• Treatment provides hope for patients suffering from devastating genetic disorder
• Public drug plans will cover Type 1 patients and Biogen will cover the most urgent Type 2 and 3 patients under an Urgent Access Program
• Biogen has resubmitted for a recommendation for broader access with updated clinical trial data

MISSISSAUGA, ON, Oct. 3, 2018 /CNW/ - Biogen Canada and the pan-Canadian Pharmaceutical Alliance (pCPA) have reached an agreement to provide SMA Type 1 patients with access to Biogen's product, SPINRAZA™(nusinersen).

SMA is a genetic disease characterized by loss of motor neurons which causes progressive muscle weakness and wasting, leading to severe handicap and death in some severely affected patients.  While SMA is rare and affects roughly only one in ten thousand people, its symptoms can be devastating, with some patients not living past their second birthday, and others dependent on respiratory and feeding equipment and confined to a wheelchair for life.

Nusinersen is the first and only approved treatment for SMA. Through an extensive clinical development program, nusinersen has shown significant improvements in survival rates and motor function across a broad range of types and ages of SMA patients. Under the terms of the agreement, participating drug plans from the pCPA will fund treatment in SMA Type 1 patients who meet clinical criteria, as recommended by the Canadian Agency for Drugs and Technologies in Health (CADTH) and l'Institut national d'excellence en sante et en services sociaux (INESSS).

In July 2018, Biogen resubmitted with updated clinical trial data, to the Health Technology Assessment (HTA) bodies, CADTH and INESSS requesting reconsideration for broader coverage of SPINRAZA to treat patients with other SMA types.  The results of the review by CADTH and INESSS due in early 2019 will inform future funding decisions for potentially broader coverage of SMA patients by public drug plans.  Meanwhile, Biogen Canada will begin to cover the most urgent Type 2 and 3 patients – defined as those without private insurance or for which private coverage has been declined and with the highest risk of losing motor function, for the period until the new HTA recommendations are issued.  To assess urgency, an Urgent Access Program guided by an independent Steering Committee of Canadian experts in SMA and rare diseases and supported by SMA 360 Canada (Biogen's SPINRAZA Patient Support Program) has been established.

This innovative approach is intended to ensure that urgent patients who require therapy will have access to nusinersen. Biogen is hopeful that the new HTA recommendations will take into account the full clinical development program for nusinersen and ensure that Canada can join over 40 other countries where nusinersen is available for a broad population of SMA patients.

According to Marina Vasiliou, vice president and general manager of Biogen Canada, "We are very pleased that we were able to reach an agreement that will make such a difference in the lives of people living with SMA in Canada and their families. This is the first step towards access to nusinersen for a broad population of Canadian patients and we believe it also marks the beginning of a partnership between Biogen and pCPA that will provide treatment and hope to patients with rare diseases and neurological conditions in Canada."

Statement from Cure SMA Canada:
"We at Cure SMA Canada are very happy to hear that an agreement was finally reached, offering treatment to our most critical patients. We will continue to advocate for our remaining patients who are left without treatment. We are hopeful and confident that the decision makers will not allow our remaining patients to continue on a path of progression while they watch others around the world, who are accessing Spinraza, gain in strength and abilities. We continue to wait anxiously for the response in January to the resubmission and expect a fast decision from the jurisdictions to start covering a broad population without extending the wait of Canadian families longer."
- Susi Vander Wyk, Executive Director, Cure SMA Canada

Statement from the Canadian Organization for Rare Disorders:
 "On behalf of those brave families who never gave up hope - or the fight, the Canadian Organization for Rare Disorders applauds the parties for coming to this agreement to provide access for the most urgent patients.  This arrangement is an important implementation of the 'risk-sharing, managed access' approach that CORD has long advocated for all rare disease drugs. It is exactly what we have submitted to the Advisory Council on National Pharmacare."
– Durhane Wong-Rieger, President and CEO, CORD

About Biogen
At Biogen, our mission is clear: we are pioneers in neuroscience. Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases. One of the world's first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Kenneth Murray and Nobel Prize winners Walter Gilbert and Phillip Sharp, and today has the leading portfolio of medicines to treat multiple sclerosis; has introduced the first and only approved treatment for spinal muscular atrophy; and is focused on advancing neuroscience research programs in Alzheimer's disease and dementia, multiple sclerosis and neuroimmunology, movement disorders, neuromuscular disorders, pain, ophthalmology, neuropsychiatry, and acute neurology. Biogen also manufactures and commercializes biosimilars of advanced biologics.

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