NICE draft guidance does not recommend Evrysdi for SMA

The UK National Institute for Health and Care Excellence (NICE) has announced that it does not recommend Roche’s Evrysdi for the treatment of spinal muscular atrophy (SMA) in draft guidance.

Evrysdi (risdiplam) is licensed for the treatment of patients with types 1, 2 and 3 SMA in the EU.

In a statement, NICE said that although clinical trials demonstrate that Roche’s drug meaningfully improves motor function for these patients, the long-term benefits are uncertain.

The committee also added that the oral administration of Evrysdi is ‘innovative’, as it offers an alternative for patients who cannot receive other SMA treatments and allows for home administration.

NICE said that, at this time, it is ‘unable’ to recommend Evrysdi as the cost-effectiveness estimates for the drug were substantially higher than what is normally considered a cost-effective use of NHS resources.

“We are disappointed not to be able to recommend this innovative new treatment as an option for people with this devastating condition, particularly where there remains a high unmet clinical need,” said Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Evaluation at NICE.

“The committee considered that there may be some benefits that have not been captured in the company’s economic model, such as improvements in respiratory function, fine motor skills, swallowing and ability to communicate. We will continue to work closely with the company to help them address the committee’s concerns ahead of its next meeting,” he added.