Therapy Areas: Hereditary Disorders
Vertex and Republic of Ireland Expand Long-Term Cystic Fibrosis Medicines Reimbursement Agreement
17 December 2019 - - US-based biopharmaceutical company Vertex Pharmaceuticals Inc. (NASDAQ: VRTX), following recent collaborative discussions, it has negotiated an agreement with the Health Service Executive in the Republic of Ireland to expand the existing long-term cystic fibrosis reimbursement agreement to include the triple combination regimen (elexacaftor, tezacaftor and ivacaftor), the company said.

The triple combination regimen  is under review and pending approval by the European Medicines Agency, for all eligible patients ages 12 and older in line with the potential future licensed indication.

This expands the existing agreement which had already included access to current and future Vertex medicines for people in Ireland with CF of any age who:

Have one of the specified mutations as included in the KALYDECO marketing authorization (G551D, G551S, S549R, G1244E, S1251N, G1349D, S1255P, G178R or S549N) or

Are homozygous for the F508del mutation or

Are heterozygous for the F508del mutation and have a specified mutation (P67L, D579G, D1152H, R117C, 711+3A→G, 2789+5G→A, L206W, S945L, 3272-26A→G, R352Q, S977F, 3849+10kbC→T, A455E or R1070W) and people ages 18 and older and have an R117H mutation.

This innovative long-term agreement also enabled rapid access for people with these mutations if the labels of the existing medicines were expanded to cover additional age groups and if new Vertex medicines were approved for these populations.

Triple combination treatment (elexacaftor, tezacaftor and ivacaftor) is not currently licensed for use in the European Union. Vertex submitted a Marketing Authorization Application to the European Medicines Agency on October 31, 2019.

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases.

The company has four approved medicines in the US and three approved medicines in Europe that treat the underlying cause of cystic fibrosis, 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 disease.

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 recognised 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.