-finding portion.ⁱⁱⁱ
  • Part 2 was the randomized double-blind placebo-controlled confirmatory portion. Patients from Part 1 did not take part in Part 2. The primary endpoint was the change from baseline score at month 12 on the Motor Function Measure-32 (MFM32).ⁱⁱⁱ

In clinical trials, EVRYSDI improved motor function in pediatric and adult patients with various levels of disease severity, including Types 1, 2, and 3 SMA.^iii?

• Analysis of the data indicates 29.3 per cent of participants with infantile-onset SMA were able to sit without support for at least five seconds as measured by Item 22 of the BSID-III gross motor scale (29.3%; 90% CI:17.8%, 43.1%; p<0.0001); a key motor milestone not normally seen in the natural course of the disease. Additionally, 85.4 per cent of participants met the criteria of event-free survival without permanent ventilation (secondary endpoint) in each case after 12 months of treatment (85.4%; 90% CI:73.4%, 92.2%; p<0.0001).ⁱⁱⁱ
  
  
• Analysis of the data indicates patients with later-onset SMA met the efficacy criteria of significant improvement in motor function assessed by the Motor Function Measure-32 (MFM32) (1.55 point mean difference; p<0.0156) at 12 months of treatment with EVRYSDI, compared to placebo (1.36 points [95% CI: 0.61, 2.11]; -0.19 points [95% CI: -1.22, 0.84], respectively).ⁱⁱⁱ

About EVRYSDI^??(risdiplam) EVRYSDI is a pre-mRNA splicing modifier of survival of motor neuron 2 (SMN2) designed to treat SMA, which is caused by mutations in chromosome 5q that lead to SMN protein deficiency. 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. EVRYSDI is a strawberry-flavoured liquid taken once daily by mouth or feeding tube.^iii? 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.^i?ii?It affects approximately one in 6,000 babies born, and about one in 40 people are genetic carriers.^iv??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.^v? About Roche 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 under one roof, combined with a focus on innovation, have made Roche the leader in personalized healthcare ? a strategy that aims to provide patients with timely access to their best possible healthcare solution. 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. Founded in 1931, Roche Canada is committed to searching for better ways to prevent, diagnose and treat diseases while making a sustainable contribution to society. The company employs more than 1,500 people across the country through its Pharmaceuticals division in?Mississauga, Ontario?and Diagnostics, as well as Diabetes Care divisions in?Laval,?Quebec. Roche aims to improve patient access to medical innovations by working with all relevant stakeholders. Roche?Canada?is 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. SOURCE Roche Canada