Biogen said Thursday that the European Commission approved Spinraza (nusinersen) for the treatment of 5q spinal muscular atrophy (SMA), making it the first therapy authorised in Europe for the condition. The company noted that 5q SMA is the most common form of the disease and represents approximately 95 percent of all SMA cases.
According to Biogen, approval of the drug was supported by data from two Phase III studies. In the ENDEAR trial of patients with infant-onset SMA, Spinraza was associated with a significantly larger percentage of patients who achieved the definition of motor milestone responder versus the control, while the drug was also linked to a significant reduction in the risk of death or permanent ventilation. Meanwhile, in a pre-specified interim analysis of the CHERISH study of subjects with later-onset SMA, the therapy produced significant improvements in motor function versus no treatment.
Spinraza was awarded accelerated assessment by the European Medicines Agency in October 2016, with the regulator's Committee for Medicinal Products for Human Use issuing a positive opinion supporting approval of the therapy in April. Biogen indicated that it has also submitted the treatment for regulatory approval in Japan, Canada, Australia, Switzerland and Brazil, while additional filings are planned for later this year.
Spinraza was approved in the US in December last year for the treatment of SMA in adults and children. Biogen later disclosed that the drug would be priced at $750 000 for the first year of treatment and $375 000 for each additional year of therapy. The company, which licensed global rights to Spinraza from Ionis Pharmaceuticals in 2012, recently reported that the product amassed $47 million in revenue in the first quarter of the year.
For related analysis, see ViewPoints: Biogen looking to make the most of an early stocking stuffer from FDA.
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