It appears there has been a revolutionary breakthrough in the treatment of cystic fibrosis. A new drug called Trikafta is producing dramatic results.
To date, only a handful of medications have been found that combat this disease, and the improvements afforded are modest. But experts in the field are calling Trikafta a once-in-a-generation game changer.
Cystic fibrosis is an inherited disorder that causes a buildup of mucus in the lungs, eventually killing the patient. Average life expectancy with this disease is 52.1 years in Canada, 10 years longer than the U.S. average. The difference is due, in part, to our universal health-care system.
One of the difficulties in finding a treatment is that there are numerous variants of the disorder, and some of the drugs currently available are effective on only small groups of patients.
That is about to change. The manufacturer of Trikafta, Vertex Pharmaceuticals, discovered that if three existing medications were combined in one regimen, the results far outmatched anything previously known.
In two separate clinical trials, the improvements in lung function were 10 per cent and 13.8 per cent respectively. Better still, 90 per cent of patients with cystic fibrosis can take Trikafta.
We’ll look more closely at these results in a moment, but first a note of caution, especially for readers who have this disease.
Vertex has said the drug will be priced at $435,000 per patient per year. When Orkambi, an earlier, less effective CF drug, was priced at $250,000, Canada’s Drug Expert Committee recommended against funding it.
The committee argued its cost far outweighed marginal improvements in lung function, which averaged two to three per cent, although some patients did better.
Whether to fund Trikafta will be a tougher call. The benefits far exceed those of Orkambi.
Yet there are 376 cystic fibrosis patients in B.C. who could potentially gain from taking the drug (though the degree of benefit would vary greatly). If the province funded every patient in this group, the cost would be north of $130 million annually. That’s a huge obstacle.
Now it’s possible Canada could negotiate a lower price; state-level authorities in the U.S. say the drug is 70 per cent overpriced.
As well, if B.C. did cover the drug, it could do so on a case-by-case basis, selecting patients who had most to gain. Even so, we’re still talking awfully big numbers.
Lastly, the company hasn’t yet approached Health Canada to ask for a licence, and past experience shows that once a licence is granted, it takes two to five years for a new drug to reach pharmacy shelves.
It’s worth noting the U.S. Food and Drug Administration rushed Trikafta through the approval process in record time. If only our own licensing authorities could speed up their game.
Nevertheless, despite all of these cautionary notes, it is clear this drug represents a huge advance in the treatment of a thoroughly dreadful disease. According to the company (and you have to take this kind of assurance with a grain of salt), patients who participated in the clinical trials had a 63 per cent reduction in lung damage — an astonishing result.
On a scale of 1 to 100, those same patients also reported a 20 point enhancement in their quality of life, meaning they were able to resume activities that had been lost as the disease progressed. Some experienced fundamental improvements in their daily routines. That, too, is remarkable.
Better still, Trikafta not only boosts lung performance, it also improves the function of various major organs, among them the liver, pancreas and various components of the digestive system. That’s exciting because it points to a broader therapeutic value than had been anticipated. As well, the drug helps diminish unwanted weight gain, which might play a role in preventing the onset of type 2 diabetes.
What comes next remains to be seen. But it’s clear researchers are closing in on the underlying biology of this disease.
In that respect, Trikafta may represent a critical step on the way to an outright cure. At any rate, there is reason to hope so.
