- If approved, up to 2,000 children with CF would be eligible for a medicine to treat the underlying cause of their disease for the first time -

LONDON – 20 May 2021 – Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announces that the European Medicines Agency (EMA) and the Medicines and Healthcare products Regulatory Agency (MHRA) of the United Kingdom have validated the post marketing applications for an expanded indication of KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) used in combination with ivacaftor to include patients ages 6 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

If approved, this will extend the indication for the triple combination therapy to children ages 6 through 11 years old in EU countries, Great Britain and Northern Ireland. The applications are supported by positive results from a global Phase 3 open-label study over 24 weeks that evaluated the safety and efficacy of ivacaftor/tezacaftor/elexacaftor plus ivacaftor in 66 children ages 6 through 11 years old who have either two copies of the F508del mutation or one copy of the F508del mutation and one minimal function mutation.1 The applications will now be reviewed by the EU Committee for Medicinal Products for Human Use (CHMP) and the UK MHRA, each of which will issue an opinion regarding the potential approval for these patients.

In children with CF symptoms and organ damage, including lung disease, manifest very early in life. “We are committed to working diligently with global regulators to expand the indication for our medicine such that younger people living with CF will also be able to access the triple combination therapy,” said Nia Tatsis, Ph.D., Executive VP, Chief Regulatory and Quality Officer. “Today’s news is an important milestone in broadening our access worldwide and we are pleased that we were able to file with the EMA and the MHRA in parallel.”

In April 2021 the European Commission granted approval of the label extension for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor 150 mg for the treatment of cystic fibrosis (CF) in patients ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.2 In May 2021 the Medicines and Healthcare Products Regulatory Agency (MHRA) approved the same label for patients in Great Britain.3

About Cystic Fibrosis

Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 80,000 people globally.4 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 cystic fibrosis transmembrane conductance regulator (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.

About KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in A Combination With Ivacaftor

KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor 150 mg was developed for the treatment of cystic fibrosis (CF) in patients ages 12 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Ivacaftor/tezacaftor/elexacaftor is designed to increase the quantity and function of the F508del-CFTR protein at the cell surface. The latest approved EU licensed indication for ivacaftor/tezacaftor/elexacaftor was supported by positive results of three global Phase 3 studies in people ages 12 years and older with CF: a 24-week Phase 3 study (Study 445-102) in 403 people with one F508del mutation and one minimal function mutation (F/MF), a four-week Phase 3 study (Study 445-103) in 107 people with two F508del mutations (F/F), and a Phase 3 study (Study 445-104) in 258 people heterozygous for the F508del-CFTR mutation and a CFTR gating mutation (F/G) or a residual function mutation (F/RF).

For complete product information, please see the Summary of Product Characteristics that can be found on www.ema.europa.eu and on https://products.mhra.gov.uk/

About Vertex

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 (CF) — 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 rapidly expanding pipeline of genetic and cell 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. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes.