Biogen (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 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
SPINRAZA is the first approved medicine for the treatment of SMA and is currently approved in the United States, the European Union, Brazil, Japan, Switzerland and Canada. Biogen has submitted regulatory filings in additional countries and plans to initiate additional filings in other countries.
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
The Galien Foundation fosters, recognizes and rewards excellence in scientific innovation to improve the state of human health. The Foundation oversees and directs activities in the USA for the Prix Galien, an international award that recognizes outstanding achievements in improving the human condition through the development of innovative therapies. The Prix Galien was created in France in 1970 in honor of Galen, the father of medical science and modern pharmacology.
Spinal muscular atrophy (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 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 Pharmaceuticals’ 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 survival motor neuron (SMN) protein.8