Evrysdi May Be Available in Early 2022 to Eligible Patients in UK

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by Marta Figueiredo PhD |

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The National Institute for Health and Care Excellence (NICE) has recommended that Roche’s Evrysdi (risdiplam) be covered by England’s national health service (NHS) and provided at low or no cost to eligible spinal muscular atrophy (SMA) patients as part of a managed access agreement.

The current recommendation and its eligibility criteria are consistent with Evrysdi’s approval in the U.K. in May for patients, 2 months and older, with a clinical diagnosis of SMA type 1, 2, or 3, or those not yet showing symptoms (pre-symptomatic) and carrying one to four copies of the SMN2 “backup” gene. The presence of that number of copies suggests the future development of any of those three disease types.

Under the managed access agreement — a special pricing deal between NHS England and Roche — the NHS will fund the oral therapy to eligible patients for three years, while more data are collected to help address the uncertainties identified by NICE during its initial evaluation, which was not in favor of Evrysdi’s coverage.

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The absence of comparative studies between Evrysdi and best supportive care for SMA type 1 and the lack of evidence of its long-term benefits, as well as its high cost-effectiveness ratio, remain NICE’s main concerns.

The therapy’s list price is nearly £8,000 (about $10,700), but the deal struck with Roche allows the NHS to access it at an undisclosed price that’s considered fair for taxpayers.

If there is no appeal to NICE’s draft guidance, its final recommendation is anticipated by Dec. 16 and the access program should start Jan. 15, 2022. After the three-year period, NICE will review the new evidence and update its guidance to indicate whether the therapy should be recommended for routine use on the NHS.

“We are pleased that there will now be the option of a convenient oral treatment for people with SMA that can be administered at home,” Meindert Boysen, deputy chief executive and director of NICE’s Centre for Health Technology Evaluation, said in a press release.

“This will not only be less burdensome, and therefore have a positive impact on the lives of both people with SMA and their caregivers, but it will also reduce the treatment administration needs to the NHS,” Boysen added. “In practical terms, the availability of an oral [therapy] should lead to greater adherence to treatment, along with giving access to a treatment to those who aren’t able to have other currently recommended options.”

Patients weighing more than 21 kilograms (about 46 pounds) are not eligible to receive the gene therapy Zolgensma, and severe scoliosis (or surgery to correct it) make Spinraza’s spinal injections challenging or unfeasible.

At least 215 type 1 or 2 patients in England who were not suitable for other disease-modifying therapies received Evrysdi via the early access scheme provided earlier this year. These patients may continue Evrysdi treatment if they meet the new agreement’s eligibility criteria.

“In the last three years the NHS has revolutionized care for people with Spinal Muscular Atrophy, by securing access to a trio of innovative treatments — Spinraza, Zolgensma and now [Evrysdi] — whereas three years ago clinicians had no effective medicines at all,” Amanda Pritchard, chief executive of the NHS, said in a separate press release.

Elizabeth Wraige, MD, consultant pediatric neurologist at Evelina London Children’s Hospital, part of Guy’s and St Thomas’ NHS Foundation Trust, said: “these are very exciting times and I am sure this news will be welcomed by those families and individuals affected by SMA as well as by their clinicians.”

Roche is in active discussions with relevant authorities in Wales and Northern Ireland to ensure NICE’s recommendation is adopted as soon as possible in these countries as well. In Scotland, Evrysdi is currently being reviewed by respective health authorities, and guidance is anticipated by early 2022.

Evrysdi, given as a flavored liquid by mouth or feeding tube once a day, works by increasing the levels of SMN, a protein essential for motor neuron and muscle health and whose production is impaired in people with SMA.

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

Results showed that the therapy 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.

Evrysdi’s clinical program also includes the global Phase 2 JEWELFISH (NCT03032172) and RAINBOWFISH (NCT03779334) trials, which are evaluating the therapy in different patient groups.

Interim findings from these studies showed that pre-symptomatic babies given Evrysdi for at least a year reached many of the same motor milestones as healthy children, while SMA patients up to 60 years old who had received prior SMA treatment and are now on Evrysdi more than doubled their SMN levels and showed stable motor function.

RAINBOWFISH is still enrolling pre-symptomatic newborns up to 6 weeks old with a genetic diagnosis of SMA.

“Ensuring access to the latest medicines for rare diseases like SMA can be challenging,” said Richard Erwin, general manager of Roche Products Limited UK. “But, from our initial commitment via a successful Early Access to Medicines Scheme to a close collaboration with [NHS England], NICE and the SMA community, today’s news shows that if everyone works together, we can achieve a positive outcome.”

In a letter to SMA UK, Roche stated: “Today’s news would not have been possible without the support, collaboration and passionate advocacy of patient organizations and individuals across the UK SMA community. We would like to thank you for all you have done to achieve this milestone.”

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