QUEBEC, Dec. 15, 2017 /CNW/ - Today the Institut national d'excellence en santé et en services sociaux (INESSS) made public its recommendation on SPINRAZA^TM (nusinersen) in Quebec, the first and only approved treatment shown to be effective in the treatment of Spinal Muscular Atrophy (SMA). Although INESSS recognized the therapeutic value attributed to the drug based on the trial results of infants with early onset SMA, it did not extend the same recognition to children who develop the disease later albeit still at a very young age or to those who are diagnosed with the disease but have yet to show symptoms. SMA is a debilitating degenerative condition and the leading genetic cause of death among infants. Children with the most severe form of SMA rarely live to see their second birthday.

Approved by Health Canada in June 2017 for the treatment of patients with 5q spinal muscular atrophy (SMA), SPINRAZA is the first and only approved treatment that has shown improvements in survival and motor function for those diagnosed with SMA.  It is estimated that 1 in 10,000 people are affected by SMA.¹

"INESSS's recommendation is disappointing and does not fully reflect the overwhelming clinical and scientific data available," said Marina Vasiliou, Managing Director of Biogen Canada. "Biogen believes that the benefits of SPINRAZA are clear for patients across the spectrum of the disease and that this recommendation clearly limits the hope many of them have for coping with their SMA."

Biogen's clinical studies in SMA provided as part of the review process and published or presented at varying congresses, include clinical and scientific data that demonstrate the value of SPINRAZA across the spectrum of the disease from pre-symptomatic, to early and later onset. In the absence of alternatives and based on the severity of the disease, the drug was considered for priority review by INESSS.  The certainty of the benefits demonstrated in the clinical trials resulted in the early termination of the studies as well as accelerated and expedited regulatory approvals across the globe, including Canada.  

"Biogen is committed to finding solutions immediately," continued Vasiliou. "We want to have an important and substantive dialog with the Quebec provincial drug formulary officials and health care providers about the demonstrated value of SPINRAZA for patients with SMA and their families and the importance of early treatment.  We are hopeful that this can be done in a manner that does not further delay patients' abilities to access the drug". 

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. Founded in 1978 as one of the world's first global biotechnology companies by Charles Weissman 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 and only approved treatment for spinal muscular atrophy; and is focused on advancing neuroscience research programs in Alzheimer's disease and dementia, neuroimmunology, movement disorders, neuromuscular disorders, pain, ophthalmology, neuropsychiatry, and acute neurology. Biogen also manufactures and commercializes biosimilars of advanced biologics. We routinely post information that may be important to investors on our website at www.biogen.com. To learn more, please visit www.biogen.com and follow us on social media – Twitter, LinkedIn, Facebook, YouTube.

About SMA ^2-6
SMA is characterized by loss of motor neurons in the spinal cord and lower brain stem, resulting in severe and progressive muscular atrophy and weakness. Ultimately, individuals with the most severe type of SMA can become paralyzed and have difficulty performing the basic functions of life, like breathing and swallowing.

Due to a loss of, or defect 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. People with Type 1 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 live beyond two years without respiratory support. People with Type 2 and Type 3 SMA produce greater amounts of SMN protein and have less severe, but still life-altering forms of SMA.

About SPINRAZA ^® (nusinersen)
SPINRAZA is being developed globally for the treatment of SMA.

SPINRAZA is an antisense oligonucleotide (ASO), using Ionis' proprietary antisense technology, that is designed to treat SMA caused by mutations or deletions in the SMN1 gene located in chromosome 5q that leads to SMN protein deficiency. SPINRAZA alters the splicing of SMN2 pre-mRNA in order to increase production of full-length SMN protein.⁶ ASOs are short synthetic strings of nucleotides designed to selectively bind to target RNA and regulate gene expression. Through use of this technology, SPINRAZA has the potential to increase the amount of full-length SMN protein in individuals with SMA.

SPINRAZA must be administered via intrathecal injection, which delivers therapies directly to the cerebrospinal fluid (CSF) around the spinal cord,⁷ where motor neurons degenerate in individuals with SMA due to insufficient levels of SMN protein.⁸

SPINRAZA demonstrated a favorable benefit-risk profile. The most common adverse reactions reported for SPINRAZA were upper respiratory infection, lower respiratory infection, and constipation. Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients. Coagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some ASOs. Individuals may be at increased risk of bleeding complications. Renal toxicity has been observed after administration of some ASOs. SPINRAZA is present in and excreted by the kidney.

1. Farrar M, Kiernan M. The genetics of spinal muscular atrophy: progress and challenges. The American Society for Experimental NeuroTherapeutics. 2015; 12:290-302 
2. Darras B, Markowitz J, Monani U, De Vivo D. Chapter 8 - Spinal Muscular Atrophies. In: Vivo BTD, ed. Neuromuscular Disorders of Infancy, Childhood, and Adolescence (Second Edition). San Diego: Academic Press; 2015:117-145.
3. Lefebvre S, Burglen L, Reboullet S, et al. Identification and characterization of a spinal muscular atrophy-determining gene. Cell.1995;80(1):155-165.
4. Mailman MD, Heinz JW, Papp AC, et al. Molecular analysis of spinal muscular atrophy and modification of the phenotype by SMN2. Genet Med. 2002;4(1):20-26.
5. Monani UR, Lorson CL, Parsons DW, et al. A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN2. Hum Mol Genet. 1999;8(7):1177-1183.
6. Peeters K, Chamova T, Jordanova A. Clinical and genetic diversity of SMN1-negative proximal spinal muscular atrophies. Brain.2014;137(Pt 11):2879-2896

SOURCE Biogen Canada Inc.

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