Vertex Announces Extended Long-Term Reimbursement Agreement with NHS England for Access to Cystic Fibrosis Medicines KAFTRIO ® , SYMKEVI ® and ORKAMBI ®

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced an extended long-term reimbursement agreement with NHS England providing access to KAFTRIO^® (ivacaftor/tezacaftor/elexacaftor), SYMKEVI^® (tezacaftor/ivacaftor) and ORKAMBI^® (lumacaftor/ivacaftor) for all existing and future eligible cystic fibrosis (CF) patients in England.

The agreement, which also includes access to any future licence extensions of these medicines, comes as the National Institute for Health and Care Excellence (NICE) issues a positive recommendation for these CFTR modulators.

Vertex, NHS England and NICE have also committed to work together on a path towards rapid access for all eligible patients for the next in class triple combination treatment for CF, the investigational therapy vanzacaftor/tezacaftor/deutivacaftor, subject to authorisation by the Medicines and Healthcare products Regulatory Agency (MHRA).

Vertex is working with the NHS authorities in Scotland, Wales and Northern Ireland to finalise similar access agreements as soon as possible.

“We are delighted to have agreed extended long-term access to KAFTRIO^® (ivacaftor/tezacaftor/elexacaftor), SYMKEVI^® (tezacaftor/ivacaftor) and ORKAMBI^® (lumacaftor/ivacaftor) for eligible CF patients in England,” said Ludovic Fenaux, Senior Vice President, Vertex International. ”I would like to acknowledge the collaboration of NHS England, NICE and the SMC, and also thank the CF community for their contribution in describing the value that these innovative medicines bring to patients.”

Vertex CF medicines are broadly available in over 60 countries worldwide including Australia, France, Italy, Germany, the Republic of Ireland, the Netherlands, Spain and the U.S.

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

In people with certain types of mutations in the CFTR gene, the CFTR protein is not processed or folded normally within the cell, and this can prevent the CFTR protein from reaching the cell surface and functioning properly. KAFTRIO^® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor is an oral medicine designed to increase the quantity and function of the CFTR protein at the cell surface. Elexacaftor and tezacaftor work together to increase the amount of mature protein at the cell surface by binding to different sites on the 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 ivacaftor, tezacaftor and elexacaftor help hydrate and clear mucus from the airways.

KAFTRIO^® (ivacaftor/tezacaftor/elexacaftor) in combination with Ivacaftor is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients aged 2 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

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

About SYMKEVI^® (tezacaftor/ivacaftor) in Combination With Ivacaftor

In people with certain types of mutations in the CFTR gene, the CFTR protein is not processed or folded normally within the cell, and this can prevent the CFTR protein from reaching the cell surface. SYMKEVI^® (tezacaftor/ivacaftor) in combination with ivacaftor is an oral medicine that is a combination of tezacaftor and ivacaftor. Tezacaftor is designed to increase the amount of mature protein at the cell surface by targeting the trafficking and processing defect of the CFTR protein to enable it to reach the cell surface. 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 tezacaftor and ivacaftor help hydrate and clear mucus from the airways.

SYMKEVI^® (tezacaftor/ivacaftor) in a combination regimen with KALYDECO^® (ivacaftor) is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or one copy of the F508del mutation and one copy of the following 14 mutations in the CFTR gene that result in residual CFTR activity: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G and 3849+10kbC→T.

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

About ORKAMBI^® (lumacaftor/ivacaftor)

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.

ORKAMBI^® (lumacaftor/ivacaftor) is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients aged 1 year and older who have two copies of the F508del mutation (F508del/F508del) in their CFTR gene.

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

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 92,000 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 30s, but with treatment, projected survival is improving.

Diagnosis of CF is often made by genetic testing and is confirmed by testing sweat chloride (SwCl), which measures CFTR protein dysfunction. The diagnostic threshold for CF is SwCl ≥60 mmol/L, while levels between 30-59 indicate CF is possible and more testing may be needed to make the diagnosis of CF. A SwCl level of <30 mmol/L is seen in people who carry one copy of the CF gene but do not have any manifestation of disease (carriers). Higher levels of SwCl are associated with more severe disease. Restoring CFTR function leads to lower levels of SwCl.

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 approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases — cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia — and continues to advance clinical and research programs in these diseases. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including acute and neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes, myotonic dystrophy type 1 and alpha-1 antitrypsin deficiency.

Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 14 consecutive years on Science magazine's Top Employers list and one of Fortune’s 100 Best Companies to Work For. For company updates and to learn more about Vertex’s history of innovation, visit www.vrtx.com/en-gb/

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