Biogen, Ionis' Spinraza cleared by FDA for treatment of spinal muscular atrophy

The FDA announced on December 23 the approval of Biogen and Ionis Pharmaceuticals' Spinraza (nusinersen) for use in children and adults with spinal muscular atrophy (SMA), making the drug the first treatment approved in the US in this indication. Billy Dunn, director of the Division of Neurology Products in the FDA's Center for Drug Evaluation and Research, remarked "we worked hard to review this application quickly," adding "we could not be more pleased to have the first approved treatment for this debilitating disease." 

The approval was supported by safety and efficacy data from clinical studies involving more than 170 patients. In study data unveiled in August, the drug was linked to statistically significant improvement in the achievement of motor milestones versus no treatment in patients with infantile-onset SMA. Additional study data released in November showed that the therapy was associated with significant improvements in motor function compared to no treatment in non-ambulatory patients with later-onset SMA. 

Biogen recently disclosed that Spinraza will cost $750 000 per patient in the first year of treatment, and fall to $375 000 per patient in subsequent years, with RBC Capital Markets analyst Michael Yee projecting that sales could reach $2 billion annually. Evercore ISI analyst John Scotti estimated that 2500 patients in the US, Europe and Japan have type 1 SMA, while an additional 10 000 people in those markets have type 2 SMA.

The news comes after Biogen announced on December 19 that chief commercial officer Michel Vounatsos had been tabbed to succeed George Scangos as CEO, effective January 6. Vounatsos had identified winning marketing authorisation of Spinraza, which has also been accepted for review in Europe, as a near-term priority for the company. For related analysis, see ViewPoints: New Biogen CEO may get a frosty receptor from investors, through little fault of his own

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