Vertex Pharmaceuticals announced the submission of a New Drug Application (NDA) to the FDA for the VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination regimen for treating Cystic Fibrosis (CF).
This can be a really big development for the CF community!
What is CF?
Cystic fibrosis is a genetic condition that causes progressive respiratory and digestive system damage. People with CF has thick, sticky mucus, versus “healthy” slippery mucus, which builds up in their system.
That build-up can clog airways, trapping bacteria, leading to breathing problems, frequent infections, lung damage and respiratory failure. Additionally, the thick mucus prevents the release of digestive enzymes, making it difficult for patients to absorb necessary nutrients.
From Breakthrough Therapy to Priority Review
The NDA is supported by positive results from two global Phase 3 studies; which showed statistically significant improvements in lung function!
“The submission of the NDA is a major step toward our goal of bringing this medicine to the largest remaining group of people with CF that still do not have an approved Vertex medicine, as well as toward providing significantly enhanced benefits to patients with two F508del mutations,” said Reshma Kewalramani, M.D., Executive Vice President and Chief Medical Officer at Vertex. “We will continue working with the FDA as they review the NDA and look forward to the potential of this triple combination regimen becoming a new treatment option for people with CF.”
This potentially critical new treatment regimen was granted ‘Breakthrough Therapy’ designation in May 2018. ‘Breakthrough Therapy’ designation is a process designed to speed-up the development of drugs intended to treat a serious condition and also already has existing clinical evidence demonstrating substantial improvement.
This NDA also includes a request for a ‘Priority Review’, which would shorten the FDA’s review of this new drug application to eight months (versus the standard 12 months).
So Vertex is very keen on pushing this treatment regimen through the FDA’s pipeline; signaling great confidence that the drug regimen can have great clinical effect.
We’ll keep a close eye on its progress!