Man with degenerative disease pleads for Manitoba to cover treatment while it asks drug agency to review

A man with a muscle-wasting disease is pleased Manitoba is asking a federal agency to reconsider its opposition to a treatment for adults over 25, but he argues that time is running out in his case.

Jeremy Bray, who has Type 2 spinal muscular atrophy — a rare degenerative disease that has gradually robbed him of the ability to move his muscles — says he cannot wait for Canada's Drug Agency (CDA) to potentially revise its recommendation.

"I hope that the province commits to an emergency measure that provides me with treatment as soon as possible, because we really don't know how long the CDA process should take," Bray said Tuesday afternoon, after meeting with Health Minister Uzoma Asagwara at the Manitoba Legislature.

The 29-year-old data consultant from Rivers, Man., has lost the capacity to move his head, arms, hands or legs. Now, he's down to the use of just one thumb and his mouth, but those muscles are slowly getting weaker.

There is no cure for spinal muscular atrophy, but treatments can stop its progression and often lead to some improvement.

However, Manitoba has refused to pay for the hugely expensive treatment, with a price tag estimated at $300,000 in the first year. The province cites guidance from Canada's Drug Agency that the drug — risdiplam, sold under the brand name Evrysdi — isn't recommended for adults over 25 because people from that age group haven't been involved in any clinical tests.

On Tuesday, Asagwara told Bray in a meeting they've asked the agency — which is responsible for co-ordinating drug policy among provinces and territories — to conduct an "urgent review" and reconsider its recommendation opposing treatment for adults of a certain age.

In its letter, dated Monday, the province states it has received a summary of "real-world evidence" from a neurologist in Manitoba around treatments, primarily risdiplam, for adults aged 25 and over.

"As such, we are formally requesting an urgent review of new data … for the use of these medications in adult patients," the letter reads.

The drug agency didn't immediately respond to a request for comment Tuesday, but Asagwara said in an interview the organization has already responded to the province's letter, saying "they understand that this is actually a really important step" and would have more to say later.

Asagwara, however, said the province isn't willing to overrule the agency in the meantime.

"In Manitoba, we have a longstanding practice of making sure that we follow those guidelines and recommendations because they come directly from experts," the minister said.

"What we've done now is taken that additional step to say, 'Can they look at real-world evidence? Can they look at cases in the real-world context and see if there's new recommendations that should be issued?'"

Manitoba pays for risdiplam for patients under the age of 25.