Ionis Pharmaceuticals announced that the European Commission (EC) has granted marketing authorization for SPINRAZA® (nusinersen) for the treatment of 5q spinal muscular atrophy (SMA). 5q SMA is the most common form of the disease and represents approximately 95% of all SMA cases. SPINRAZA is the first approved treatment in the European Union (EU) for SMA, a leading genetic cause of death in infants that is marked by progressive, debilitating muscle weakness. SPINRAZA was reviewed under the European Medicines Agency’s (EMA) accelerated assessment program, intended to expedite access to patients with unmet medical needs.
In conjunction with the SPINRAZA approval in the EU, Ionis earned a $50 million milestone payment from Biogen. Ionis is also eligible to receive tiered royalties on global sales of SPINRAZA. To date, Ionis has earned more than $375 million from Biogen related to SPINRAZA.
“This is a landmark day for the European SMA community. The approval of SPINRAZA to treat a broad range of patients with SMA provides patients currently living with SMA hope for disease stabilization or improvement. Further, I am encouraged that the approval of SPINRAZA may demonstrate the potential of other antisense oligonucleotides to treat more neurodegenerative disorders,” Eugenio Mercuri, MD, Università Cattolica del Sacro Cuore, Rome, Italy.
The approval of SPINRAZA was primarily based on results from two pivotal multicenter, controlled studies, including end of study data from ENDEAR (infantile-onset SMA) and an interim analysis of CHERISH (later-onset SMA), both of which demonstrated the clinically meaningful efficacy and favorable benefit-risk profile of SPINRAZA. The approval was also supported by results from open-label studies in pre-symptomatic and symptomatic individuals with, or most likely to develop, Types 1, 2 or 3 SMA.
“We would like to convey our gratitude to the patients, families, physicians and their teams in the EU who participated in our clinical trials. Their support and dedication have been critical in achieving this important milestone,” said B. Lynne Parshall, chief operating officer at Ionis Pharmaceuticals. “In the U.S., the initial launch of SPINRAZA is off to a strong start and we continue to be pleased with the dedication, passion, and commitment of our partners at Biogen. We are pleased that this approval makes SPINRAZA widely available in the EU to treat a broad range of people with SMA.”
Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals in August 2016 and is now responsible for all development, regulatory and commercialization activities and costs for SPINRAZA. SPINRAZA was first approved by the U.S. Food and Drug Administration (FDA) on December 23, 2016, within three months of regulatory filing.