The U.S. Food and Drug Administration (FDA) has approved Trikafta (elexacaftor/ivacaftor/tezacaftor), a new treatment plan for cystic fibrosis patients. This is the first triple combination therapy for the patients suffering from cystic fibrosis mutation.
It is only approved for patients who are above 12 years and older having cystic fibrosis with at least one F508del mutation in the CFTR gene. This makes roughly 90% of the cystic fibrosis patients that means this new treatment will improve the condition for 90% cases
FDA is open to find new treatment plans
FDA is looking forward to find new therapies for persistent diseases while maintaining its standards. This new treatment plan for cystic fibrosis is a landmark approval, which is a novel treatment for those who had no options before.
During the past years, there were a number of breakthrough therapies in front of the FDA for approval. However, there was no approved treatment for it. That is why patients were bound to use only the available options that include Priority Review, Fast Track, and orphan drug designation. All these options were helpful to get this advanced therapy that is available today.
What is cystic fibrosis?
Cystic fibrosis, rare and progressive diseases that can even take the life of the patient. It causes the formation of thick mucus in the lungs, digestive tract, and other parts of the patient’s body. After this, it leads to severe respiratory and digestive problems and also increases the risk of infections. Cystic fibrosis is also a risk factor to develop diabetes in adults.
The only reason behind all this is a defective protein, which is CFTR gene. This malfunctioning results from mutations in this gene. While there are already some 2000 mutations of the CFTR gene possible, the most common of all these is the F508del mutation.
How does Trikafta help in cystic fibrosis?
This new treatment plan Trikafta uses a combination of three drugs that target the malfunctioning CFTR protein and make it work normal again. Among all the available therapies for this defective protein, many other types of mutations are ineligible for treatment. But Trikafta is the first-ever treatment which works best on patients with at least one F508del mutation. This may sound less but actually it makes up to 90% of the population with cystic fibrosis. However, this treatment is only suitable for 12 years old and above. It is not suitable for underage children.
Trikafta passed two trials before its launch as a treatment plan for patients with cystic fibrosis aged 12 years and older. The first trial was a randomized, double-blind, placebo-controlled trial that used 403 patients with F508del mutation for 24-weeks. The second trial was on 107 participants with a randomized, double-blind, active-controlled approach for four weeks.
The FDA finally granted this application a Priority Review this October. Trikafta now shares orphan drug designation that encourages to develop new treatment drugs for rare, chronic diseases like cystic fibrosis.
The estimated review goal date was March 19, 2020, but the FDA reviewed and approved Trikafta in only three months. This approval is granted to Vertex Pharmaceuticals Incorporated. They will get a Rare Pediatric Disease Priority Review Voucher for this remarkable treatment therapy.