Therapy Areas: Hereditary Disorders
Vertex Receives Australian TGA Approval for Trikafta to Treat People with Cystic Fibrosis Ages 12 Years and Older Who Have At Least One F508del Mutation
25 March 2021 - - The Australian Therapeutic Goods Administration has approved the use of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for people with cystic fibrosis ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator gene, the most common CF-causing mutation worldwide, US-based biotechnology company Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) said.

Up to 90% of people living with CF worldwide have at least one F508del mutation.

CF affects approximately 3,500 people in Australia. It is caused by a defective and/or missing CFTR protein resulting from mutations in the CFTR gene.

The TGA approval of Trikafta was based on the results of four global Phase 3 studies, which included multiple trial sites and patients from Australia.

Cystic Fibrosis is a rare, life-shortening genetic disease affecting more than 80,000 people globally. 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.

In Australia, Trikafta is indicated for the treatment of cystic fibrosis in patients ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator gene.

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

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

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

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America.
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