Vertex Announces Long-Term Access Agreement in Sweden for Cystic Fibrosis Medicine ORKAMBI® (lumacaftor/ivacaftor)
- The agreement allows for reimbursement of ORKAMBI for people with
cystic fibrosis who have two copies of the F508del mutation from
- A framework for assessment and access to our future cystic fibrosis medicines is included as part of the agreement -
“We are delighted that people with CF in
CF is a devastating rare disease that causes continuous damage to
multiple organs from birth. In the lungs, a build-up of sticky mucus
causes progressive and permanent damage, severe infections and
ultimately premature death. In addition to
About Cystic Fibrosis
Cystic fibrosis is a rare, life-shortening genetic disease affecting
approximately 75,000 people in
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 CFTR gene. 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.
About ORKAMBI® (lumacaftor/ivacaftor) and the F508del mutation
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.
For complete product information, please see the Summary of Product Characteristics that can be found on www.ema.europa.eu.
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
For additional information and the latest updates from the company, please visit www.vrtx.com.
Vertex Pharmaceuticals Incorporated
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