Vertex Pharmaceuticals on Monday won approval for its fourth treatment for cystic fibrosis, a combination drug that extends a new form of therapy to 90% of CF patients.
The drug will be marketed as Trikafta. The list price is $311,000 per year — the same as one of Vertex’s earlier CF treatments.
In a statement, acting Food and Drug Administration Commissioner Ned Sharpless said the approval makes “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.”
Cystic fibrosis is a progressive disease that damages the lungs and is caused by mutations in the CFTR gene. In patients with the disease, mucus clogs up the lungs and leads to frequent infections. Vertex’s drugs work by tweaking the faulty protein made by the gene to allow it to function properly.
The new drug is a triple combination regimen and was approved for patients 12 years and up with a type of genetic error called an F508del mutation. It is estimated to cover 90% of the 27,000 CF patients in the country, according to the FDA.
Earlier drugs from the Boston-based Vertex — the first CF drugs to target the underlying cause of the disease — worked on mutations held by smaller segments of CF patient population. The first, Kalydeco, was approved in 2012.
Vertex drugs already on the market have been transformational for some patients whose mutations the therapies target, though they have come under scrutiny for their prices. Health authorities in Europe have argued that the drugs do not offer adequate benefits to earn their costs.
In clinical trials, patients who received Trikafta experienced significant improvements in lung function.
The approval also helps Vertex achieve its goal of making available treatments that cover the large majority of CF-causing mutations. As Vertex’s CF work has crested, it has been diversifying its disease targets. It has turned to synthetic messenger RNA and the genome editor CRISPR as it works on future therapies, and it is acquiring Semma Therapeutics, which is developing on a stem cell-based diabetes treatment.
About the Author Reprints
My great niece started on this new drug a couple weeks ago. It is a Godsend for sure. Her friend, who is much worse than her, started on the drug at the same time and literally had mucous starting to run from everywhere after taking it. It starts working almost immediately, at least for these two little girls. It doesn’t treat the symptoms, it treats the cause! Thank you so much for this wonderful drug!
Hi, my son is diagnosed with CF. He is 8 months old. Is this new drug allowed for this age
What is the hope for children under the age of 12?
Triple drug needed in uk .
we dont want to await another 4 years Nhs verses nice and vertex ,
Please keep me posted.
My granddaughter has CF and we have prayed for this day. Please keep me posted on when it will be available and what the patient has to do to receive it.
Would this medicine help people with COPD.
I was thinking the same thing
Any positive resperatory advancement is a good thing . Good luck to those who are fortunate enough to try this nebulizer
Jeffrey Leiden man of the year award..
he keeps to his words
Im julian leat age 47 cf. dont drink or smoke my life away .
at last a drug for us adults thanks.
but still i nothing uk await the drugs triple takes years i think .
also my hosptial still awaits the other 2 drugs . when nhs uk allows
This is a prayer answered. My grandson is 42 years old, married with twin girls who are 4-years-old. If and when he can be treated with this protocol, it will help him to have a better and more comfortable life. Thank you so much for your wonderful research. I donate a little every year. I wish I could do so much more.
Bless each of you who have developed this medication.
My daughter has CF and this is a great announcement for her. I am so excited that the FDA has taken this step and that Vertex has continued in its research.
Do u know if this medication is helping some one like my daughter with one delta 508 and one none sence mutation
This is great news!