Vertex Pharmaceuticals announced in late August that the U.S. FDA accepted applications of tezacaftor/ivacaftor for patients with cystic fibrosis (CF).
Specifically, this combination treatment can be used in patients ages 12 and older who have CF caused by the F508del mutation.
Cystic fibrosis, or CF for short, is a rare genetic condition that causes progressive respiratory and digestive system damage. Current treatment aims to prevent or control infections, but there is currently no cure for the disease. To learn more about CF from Patient Worthy, click here.
However, Vertex Pharmaceuticals is working tirelessly to make sure every CF patient has access to adequate treatment. The tezacaftor/ivacaftor treatment combination would actually become the company’s third CF medicine, and it could potentially work for a significant group of patients who are lacking in treatment options.
Teacaftor works to mitigate the processing defect in mutated CF cells, while ivacaftor works to enhance the function of the protein itself. In this way, both drugs work alongside each other to treat the biological root cause of the disease.
Because of their huge potential, tezacaftor/ivacaftor were granted Priority Review designation. This label shortens the FDA’s total review time by four months, and tezacaftor/ivacaftor was also granted Breakthrough Therapy Designation to further expedite its development and analysis.
In addition to the United States, this combination treatment is also being reviewed in the European Union; a final decision is likely within a few months.
A formal action date by the U.S. FDA for this treatment has been set for February 28, 2018, but the submission is supported by positive results from two global Phase 3 studies, so Vertex Pharmaceuticals is optimistic.
Hopefully once approved, Vertex can start with the reimbursement approval process in each country, and things will really start to get going.
In the meantime, if you want to learn more about the ongoing application process for tezacaftor/ivacaftor, click here!