NICE Rejects Spinal Muscular Atrophy Drug But Welcomes Further Talks

Citing the high price tag and uncertain long-term effectiveness, NICE did not recommend Biogen’s Spinraza (nusinersen) for routine National Health Service use, but said it is open to  further discussion about access to the spinal muscular atrophy (SMA) treatment.

“We are actively engaging with Biogen to discuss how they might address the uncertainties identified by the committee, while demonstrating the potential for nusinersen to be considered cost effective and managing the risk to the NHS of allowing access to this treatment,” said Meindert Boysen, director of NICE’s center for health technology evaluation.

Evidence showed that the medication provided a significant clinical benefit, but concerns about its long-term benefits raised doubts about recommending it for routine use.

SMA affects the spinal cord, impacting nerves that control movement. It can cause progressive movement loss, difficulty breathing and swallowing and muscle weakness.