Despite having made a name in the pharmaceutical industry, Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) has continued to invest in scientific innovation and transformative medicines for people with serious diseases. With multiple sites and commercial offices in Latin and North America, the company acknowledges that even though it is rare, cystic fibrosis (CF) can be a life-threatening genetic disease.
There are more than 75,000 people globally living with Cystic Fibrosis (CF). Earlier on, Vertex had submitted three supplemental New Drug Applications (sNDAs) to the FDA. They are for TRIKAFTA®, SYMDEKO®, and KALYDECO. It was seeking for the expansion of their labels to include additional rare CFTR mutations. The FDA has since accepted the three sNDAs.
Transforming the Lives of Everyone Living with CF
The approval of the regulatory submissions of the sNDAs was based on data from an in vitro cell assay, which demonstrated a positive response of rare CFTR mutations. The good news is that the expansion of labels will give non-eligible candidates of the treatments access to them. Vertex says that more than 600 non –eligible candidates are expected to gain from CFTR modulators. Another 1,100 people with CF will also gain from the new therapeutic options.
The Global Research and Chief Scientific Officer of Vertex, David Altshuler, feels that the new milestone is a dream come true. He said, “We have spent the last 20 years discovering, developing and bringing new medicines to thousands of people with CF… we have been able to generate data providing evidence…of treating the underlying cause of their disease with CFTR modulators.”
The Vast Majority of All People with CF Have At Least One F508del Mutation
The three medicines are expected to be effective on various or additional rare CFTR mutations. However, Vertex has not disclosed which rare mutations would be taken care of by the medicines. On the other hand, many people with CF tend to have at least one F508del mutation even though a mutation is determined through a genetic test or genotyping test.
More often, the cause of CF is usually a defective and/or missing CFTR protein. The progressive disease affects multiple organs, including sweat glands, the reproductive tract, the liver, the GI tract, the pancreas, and the lungs.