Vertex Announces European Authorization for Third Cystic Fibrosis Medicine SYMKEVI® (tezacaftor/ivacaftor), to be used in combination with ivacaftor (KALYDECO®), for People with CF Aged 12 and Older with Certain Mutations in the CFTR gene
- A new treatment option for patients with two copies of the F508del mutation, the most common mutation in cystic fibrosis -
- First medicine in the EU to treat the CFTR protein defect in patients who have one copy of the F508del mutation and one copy of one of 14 mutations that result in residual CFTR activity -
“The authorization of tezacaftor/ivacaftor in combination with ivacaftor
is welcome news for European CF patients, their families and everyone
involved in their treatment and care. This new medicine is especially
important for patients with residual function mutations and those who do
not tolerate ORKAMBI® (lumacaftor/ivacaftor),” said
The EU Marketing Authorization was based on results from two pivotal
Phase 3 studies, EVOLVE and EXPAND, published in the
“Today marks an important milestone for many CF patients in
Tezacaftor/ivacaftor in combination with ivacaftor was approved by the
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 tezacaftor/ivacaftor and ivacaftor
Some mutations result in CFTR protein that is not processed or folded normally within the cell, and that generally does not reach the cell surface. Tezacaftor is designed to address the trafficking and processing defect of the CFTR protein to enable it to reach the cell surface where ivacaftor can increase the amount of time the protein stays open.
For complete product information, please see the Summary of Product Characteristics that can be found on www.ema.europa.eu once posted.
About EVOLVE and EXPAND
Data from the two Phase 3 studies EVOLVE and EXPAND were published in the
About orphan designation for medicines
Orphan designation is granted by the European Medicines Agency’s Committee for Orphan Medicinal Products to treatments which either address an existing unmet need or can provide significant benefit for people with life-threatening or chronically debilitating diseases, affecting a small number of patients.
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
Special Note Regarding Forward-looking Statements
This press release contains forward-looking statements, as defined in the Private Securities Litigation Reform Act of 1995, as amended, including the quotes in the second and fourth paragraphs of this press release. While the company believes the forward-looking statements contained in this press release are accurate, there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, risks related to commercializing SYMKEVI in
Vertex Pharmaceuticals Incorporated
Michael Partridge, +1-617-341-6108
Eric Rojas, +1-617-961-7205
Zach Barber, +1-617-341-6470
Heather Nichols, + 1-617-341-6992
Europe & Australia:
Marie von Seyfried, + 44 7933 500887