A branch of the Canadian Agency for Drugs and Technologies in Health (CADTH) is recommending that Evrysdi (risdiplam) be added to public health plans across Canada, making it available to spinal muscular atrophy (SMA) patients at low or no cost between 2 months and 25 years of age, provided certain conditions are met.

Among the conditions required for Evrysdi reimbursement are that all eligible patients have a genetic SMA diagnosis, and those between 2 and 7 months old have two or three copies of the SMN2 “backup” gene, while those 8 months to 25 years old be unable to walk without assistance (nonambulatory) and have a like number of SMN2 copies.

“We are pleased to see Canadian regulatory bodies are recognizing the value of innovative medicines for this patient group,” Susi Vander Wyk, the executive director of Cure SMA Canada, said in a press release. “This is a positive step towards filling in gaps where the end goal is achieving access to treatment for all SMA patients.”

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Other requirements in the recommendation from CADTH”s Canadian Drug Expert Committee include:

  • Eligible patients are those with 5q SMA gene deletion, as confirmed by a genetic test. 5q SMA refers to cases in which both copies of the SMN1 gene — one inherited from each biological parent — are mutated, which account for about 95% of SMA cases. Patients categorized as compound heterozygous, meaning they have a mutation in one gene copy and a different mutation in the other copy, are also eligible.
  • Patients do not need permanent invasive ventilation.
  • Patients demonstrate treatment benefit — attaining or maintaining motor milestones — in two assessments taken at time of the program’s initial annual renewal (after 12 months of Evrysdi use), each done within six weeks of that renewal date.
  •  Roche, which markets Evrysdi, lowers its price in the Canadian healthcare market to that of Spinraza (nusinersen), which the committee considers a comparable therapy. “There is no evidence to suggest that risdiplam should be priced higher than nusinersen,” it stated.
  • Evrysdi should not be given in combination with either of its other approved disease-modifying treatments, Spinraza or the gene therapy Zolgensma.

The recommendation will now be evaluated by provincial governments, which will make the final decision on public reimbursement. Quebec’s plan is not among those covered in the CADTH document, as its local government already has a plan in place.

“These recommendations are such an important step forward for the SMA community,” said Colleen O’Connell, an assistant professor in rehabilitation medicine at Dalhousie University, in Halifax, Nova Scotia. “I know this represents hope for so many who live with this disease. Moving forward, we must continue to study how such treatments help our patients throughout their lives, and particularly by treating them before the muscles become paralyzed.”

Evrysdi acts through the SMN2 gene to boost the production of working SMN protein, which is impaired in SMA. It was developed by Roche and its subsidiary Genentech, in collaboration with PTC Therapeutics and the SMA Foundation. Given as strawberry-flavored liquid, Evrysdi is administered daily at home by mouth or feeding tube.

Spinraza, developed and marketed by Biogen, also works through SMN2, but is administered through a spinal canal (intrathecal) injection every four months after an initial loading period.

According to Roche, patients in Canada now wait more than 450 days (over one year) longer than those in the U.S. and Europe to access new medicines.

“We are pleased to see Canadian regulatory bodies are recognizing the value of innovative medicines for this patient group,”  Vander Wyk said. “Rapid access to treatments for SMA are vital to Canadians living with the condition. We look forward to a future when all SMA patients are able to access treatment in a timely manner, so the effects of this condition are halted.”

While the CADTH recommendation will allow more SMA patients in that country to access Evrysdi, its required clinical criteria imposes limitations, particularly for patients over age 25 and those younger but able to walk independently.

“These recommendations will be disadvantageous for Canadian SMA patients, many who have no other treatment options,” said Durhane Wong-Rieger, president and CEO of the Canadian Organization for Rare Disorders (CORD).

“In stark contrast, many other countries, as well as INESSS [National Institute for Excellence in Health and Social Services] in Quebec, have approved broader access in adult patients,” Wong-Rieger said. “From the perspective of CORD, as Canada moves toward implementing the National Rare Disease Drug Strategy, our hope is that these Health Technology Assessments in Canada are ready to contribute to innovative access programs for innovative therapies.”

CADTH’s recommendation was based on positive results from the ongoing Phase 2/3 FIREFISH (NCT02913482) and SUNFISH (NCT02908685) trials, both evaluating Evrysdi’s safety and effectiveness in patients 1 month to 25 years old with SMA types 1, 2, and 3.

Top-line SUNFISH results showed that Evrysdi significantly improved or stabilized motor function among children and young adults, ages 2 to 25, with SMA type 2 or 3. The improvements were either sustained or increased in the second year of treatment.

Two-year data from the FIREFISH trial, evaluating Evrysdi in infants ages 1–7 months with type 1 SMA, indicated that gains made in the first year were maintained in the second year.

Evrysdi was approved by Health Canada for at-home treatment of SMA in people ages 2 months and older in April. Its annual costs for infants and toddlers, ages 2 to 24 months, are expected to reach CA$93,456 per patient, and an estimated CA$354,000 for those older than 24 months (weighing 20 kg or more), CADTH reported.

In the U.S., Evrysdi’s list price — which generally is not the treatment’s cost to patients — is set at a maximum of $340,000 a year for patients weighing 44 lbs or more (20 kg); before that, price is based on a child’s weight. Spinraza carries a U.S. list price of $750,000 for a first year of treatment, and $375,000 for each subsequent year; Zolgensma, a one-time treatment by Novartis, is listed at $2.125 million.

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