Biogen (NSDQ:BIIB) said last week that it won FDA approval for its spinal muscular atrophy treatment, Spinraza. The drug is the 1st treatment approved in the U.S. for spinal muscular atrophy, a progressive and often fatal genetic disease that causes muscle weakness in infants and toddlers.
The FDA previously granted Biogen’s application fast track designation and priority review, and Spinraza also received orphan drug designation.
Approval for Spinraza was based on a clinical trial that enrolled 121 patients with infantile-onset SMA. Patients were randomized into 2 groups – they either received an injection of Spinraza into the fluid surrounding the spinal cord or they received a mock procedure without drug injection. Researchers evaluated the percentage of patients in both groups that demonstrated improvement in milestones such as head control, sitting, rolling and standing.
Biogen conducted an interim analysis of 82 of the patients and reported that 40% of patients treated with Spinraza saw improvement in motor milestones, while none of the patients in the control arm improved. The most commonly reported side effects were upper respiratory infection, lower respiratory infection and constipation.
The drug will be available for shipment throughout the U.S. starting this week, according to Biogen.
“Spinraza offers new hope for the SMA community and exemplifies our mission of applying cutting-edge science to make a meaningful difference in the lives of patients with devastating, life-altering diseases,” Biogen CEO George Scangos said in prepared remarks. “We are humbled and grateful for the commitment of the patients and families who participated in the Spinraza clinical trial program, the tireless efforts of our investigators, and the urgency demonstrated by the FDA in rapidly reviewing and approving this treatment. We also want to acknowledge the important work of our colleagues at Ionis, who initiated this program.”
Ionis Pharmaceuticals (NSDQ:IONS) licensed the rights to develop, manufacture and commercialize Spinraza to Biogen. The Carlsbad, Calif.-based pharmaceutical company received a $60 million milestone payment from Biogen following Spinraza’s approval.
“At Ionis, we have had the privilege of discovering and, together with Biogen, developing Spinraza. We are very pleased with today’s announcement, which is an important milestone for the entire SMA community. Now we look forward to the benefit that Spinraza can bring to patients with SMA and their families. Spinraza is truly a precision medicine that works by altering the processing of a single cellular RNA. We are proud that Spinraza exists because Ionis created and validated a new platform for drug discovery, antisense technology,” Ionis’ chairman & CEO Dr. Stanley Crooke added. “We want to thank the families, physicians and their staff who participated in our clinical trials. Their dedication and support have been crucial to the approval of Spinraza for all those with SMA.”