Health Canada Grants Marketing Authorization for KALYDECO® (ivacaftor) for Patients With Cystic Fibrosis Between the Ages of 4 Months and 18 Years With the R117H Mutation in the CFTR Gene

TORONTO, March 25, 2022 /CNW/ - Vertex Pharmaceuticals (Canada) Incorporated (NASDAQ: VRTX) today announced that Health Canada has granted Marketing Authorization for the expanded use of ^PrKALYDECO® (ivacaftor) in patients from 4 months to 18 years of age and weighing at least 5 kg with the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

"KALYDECO was first approved in Canada in 2012 as the first medicine to treat the underlying cause of cystic fibrosis in patients with specific mutations," said Michael Siauw, General Manager, Vertex Pharmaceuticals (Canada). "Since then, it's been our goal to ensure that as many people with CF as possible are eligible for our treatments, and today's announcement means that approximately 25 young people with CF in Canada are now newly eligible for KALYDECO."

Vertex will work closely with all provinces, territories and private payers to secure access for eligible patients as swiftly as possible.

In Canada, ^PrKALYDECO® is already approved for the treatment of people with CF ages 18 and older with the R117H mutation, and in people with CF ages 4 months and older weighing at least 5 kg who have one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.

Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 83,000 people globally. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands 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, and these mutations can be identified by a genetic test. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. CFTR mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein 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, chronic lung infections and progressive lung damage that eventually leads to death for many patients. The median age of death is in the early 30s.

In people with certain types of mutations in the CFTR gene, the CFTR protein at the cell surface does not function properly. Known as a CFTR potentiator, ivacaftor is an oral medicine designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane, which helps hydrate and clear mucus from the airways. ^PrKALYDECO® (ivacaftor) was the first medicine to treat the underlying cause of CF in people with specific mutations in the CFTR gene.

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, cell and genetic therapies in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, pain, type 1 diabetes, alpha-1 antitrypsin deficiency and Duchenne muscular dystrophy.

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 12 consecutive years on Science magazine's Top Employers list and one of the 2021 Seramount (formerly Working Mother Media) 100 Best Companies.

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Michael Siauw in this press release, and statements regarding our expectations for the eligible patient population for and access to KALYDECO, and our beliefs regarding the benefits of our medicine. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons and other risks listed under the heading "Risk Factors" in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at www.sec.gov and available through the company's website at www.vrtx.com. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

SOURCE Vertex Pharmaceuticals (Canada) Inc.