Draft CADTH Recommendation for SPINRAZA™ (nusinersen) Fails Adults in Need of Treatment for Spinal Muscular Atrophy (SMA)

• CADTH does not accept new evidence demonstrating SPINRAZA™'s effectiveness and safety for treating SMA – a progressive, life-altering neurodegenerative disease
• SMA treatment access remains unequal across Canada, with only Quebec funding SMA care for patients over 18
• Implementing the draft recommendation would put Canada's health system out of step with those of over 25 countries, which fund treatment access for adult SMA patients

TORONTO, May 20, 2022 /CNW/ - Biogen Canada is deeply disappointed for the SMA community due to the draft recommendation from the Canadian Agency for Drugs and Technologies in Health (CADTH) that has advised against SPINRAZA™ (nusinersen) reimbursement for adult patients with spinal muscular atrophy (SMA)¹. SPINRAZA™, approved in Canada in 2017 as the first treatment for SMA, has been widely used and reimbursed by multiple provinces for patients under 18 as well as for patients of all ages in Quebec. More than two-thirds of Canadian adults with SMA are currently ineligible for public treatment access² and are progressively losing strength, abilities, and independence to the disease.

The draft CADTH recommendation acknowledges the unmet need for treatment options in adults with SMA. However, it did not accept the growing body of real-world evidence (RWE) that demonstrates SPINRAZA™'s safety and effectiveness when used in practice³, and effectively denies adult patients with access to life-altering treatment. Quebec is the only province that has aligned its reimbursement model with the health systems of over 25 countries, in which SMA patients of all ages – adults included – have access to treatment.⁴

Reaction from the SMA Community :

"Cure SMA Canada is devastated to hear of the negative draft recommendation from CADTH. Adult patients have been declining in physical and mental health as they have waited for access to treatment that has been proven safe and effective," said Susi Vander Wyk, Executive Director of Cure SMA Canada. "Many adult patients globally and in Quebec have accessed SPINRAZA™ and had a life-changing impact on their quality of life, and we urge the members of CADTH to reconsider their decision."

"The Canadian Organization for Rare Disorders feels betrayed by CADTH with this devastating recommendation NOT to provide access to adults with SMA despite the compelling real-world evidence that other countries have accepted and used to expand coverage," said Durhane Wong-Rieger, President and CEO of the Canadian Organization for Rare Disorders (CORD). "We desperately need a national Rare Disease Drug Strategy that is patient-centred."

"A very disappointing and unexpected response for the adult Canadian SMA community. All the more incomprehensible in the face of evidence of clinical effectiveness after three years of use in Quebec," said Dr. Xavier Rodrigue, doctor of physical medicine and rehabilitation at Hôpital de l'Enfant-Jésus, Quebec City. "It is important to support access to treatment that changes the course of a degenerative disease, restores hope and quality of life. But today, we must highlight this form of injustice and this inequitable access across Canada to effective treatment."

"The Canadian community of adults with spinal muscular atrophy remains in a challenging position where they are unable to access a treatment approved for them by Health Canada except in Quebec where SPINRAZA™ is available to patients of all ages," said Dr. Lawrence Korngut, Neuromuscular Neurologist with the University of Calgary's Department of Clinical Neurosciences. "Real world evidence has been collected around the world and has demonstrated benefits of SPINRAZA™ in adults with SMA." 

CADTH will be accepting stakeholder feedback on its draft recommendation through to June 3 prior to issuing its final recommendation. Biogen Canada remains unwavering in its commitment to the SMA community and will continue to work with all stakeholders in finding sustainable solutions to ensure equitable access for all SMA patients across Canada.

About Spinal Muscular Atrophy (SMA)
SMA is a rare, debilitating neurodegenerative condition that is characterized by loss of motor neurons in the spinal cord and lower brain stem, resulting in severe and progressive muscular atrophy and weakness.⁵ 

Due to a deletion of, or mutation in, the SMN1 gene, people with SMA do not produce enough survival motor neuron (SMN) protein, which is critical for the maintenance of motor neurons. The severity of SMA correlates with the amount of SMN protein an individual has. Ultimately, individuals with SMA can become paralyzed and have difficulty performing the basic functions of life, like breathing and swallowing.

People with Type I (infantile-onset) SMA, the form that requires the most intensive and supportive care, produce very little SMN protein and do not achieve the ability to sit without support or typically live beyond two years without respiratory support. People with Type II and Type III SMA produce greater amounts of SMN protein and have less severe, but still life-altering forms of SMA.

About SPINRAZA™ (nusinersen)
SPINRAZA™ (nusinersen) is approved to treat infants, children, and adults with all types of spinal muscular atrophy (SMA). As a foundation of care in SMA, more than 11,000 individuals have been treated with SPINRAZA™ worldwide.⁶ It is supported by the largest clinical data set in SMA with a clinical development program that encompasses 10 clinical studies, which have included more than 300 individuals across a broad spectrum of patient populations.⁷ SPINRAZA™ has demonstrated sustained efficacy across ages and SMA types with a well-established safety profile based on data in patients treated up to 7 years, combined with unsurpassed real-world experience.

About Biogen
As pioneers in neuroscience, Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological diseases as well as related therapeutic adjacencies. One of the world's first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Sir Kenneth Murray, and Nobel Prize winners Walter Gilbert and Phillip Sharp. Today, Biogen has the leading portfolio of medicines to treat multiple sclerosis, has introduced the first approved treatment for spinal muscular atrophy, and is providing the first and only approved treatment to address a defining pathology of Alzheimer's disease. Biogen is also commercializing biosimilars and focusing on advancing the industry's most diversified pipeline in neuroscience that will transform the standard of care for patients in several areas of high unmet need.

Since 1998, Biogen Canada has been delivering life-changing medicines and services that support unmet treatment needs and the lives of Canadians affected by neurological conditions. As a company that founds its culture on principles of equity, diversity and inclusion, Biogen Canada is proud to have been named a Best Workplace™ in Healthcare, for Inclusion, Giving Back and Hybrid Work, and Managed by Women. To learn more, please visit www.biogen.ca.

References

¹Canadian Agency for Drugs and Technology in Health. CADTH Reimbursement Recommendation Nusinersen (Spinraza). May 2022.

²Internal Biogen estimate as at May 2022.

³Hagenacker T, et al. Nusinersen in adults with 5q spinal muscular atrophy: a non-interventional, multicentre, observational cohort
study. Lancet Neurol. 2020 Apr;19(4):317-325.

⁴Biogen data on file.

⁵Kolb et al., 2015 Nov;33(4):831-46. doi: 10.1016/j.ncl.2015.07.004. J Kolb et al. 

⁶Coratti et al. Motor function in type 2 and 3 SMA patients treated with Nusinersen: a critical review. Orphanet Journal of Rare Diseases. 2021. 16:430.

⁷Biogen data on file.

SOURCE Biogen

MEDIA CONTACT: Biogen, Marija Mandic, + 1 416 234 7901, [email protected]