British Columbia Will Pick Up Cost of Evrysdi for SMA Patients There

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by Mary Chapman |

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The provincial government of British Columbia will cover the cost of Evrysdi (risdiplam), the oral spinal muscular atrophy (SMA) therapy approved by Health Canada in April for the at-home treatment of eligible patients age 2 months and older.

Evrysdi’s annual costs are reported to be CA$93,456 (about $73,310) for infants and toddlers ages 2 to 24 months, and CA$354,000 for those older than 24 months (weighing at least 20 kg).

This decision followed negotiations with the treatment’s manufacturer and the pan-Canadian Pharmaceutical Alliance, a group of federal, provincial, and territorial governments working together on public treatment plan initiatives. Evrysdi is marketed by Roche and its subsidiary Genentech; was developed by those companies in collaboration with PTC Therapeutics and the SMA Foundation.

For qualified patients, funding will be provided by the Ministry of Health through the province’s Expensive Drugs for Rare Diseases (EDRD) process.

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“EDRDs are considered non-benefits, but some drugs and patients may be eligible on a case-by-case, last resort basis,” the government of British Columbia stated in a press release. “Patients will need to discuss their eligibility with their healthcare provider.”

Currently, about 23 children and 10 adults are thought to be eligible for Evrysdi in British Columbia. Its Ministry of Health also funds two other approved SMA treatments, Spinraza (by Biogen) and Zolgensma (by Novartis).

A branch of the Canadian Agency for Drugs and Technologies in Health (CADTH) recommended last year that Evrysdi be added to public health plans across Canada and made available at low or no cost to patients between 2 months and 25 years old, provided certain conditions are met, including a genetic SMA diagnosis and demonstration of treatment benefit.

The recommendation was based on positive results from the ongoing FIREFISH (NCT02913482) and SUNFISH (NCT02908685) clinical trials, which are assessing Evrysdi’s safety and effectiveness in people 1 month to 25 years old with SMA types 1, 2, and 3.

Following CADTH’s recommendation, it was left to provincial governments to decide on public reimbursement. Quebec’s local government was exempt from the CADTH document, because it already had such a plan in place.

Evrysdi is a daily treatment, a flavored liquid that’s administered at home by mouth or feeding tube. It addresses the underlying cause of SMA, most often a mutation in the SMN1 gene that causes little or no SMN protein to be produced. The disease is characterized by progressive muscle weakness and atrophy, mainly affecting motor function.

In the U.S., which approved Evrysdi in 2020 for adults, children, and infants starting at 2 months old, the list price is capped at $340,000 annually, a price that comes into effect when a patient’s weight reaches 44 pounds (20 kg). Before then, pricing is based on a child’s weight. The therapy’s list price is generally not its cost for patients.