Biogen responds to coverage recommendation by the Canadian Drug Expert Committee (CDEC) for SPINRAZA, the only treatment approved for treatment of 5q Spinal Muscular Atrophy (SMA)

OTTAWA, Dec. 22, 2017 /CNW/ - Today the Canadian Drug Expert Committee (CDEC) made public its recommendation to the Common Drug Review (CDR) participating drug plans to reimburse SPINRAZA (nusinersen), the first and only approved treatment shown to be effective in the treatment of Spinal Muscular Atrophy (SMA). The recommendation is limited to a subset population of patients most likely to have or to develop Type 1 and diagnosed within a narrow clinical window. 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. 

CDEC's recommendation, although positive for a subset of patients, comes with disappointment in that the clinical criteria and conditions that accompany the recommendation greatly limit the population of patients with SMA that could potentially benefit from SPINRAZA. Furthermore, the recommendation limits the timeframe in which therapy can be initiated post diagnosis. SPINRAZA was approved by Health Canada in June 2017 for the treatment of patients with 5q SMA, and is the first and only approved treatment that has shown improvements in survival and motor function. It is estimated that 1 in 10,000 people are affected by SMA.¹

"Although positive for a select group patients, CDEC's recommendation is disappointing in that it does not currently recommend coverage for all existing populations of patients with SMA that could benefit from the treatment and where there is a highly significant unmet need," 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 limits the hope many of them have for coping with their SMA and delays their ability to receive treatment."

Biogen's clinical program, the largest in SMA to date, is comprised of multiple studies across a range of patients with SMA, including pre-symptomatic and symptomatic patients who had or were likely to develop Type 1, 2, or 3 SMA.  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. "The early termination of the studies is a strong recognition of the proven benefits of SPINRAZA and of the sense of urgency to make this treatment available. Governments, regulators, payors and medical communities around the world have recognized the broad benefit of SPINRAZA," explained Vasiliou. 

 "Biogen is committed to finding solutions and moving forward," continued Vasiliou. "We want to have an important and substantive dialog with the provincial drug formulary officials across Canada and continue the scientific dialogue with health care providers about the demonstrated clinical value of SPINRAZA for all patients with SMA who could benefit from the treatment.  We are hopeful that this can be done in a timely and constructive 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 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.

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SOURCE Biogen Canada Inc.

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