Vertex Receives CHMP Positive Opinion for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor to Treat People With Cystic Fibrosis With At Least One F508del Mutation .

- If approved, people ages 12 years and older who have one copy of the F508del mutation and a gating (F/G) or residual function (F/RF) mutation will now be eligible for triple combination therapy -

LONDON – [26 March 2021] – Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the label extension of KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination with ivacaftor 150 mg tablets for the treatment of cystic fibrosis (CF) in all patients ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, the most common CF-causing mutation worldwide. If the European Commission follows the recommendation, the majority of people with CF in Europe will be eligible for the medicine.

The CHMP positive opinion was based on the results from a global Phase 3 study (Study 445-104) evaluating the triple combination therapy in CF patients ages 12 years and older who are heterozygous for the F508del-CFTR mutation and a CFTR gating mutation (F/G) or a residual function mutation (F/RF). The study was conducted by Vertex to complement the prior Phase 3 trials, which showed positive results for ivacaftor/tezacaftor/elexacaftor in combination with ivacaftor in people ages 12 years and older with CF with two F508del mutations (F/F) or one F508del mutation and one minimal function mutation (F/MF) genotype. It showed statistically significant and clinically meaningful improvements in primary and key secondary endpoints, including lung function in patients treated with ivacaftor/tezacaftor/elexacaftor in combination with ivacaftor.

“Today’s opinion is an important step towards bringing this medicine to any patient with at least one F508del mutation, including those with a gating or residual function mutation who were not previously eligible for the triple combination therapy,” said Nia Tatsis, Ph.D., Executive Vice President, Chief Regulatory and Quality Officer at Vertex.

In Europe, KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor is currently licensed for the treatment of people with CF ages 12 years and older with an F/F or F/MF genotype.

About Cystic Fibrosis

Cystic Fibrosis (CF) 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.

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 with two F508del mutations (F/F) or one F508del mutation and one minimal function mutation (F/MF) 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 current approved EU licensed indication for ivacaftor/tezacaftor/elexacaftor was supported by positive results of two global Phase 3 studies in people ages 12 years and older with CF: a 24-week Phase 3 study in 403 people with one F508del mutation and one minimal function mutation (F/MF) and a four-week Phase 3 study in 107 people with two F508del mutations (F/F).

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 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. 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 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign.