Dylan Stoll | Health Editor
Featured Image: Those who suffer from cystic fibrosis feel like they’re drowning on a daily basis. | Courtesy of Pixabay
A new cystic fibrosis treatment drug, referred to as Trikafta, was approved recently by the FDA. The drug, dubbed by one patient as a “miracle,” is intended to be used by individuals aged 12 years and older, and with at least one mutation in the F508del cystic fibrosis transmembrane conductance regulator gene (CFTR).
“At the FDA, we’re consistently looking for ways to help speed the development of new therapies for complex diseases, while maintaining our high standards of review,” said acting FDA Commissioner Ned Sharpless in a press release issued October 21.
“Today’s landmark approval is a testament to these efforts, making a novel treatment available to most cystic fibrosis patients, including adolescents, who previously had no options and giving others in the cystic fibrosis community access to an additional effective therapy.”
The disease itself is most commonly the result of an aforementioned mutation in the F50del CFTR gene. This gene is responsible for the production of a protein necessary for the expulsion of water from cells; when the gene is mutated, the protein is ineffective in its function, thus causing a buildup of mucus in the lungs, digestive tract, and other areas of the body.
In effect, those who suffer from this illness feel as if they are drowning from the inside as they lose approximately two per cent lung function on a yearly basis.
The average lifespan of a person afflicted with cystic fibrosis is 37 years, with their death often being due to lung complications. Those who have the disease are still able to lead productive and happy lives, but it is required that they take consistent medications such as antibiotics to treat chest infections, mucus thinners, and anti-inflammatories. Special techniques and devices are also encouraged to assist in clearing the excess mucus of the lungs.
Though all these treatment options are available, it is of no surprise to anyone that an actual cure would be preferable. The Trikafta treatment is a monumental step towards that goal, requiring a simple combination of three drugs.
“It’s huge. It’s absolutely huge for this community,” said John Wallenburg, chief scientific officer with Cystic Fibrosis Canada. “In terms of progress, scientifically and medically, this is fantastic.”
Unfortunately, Trikafta has not yet been made available for use by Canadians, which is a decision requiring a submission of approval by the drug’s developer. And of course, money has a considerable pull in this decision.
That being said, Canada is considered a top-tier candidate; in other words, we pay big for life-saving drugs.
“We’ve usually been pretty rapid in terms of at least seeing a company submit something for regulatory approval,” said Wallenburg.
