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Vertex Pharmaceuticals inks deal with NHS for access to 3 cystic fibrosis drugs

Vertex Pharmaceuticals Inc. said its cystic fibrosis medicines Orkambi, Symkevi and Kalydeco will be available on the National Health Service in England following an access agreement between the company and NHS.

Cystic fibrosis is a life-threatening and rare inherited disease which causes the buildup of sticky mucus in the lungs and digestive system, potentially leading to severe damage of the affected organs. The disorder is due to alterations in the CFTR gene, with F508del mutation being the most common gene defect.

Under the agreement, patients will have full access to the three therapies for free through the NHS, with no limit on patient numbers. The deal covers all current and future license indications of Orkambi, Symkevi and Kalydeco.

Orkambi, or lumacaftor/ivacaftor, is approved to treat cystic fibrosis in patients aged two years and older whose disease has the F508del mutation in the CFTR gene. Additionally, Symkevi, a combination of tezacaftor/ivacaftor, is for the same indication in patients who are 12 years old and above.

Kalydeco, also known as ivacaftor, is indicated for patients aged one year and older and whose cystic fibrosis is positive for one of nine mutations. Boston-based Vertex said Kalydeco is the first approved treatment targeting the underlying cause of cystic fibrosis in people with specific mutations in the CFTR gene.

The National institute for Health and Care Excellence has backed the agreement. As part of the transaction, NICE will conduct a full appraisal of the Vertex drugs, along with the company's new triple therapy.

Commercial terms of the agreement are confidential.