It's what Cystic Fibrosis (CF) patients and advocates have been fighting for — for more than a year.
Cystic Fibrosis Canada announced on Monday that it understands the "life-changing" drug Trikafta is on its way to availability in Canada.
For 22-year-old Windsorite Aalaya Fleming, who lives with the disease, this news is "amazing."
"Like a little kid opening a present on Christmas," she said, describing the moment she learned the news.
Trikafta has been hailed a game changer for those living with the progressive, life-threatening, genetic disease, which mainly affects the digestive system and the lungs.
The manufacturer of the drug Vertex Pharmaceuticals told CBC in a statement that it has "made the decision to move forward with our new CF medicines in Canada."
However, Health Canada says it has yet to receive a new drug submission from the manufacturer to market Trikafta in Canada.
In an e-mailed statement to CBC, the federal agency said there are currently no cystic fibrosis products under review, and it cannot release any information around potential submissions prior to an official filing.
But Kim Steele, the director of government relations for Cystic Fibrosis Canada, says she feels confident about what it announced.
"We wouldn't have that type of communication unless something was to happen," Steele said.
"This is very tremendous news for our community."
Community is 'fed up' with the wait
Cystic Fibrosis is caused by a genetic mutation that affects protein. While previous methods worked to address the symptoms of cystic fibrosis, Trikafta binds to the protein so that it can function forever.
The medicine has the potential to treat 90 per cent of people with cystic fibrosis, and increase the median age of survival by nine years, research has shown.
The drug was approved by the U.S. federal drug agency in October of last year, and the European Medicines Agency's human medicines committee recommended granting market access to the drug in the United Kingdom earlier this summer, but to date, it has not been approved in Canada.
"The road to here has really been our community being quite frankly fed up," Steele said.
"We're losing people. There are medicines that could help slow the progression of the disease, and for some, it seems to be halting the disease ... So our community cannot just sit idly by while we watch other countries get this."
Fleming, who is a St. Clair College student and a liaison with CF Canada, agreed that it shouldn't have taken this long for the process to start.
"It's kind of heartbreaking," she said.
Concerns over pricing guidelines
Right now, the drug is only accessible in Canada through the federal Special Access Programme, which grants non-marked drugs when other therapies have failed or are unavailable. Health Canada stated that as of this month 160 Canadians had qualified for it through the program.
But according to Steele, that program helps only the sickest patients. While that's important, she said the drug should be accessible to everyone who needs it, so that it can prevent others from getting extremely sick.
According to CF Canada, the manufacturer had not previously submitted an application to bring the drug to Canada over concerns and uncertainty around the changes to the Patented Medicine Prices Review Board (PMPRB). The new drug regulations are aimed at lowering drug costs.
Trikafta costs roughly $300,000 US a year.
Vertex Pharmaceuticals said in its statement to CBC that the decision to move forward with medicines in Canada came after reviewing the final PMPRB guidelines.
The company said that it's still "genuinely concerned" that the guidelines might impact access for Canadians to new innovative medicines in the future, adding that it continues to share its concerns with the federal government.
With the new pricing guidelines in place, Steele, said it's not clear yet how pricing will work if the drug is approved in Canada.
"We have asked for clarification from the manufacturer and Health Canada," she said.
Steele also hopes to see Trikafta's application for approval and pricing review expedited, so that it can be approved within the next year.
CF Canada has also said that in a recent meeting with the Federal Minister of Health, a commitment was made to fast track Trikafta through Health Canada for approval and pricing review.
Minister Patty Hajdu made that same commitment during Question Period on Friday.
"We have had promising conversations with Vertex and we stand by, ready to rapidly review the drug using evidence from other regulatory bodies," she said, adding that she encourages Vertex to accelerate its work with Health Canada.
Meanwhile, Fleming is just excited for the moment when she hopes to see Trikafta officially approved by Health Canada — and she looks forward to the ways in which she expect the drug to improve her day-to-day life.