“We went from being able to treat no patients to being able to treat nearly all patients in seven years.”
90% of cystic fibrosis (CF) patients now have a new treatment option thanks to recent FDA approval for a combination therapy created by Vertex Pharmaceuticals. The therapy is a combination of three different drugs. It’s called Trikafta. This medication was created to target F508del gene mutations which occur in about 90% of all CF patients.
Development began for this combination therapy about four years ago. After its application was submitted to the FDA, the FDA announced their deadline to make a decision on the treatment was March 19, 2020. That means this approval has come 5 months early.
Ultimately, this means it can be in the hands of patients sooner. Researchers and the FDA understood the dire need this patient community had for a new, effective option.
Unfortunately, this medication isn’t cheap. Vertex’s price tag for the drug annually is 311,000 dollars. But the fact that it not only will help patients but also addresses the underlying cause of the disease is huge.
Vertex has already been in discussion with European drug regulators, with the goal to get the drug approved in Europe as well. So far, it has not yet received approval due to a dispute about insurance reimbursement. But Vertex is not giving up.
The 10%
So what about the other 10% of CF patients that won’t benefit from this drug? Vertex isn’t giving up on their cystic fibrosis investigation just because of this success. They are currently investigating other options for this remaining segment of the population. These investigations include gene editing, RNA-targeting therapies, and gene therapies.
However, despite this work, Vertex estimates an option for this part of the community is still 10 or 15 years away.
Nonetheless, the success of Trikafta has brought new hope to CF patients, and we all hope to see more developments in the near future.
You can read more about this approval here.
