Biogen, NHS England reach deal allowing funding of spinal muscular atrophy therapy Spinraza

The National Institute for Health and Care Excellence announced Wednesday that Biogen's Spinraza (nusinersen) will be available to patients with spinal muscular atrophy after a deal was reached between the company and NHS England. Under the managed access agreement, NHS England will fund treatment for a time-limited period, allowing further data to be collected on Spinraza's effectiveness.

NICE was previously unable to recommend routine use of the drug, which is the first treatment that targets the underlying cause of SMA, because of uncertainties over its long-term effectiveness and its high cost. Spinraza's list price is 450 000 pounds ($579 628) for the first year of treatment, and 225 000 pounds ($289 814) for subsequent years, with Biogen offering a confidential price reduction.

According to NICE, Spinraza will be made available to SMA type 1 patients immediately by Biogen, with NHS England offering funding once final guidance is published by the agency next month. Meanwhile, for older babies, children and young adults with SMA types 2 and 3, the NHS will begin to roll out Spinraza shortly after the guidance is published.

Commenting on the news, Simon Stevens, NHS England chief executive, said "the NHS has now reached one of the most comprehensive deals in the world, which allows us to assess real-world evidence of its long-term benefits." Stevens added "this latest deal…demonstrates that there is no reason for other companies not to show equivalent flexibility in order to benefit NHS patients, taxpayers and indeed themselves."

Spinraza gained European approval in 2017 for the treatment of 5q SMA, making it the first therapy authorised in the region for the condition, having previously received clearance in the US. Last month, Biogen reported that first-quarter sales of the drug, which was licensed from Ionis Pharmaceuticals in 2012, jumped 42 percent year-over-year to $518 million.

For related analysis, see KOL Views: Sizing up the bogeys on Spinraza's radar in SMA.

To read more Top Story articles, click here.