Vertex Announces Health Canada Market Authorization for ORKAMBI& (lumacaftor/ivacaftor) in Children With Cystic Fibrosis Ages 1 to 2 Years

- With this approval, approximately 30 children with two copies of the F508del mutation
are eligible for a medicine that treats the underlying cause of their disease for the first
time -

TORONTO, April 10, 2023 /CNW/ - Vertex Pharmaceuticals Pharmaceuticals (Canada) Incorporated (Nasdaq: VRTX) today announced that Health Canada, as a partner of the Access Consortium, has granted Marketing Authorization for the expanded use of ^PrORKAMBI^® (lumacaftor/ivacaftor) for the treatment of cystic fibrosis (CF) in children ages 1 to <2 years who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

“With this approval, we are delighted that this new patient group is eligible for a medicine that treats the underlying cause of their disease,” said Michael Siauw, General Manager, Vertex Pharmaceuticals (Canada) Incorporated. “This approval enables us to advance closer toward our goal of developing medicines for people of all ages living with CF.”

“As a trial investigator, I have seen the benefit ORKAMBI can bring to children ages 1-2 living with cystic fibrosis,” said Larry C. Lands, M.D., Ph.D., Director, Pediatric Respiratory Medicine, Pediatric Cystic Fibrosis Clinic, and Pediatric Pulmonary Function Laboratory, Montreal Children’s Hospital, McGill University Health Center, and Professor, Department of Pediatrics, Faculty of Medicine, McGill University. “This approval is a great step towards initiating eligible patients earlier to potentially slow the progression of the disease.”

This approved label expansion is based on data from a 24-week, Phase 3, open-label, multi-center study in 46 children ages 1 to less than 2 years who have two copies of the F508del mutation. ORKAMBI^® was generally well tolerated, and the safety profile and pharmacokinetics were similar to that observed in studies in patients ages 2 years and older.

Vertex will work closely with payers to secure access for this new patient population as soon as possible.

^PrORKAMBI^® was previously approved by Health Canada for use in people with CF ages 2 years and older with two copies of the F508del mutation.

Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 88,000 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 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.

ORKAMBI^® (lumacaftor/ivacaftor) is an oral medicine that is a combination of lumacaftor and ivacaftor. Lumacaftor 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. Ivacaftor, which is known as a CFTR potentiator, is designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane. The combined actions of lumacaftor and ivacaftor help hydrate and clear mucus from the airways.

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 clinical 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 and alpha-1 antitrypsin deficiency.

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 13 consecutive years on Science magazine’s Top Employers list and one of Fortune’s Best Workplaces in Biotechnology and Pharmaceuticals and Best Workplaces for Women.

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements made by Michael Siauw and Larry C. Lands, M.D., Ph.D., in this press release, and statements regarding the estimated number of children eligible for ORKAMBI^® for the first time, our intent to work with payers to secure access as soon as possible and our beliefs regarding the benefits of our medicines. 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 or the scientific data presented. 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.