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Good News For Bringing Vertex Pharmaceuticals’ Orphan Drug Kalydeco (Ivacaftor) To England

December 19, 2012

The Orphan Druganaut Blog has been following for the past 6 months, the efforts of patient advocacy groups, the Cystic Fibrosis Trust UK, CF patients and their families, for getting Vertex Pharmaceuticals’ orphan drug Kalydeco (Ivacaftor) for the treatment of Cystic Fibrosis (CF) to the UK.  Now there is good news to report !!

Vertex Pharmaceutials announces on December 19, 2012, that a decision is made by the National Health Service (NHS) in England to fund starting January 1, 2013, Kalydeco (Ivacaftor) for patients ages 6 and older who have at least one copy of the G551D mutation in the CFTR gene. Kalydeco is effective for the 4% of patients in the UK who have this gene mutation in CF.

This announcement follows a thorough clinical and economic evaluation of Kalydeco by the NHS in England. Vertex Pharmaceuticals agrees to a patient access scheme with the NHS. The details of this patient access scheme are confidential. The company is to make Kalydeco available to eligible patients as fast as possible. Reimbursement is to begin in the 2nd Quarter 2013.

This decision is only for CF patients who live in England. The CF Trust UK is continuing their efforts to make Kalydeco available in each of the UK nations.  Scotland is due to make a decision on January 14, 2013. Decisions still have to be made in Northern Ireland and Wales.


Cystic Fibrosis Trust UK December 19, 2012 Press Release titled, “Victory for Kalydeco Campaign in England”

Statement from North of England Specialized Commissioning Group (SCG) December 19, 2012 Statement

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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