Therapy Areas: Diabetes
Health Canada Accepts Vertex New Drug Submission for Investigational Triple Combination Medicine for the Treatment of Cystic Fibrosis
28 December 2020 - - US-based biotechnology company Vertex Pharmaceuticals Inc's (NASDAQ: VRTX) New Drug Submission for Trikafta, Vertex's investigational triple combination medicine, has been accepted for Priority Review by Health Canada for the treatment of cystic fibrosis in people ages 12 years and older, the company said.

With Priority Review, the conventional review timeline of 300 days is reduced to 180 days. The expected approval target by Health Canada is in the first half of 2021.

Cystic fibrosis is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract.

CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene.

Children must inherit two defective CFTR genes -- one from each parent, to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation.

These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface.

The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs.

In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death.

The median age of death is in the early 30s.

Vertex is a global biotechnology company that invests in scientific innovation to create medicines for people with serious diseases.

The company has multiple approved medicines that treat cystic fibrosis -- a rare, life- threatening genetic disease, and has several ongoing clinical and research programs in CF.

Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases.

In addition, Vertex has a pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.


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