Therapy Areas: Hereditary Disorders
Vertex Receives a Positive PBAC Recommendation for Reimbursement of Orkambi to Treat Australians Ages Six and Over with Cystic Fibrosis and Two Copies of the F508del Mutation
22 August 2018 - - US-based biotechnology company Vertex's Vertex Pharmaceuticals (Europe) Ltd. business has received the Pharmaceutical Benefits Advisory Committee's recommendation for Orkambi (lumacaftor/ivacaftor) to be listed on the Pharmaceutical Benefits Scheme in Australia for people ages six and over with cystic fibrosis who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator gene, the company said.

Lumacaftor/ivacaftor is the first medicine to treat the underlying cause of CF in people ages six and older who have two copies of the F508del mutation.

Many thousands of patients worldwide are already receiving lumacaftor/ivacaftor in countries where it is reimbursed including Austria, Denmark, Germany, Ireland, Italy, Sweden, the Netherlands and the US
Vertex remains steadfast in its commitment to ensuring people living with CF have rapid access to its medicines.

Cystic fibrosis is a rare, life-shortening genetic disease affecting approximately 75,000 people including countries within North America and Europe, as well as Australia.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes -- one from each parent -- to have CF.

There are approximately 2,000 known mutations in the CFTRgene. Some of these mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working or too few CFTR proteins at the cell surface.

The defective function or absence of CFTR protein results in poor flow of salt and water into and out of the cell in a number of organs. In the lungs, this leads to the build-up 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 mid-to-late 20s.

In people with two copies of the F508del mutation, the CFTR protein is not processed and trafficked normally within the cell, resulting in little-to-no CFTR protein at the cell surface. Patients with two copies of the F508del mutation are easily identified by a simple genetic test.

Orkambi is a combination of lumacaftor, which is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del-CFTR protein, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface. Lumacaftor/ivacaftor is available as tablets and is typically taken twice per day.

The PBAC is an independent, expert statutory body which reviews submissions from pharmaceutical companies regarding new medicines to be considered for subsidy within the Pharmaceutical Benefits Scheme. The PBAC makes recommendations to the government as to whether or not a medicine should be subsidized and the Health Minister cannot add a medicine to the PBS without prior recommendation from the PBAC.

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

In addition to clinical development programs in CF, Vertex has more than a dozen ongoing research programs focused on the underlying mechanisms of other serious diseases.

Founded in 1989 in Cambridge, Mass., Vertex's headquarters is now located in Boston's Innovation District. Today, the company has research and development sites and commercial offices in the United States, Europe, Canada, Australia and Brazil.

Vertex is consistently recognized as one of the industry's top places to work, including being named to Sciencemagazine's Top Employers in the life sciences ranking for eight years in a row.
Login
Username:

Password:


Related Headlines