Spinraza (nusinersen) was granted the 2017 Prix Galien USA Award for Best Biotechnology Product in recognition of its achievement in scientific innovation for the treatment of spinal muscular atrophy (SMA), Biogen and Ionis announced.
The award was presented at a ceremony in New York City, on Oct. 26.
“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,” Michel Vounatsos, chief executive officer at Biogen, said in a press release. “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.”
Spinraza is an antisense oligonucleotide (ASO) that is made of small pieces of synthetic material that bind ribonucleic acid (RNA). The therapy was developed to treat children with SMA and targets the SMN2 gene, increasing the production of the SMN protein.
Biogen and Ionis achieved several key developmental goals for the therapy, which eventually led to Spinraza being given orphan drug status for the treatment of SMA by both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Soon after, Spinraza became the first treatment approved to treat SMA in the United States, in December 2016, under a priority review program for rare diseases.
The Galien Foundation is a nonprofit organization that oversees and directs activities in the U.S. for the Prix Galien, an international award that recognizes outstanding achievements that improve the human condition through innovation.
Today, the foundation fosters and rewards excellence in research and aims to serve as a vehicle for the open exchange of ideas that drive science. Activities fostered by the foundation include innovation roundtables, working groups, patient advocacy and collaboration, the Gallien International Network, and the Galien Young Leadership Series.
Founded in France 45 years ago by Rolan Mehl, the foundation was established to honor Galen, considered the father of medical science and modern pharmacology. Prix Galien is considered by some as the equivalent of the Nobel Prize in biopharmaceutical research.