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Biogen and Ionis Win Prestigious Prix Galien Award for SPINRAZA as Best Biotechnology ProductBiogen (NASDAQ: BIIB) and Ionis have been awarded the prestigious 2017 Prix Galien USA Award for Best Biotechnology Product for SPINRAZA® (nusinersen). The Prix Galien USA Award recognizes extraordinary achievement in scientific innovation that improves the state of human health. The award was presented at a ceremony in New York City on October 26, 2017. "We are humbled to be recognized by the Galien Foundation with Ionis for SPINRAZA, the first and only therapy to treat the devastating condition of spinal muscular atrophy," said Michel Vounatsos, chief executive officer at Biogen. "SPINRAZA's profound clinical impact is certainly the most rewarding outcome for all of us involved, and I want to thank our Biogen colleagues around the world for their passion and commitment to making a difference in this area of medical need." The U.S. Food and Drug Administration (FDA) approved SPINRAZA on December 23, 2016 under priority review for the treatment of SMA (News - Alert) in pediatric and adult patients. SMA is a rare disease and leading genetic cause of death in infants marked by progressive, debilitating muscle weakness taking away a person's ability to walk, eat and ultimately breathe. SPINRAZA is an approved therapy for the treatment of SMA. For more information about SPINRAZA and prescribing information in the United States, please visit www.SPINRAZA.com.
SPINRAZA Program Status Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals, a leader in antisense therapeutics. Biogen and Ionis conducted an innovative clinical development program that moved SPINRAZA from its first dose in humans in 2011 to its first regulatory approval in five years.
About The Galien Foundation
About SMA1-5 Due to a loss of, or defect in, the SMN1 gene, people with SMA do not produce enough 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 an antisense oligonucleotide (ASO), using Ionis Pharmaceuticals' proprietary antisense technology, that is designed to treat SMA caused by mutations r 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 survival motor neuron (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 antisense oligonucleotides. Individuals may be at increased risk of bleeding complications. Renal toxicity has been observed after administration of some antisense oligonucleotides. SPINRAZA is present in and excreted by the kidney.
About Biogen
Biogen Safe Harbor 1. 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. 2. Lefebvre S, Burglen L, Reboullet S, et al. Identification and characterization of a spinal muscular atrophy-determining gene. Cell.1995;80(1):155-165. 3. 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. 4. Monani UR, Lorson CL, Parsons (News - Alert) 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. 5. Peeters K, Chamova T, Jordanova A. Clinical and genetic diversity of SMN1-negative proximal spinal muscular atrophies. Brain.2014;137(Pt 11):2879-2896. 6. Hua Y, Sahashi K, Hung G, Rigo F, Passini MA, Bennett CF, Krainer AR. Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model. Genes Dev. 2010 Aug 1; 24(15):16344-44. 7. Evers MM, Toonen LJ, van Roon-Mom WM. Antisense oligonucleotides in therapy for neurodegenerative disorders. Adv Drug Deliv Rev. 2015;87:90-103. 8. Lunn MR, Wang CH. Spinal muscular atrophy. Lancet. 2008;371(9630):2120-2133.
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