The initial position of England’s National Institute for Health and Care Excellence (NICE) is to recommend against adding Roche’s Evrysdi (risdiplam) to the list of medications available to spinal muscular atrophy (SMA) patients through the country’s national health service (NHS).

Lack of evidence of Evrysdi’s efficacy in babies genetically diagnosed with SMA but not yet showing symptoms (pre-symptomatic) and of its long-term benefits, as well as its high cost, were cited as the main concerns in NICE’s draft guidance.

Still, the NICE appraisal committee acknowledged the therapy’s meaningful benefits in motor function of SMA patients, as well as its innovative, more convenient oral administration that also provides an alternative for people who cannot receive Spinraza’s spinal cord injections.

Patients, caregivers, healthcare professionals, and advocates now have the opportunity to comment on NICE’s draft guidance. A consultation period will run until June 23.

“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,” Meindert Boysen, deputy chief executive and director of NICE’s Centre for Health Technology Evaluation, said in a press release.

“The committee considered that there may be some benefits that have not been captured in [Roche]’s economic model, such as improvements in respiratory function, fine motor skills, swallowing and ability to communicate,” Boysen said.

“We will continue to work closely with the company to help them address the committee’s concerns ahead of its next meeting,” Boysen added.

While expressing their disappointment, SMA groups across the U.K. emphasized that is not unusual for NICE to have certain questions at this stage. Indeed, many recalled the long process of Spinraza’s appraisal by NICE before being approved in 2019.

The patient groups will now prepare their comments on the draft guidance and they urge anyone interested in having a say to do the same. The consultation will be open for comments until Wednesday, June 23 at 5 p.m.

In a blog post, Robert Burley, director of campaigns, care, and support at Muscular Dystrophy UK — which is an official consultee in the appraisal process — said that the nonprofit is “extremely disappointed” with NICE’s announcement, especially because Evrysdi “could be potentially life-changing for people living with SMA.”

“Muscular Dystrophy UK will therefore continue campaigning with SMA UK to represent the experiences of those affected by [NICE’s] announcement and strongly encourage as many people as possible to complete the consultation,” Burley said.

The patient groups also urge NICE, NHS England, and Roche to “work together as quickly as possible to find a cost-effective solution that will see the initial recommendation overturned,” Burley added.

The appraisal committee will meet again on July 13 to consider the evidence, the consultation documents, and comments from consultees before making a final recommendation.

NICE’s initial recommendation comes a little more than two months after Evrysdi was approved by the European Commission for patients 2 months and older. The EU approval covers individuals with a clinical diagnosis of SMA type 1, 2, or 3, or those carrying one to four copies of the SMN2 “backup” gene. The regulatory ruling itself came two weeks after Evrysdi was approved in the U.K. for the same indication.

Evrysdi will remain available in the U.K. for patients currently receiving treatment through funding arrangements already in place, including those with type 1 or 2 disease being treated under the established early access to medicines scheme, known as EAMS.

New SMA patients currently can enter EAMS only if they were identified before May 20 and have been assessed. Additionally, their paperwork must be completed and submitted by July 1.

Roche, Evrysdi’s manufacturer and one of its developers, plans to submit a similar marketing application to the Scottish Medicines Consortium. NICE recommendations usually are followed by health authorities in Northern Ireland and Wales.

To date, Evrysdi has been approved in more than 35 countries, including Brazil, Canada, Chile, Georgia, Russia, South Korea, and Ukraine.

A small molecule, Evrysdi works by increasing the levels of SMN, a protein essential for motor neuron and muscle health whose production is impaired in people with SMA. Given as a flavored liquid, it is administered daily at home by mouth or feeding tube.

Regulatory approvals were based on top-line data from the ongoing Phase 2/3 FIREFISH (NCT02913482) and SUNFISH (NCT02908685) trials, which are evaluating Evrysdi’s safety and effectiveness in a total of 221 patients, 1 month to 25 years old, with SMA types 1, 2, and 3.

Results showed that Evrysdi was generally safe and led to significant improvements in survival, swallowing, breathing, and motor milestones in type 1 infants, and in motor function in children and young adults with types 2 and 3 disease.

The therapy’s clinical program also includes the global, Phase 2 JEWELFISH (NCT03032172) and RAINBOWFISH (NCT03779334) trials.

The fully enrolled JEWELFISH study involves patients ages 6 months to 60 years, who were treated previously with other SMA-targeting therapies. RAINBOWFISH is still recruiting pre-symptomatic newborns up to 6 weeks old with SMA; more information is available here.

According to Roche, more than 3,000 patients have been treated with Evrysdi in clinical trials, compassionate use programs, and real-world settings.

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