Evrysdi Soon Available at Low or No Cost to SMA Patients in Scotland

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by Marta Figueiredo PhD |

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The Scottish Medicines Consortium (SMC) has added oral Evrysdi (risdiplam) to the list of medications available through the country’s National Health Service (NHS) at low or no cost to eligible spinal muscular atrophy (SMA) patients, SMA UK announced.

These include those 2 months and older diagnosed with SMA type 1, 2, or 3, or carrying one to four copies of the SMN2 “backup” gene if not yet showing symptoms (pre-symptomatic). The presence of that number of SMN2 copies supports the development of any of those disease types.

Evrysdi, marketed by Roche, is expected to be available through the NHS in about three months. Patients in Scotland using this daily and at-home treatment through a U.K.-wide early access scheme initiated in September 2020 are expected to be moved to NHS access.

“Today’s announcement is brilliant news for families in Scotland who are affected by SMA,” Kate Adcock, PhD, the director of research and innovation at Muscular Dystrophy UK (MDUK), said in the SMA UK announcement.

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The SMC initially opposed supporting the therapy, but a confidential discount offered by Roche and opinions expressed at a patient and clinician engagement (PACE) meeting last month helped to change the agency’s views.

At the PACE meeting, SMA patients using Evrysdi and doctors working in this disease supported its benefits. Among their points was this treatment’s potential to help patients “to be less dependent on family and carers in many ways, including needing assistance with activities of daily living and self-caring and for support during the night,” the SMC wrote in its decision document.

Adcock and Liz Ryburn, SMA UK’s support team manager, participated in the meeting.

“We’ve worked closely with SMA UK to represent the SMA community throughout this whole process and we are delighted that eligible patients in Scotland will soon be able to access [Evrysdi] on the NHS,” Adcock said.

“As someone living with SMA myself and a Trustee of SMA UK I am proud that our Scottish SMA community, our charity and MDUK have worked so well together and advocated so strongly for access to this treatment,” added Alistair Emslie-Smith, MD.

The Scottish agency’s decision comes about nine months after the therapy was approved for these patient groups in the U.K. and follows a similar recommendation by the National Institute for Health and Care Excellence (NICE) — the agency that decides which therapies are added to the NHS in England and Wales.

Patients in those two countries, as well as in Northern Ireland, have access to Evrysdi at low or no cost through a three-year managed access program, during which Roche will provide the therapy at a special, confidential price and additional data will be collected.

These data are meant to help address concerns identified by NICE during its initial evaluation, which also did not favor Evrysdi’s coverage. After evaluating the collected evidence, the agency will update its guidance on whether the therapy should be recommended for routine use through the NHS.

“We have been pleased to work collaboratively with the SMC, NHS Scotland, patient organizations and the SMA community to reach this important milestone for individuals and families affected by SMA in Scotland,” Roche stated in a letter to SMA UK.

“We are delighted to have now secured broad access across the UK to this new treatment option,” but “recognize that improving access to treatment is just one element of supporting individuals affected by SMA,” Roche added. “We are committed to continuing to work with you [SMA UK] into the future to improve the lives of everyone affected across the UK.”

Evrysdi 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. It is given as a flavored liquid by mouth or feeding tube once a day at home.

The therapy’s favorable safety and efficacy profiles were established based on data from the ongoing Phase 2/3 FIREFISH (NCT02913482) and SUNFISH (NCT02908685) trials, which involved a total of 221 patients, 1 month to 25 years old, with SMA types 1, 2, and 3.

Results showed that Evrysdi safely and significantly improved survival, breathing, swallowing, and motor milestones in type 1 infants, as well as motor function in children and young adults with types 2 and 3 disease.

Evrysdi’s clinical program also includes two global Phase 2 trials — JEWELFISH (NCT03032172) and RAINBOWFISH (NCT03779334) — that are testing the therapy in different patient groups.

Interim data 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 patients up to age 60 who had used other disease-modifying treatments more than doubled their SMN levels and showed stable motor function with Evrysdi.

RAINBOWFISH is still enrolling pre-symptomatic newborns up to 6 weeks old with a genetic diagnosis of SMA. More information can be found here.

Evrysdi was approved in the U.S. in August 2020 for adults and children ages 2 months and older with all types of SMA and in the European Union in March 2021 for the same indication as in the U.K. Similar decisions soon followed in other countries, such as Canada, Japan, and Brazil.

Based on promising RAINBOWFISH interim findings, a regulatory application requesting the therapy’s use be extended to pre-symptomatic infants younger than 2 months old is currently under priority review in the U.S.