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Update (December 30-31, 2012) On The Status Of Vertex Pharmaeuticals’ Orphan Drug Kalydeco (Ivacaftor) In The UK

December 30, 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.

As reported widely in the media, 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 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. Decisions still have to be made in Northern Ireland and Wales

Scotland is due to make a decision on January 14, 2013 on Kalydeco. According to a December 31, 2012 CF Trust UK Press Release, Kalydeco must also be made available to Scottish patients as soon as possible. About 4% of CF patients in the UK have the G551D mutation – compared with about 11% of the Scottish CF population. This comes to approximately 80 people in Scotland who would be eligible for Kalydeco.

The Belfast Telegraph in Ireland in a December 30, 2012 article titled, “New drug set to be made available”,  says that the Commissioners in Northern Ireland are aware of the recent decision in England for Kalydeco, and “ …. Expect to be in a position early in 2013 to confirm the arrangements by which people with CF in Northern Ireland will be able to access this new treatment.” Kalydeco will be effective for about 6% of patients with CF in Northern Ireland.

The CF Trust UK, announces that on January 12, 2013 , there is a virtual conference of the new research-based website CFUnite, where Dr. David Sheppard, from The University of Bristol, will discuss Kalydeco. Also, there will be a presentation on clinical trial results of Kalydeco for 2 different types of CF mutations. One can access the website, can follow on social media via Twitter at @CFUnite and Facebook/CFUnite.

According to a December 27, 2012 Forbes article titled, “The Most Important New Drug Of 2012”, Vertex Pharmaceuticals’ orphan drug Kalydeco (Ivacaftor) for Cystic Fibrosis, “stands out for its scientific and medical importance … is a triumph of genetics and drug development, the first medicine to directly affect the genetic defect that causes the disease …”.

According to the article, Kalydeco makes medical history for 3 reasons :

1) Triumph of Personalized Medicine

2) Power of a patient advocacy organization, the Cystic Fibrosis Foundation, for helping in the development of the drug

3) Price : $294,000/year/patient.

Vertex Pharmaceuticals is scheduled to present on January 7, 2013, at the 31st Annual J.P. Morgan Healthcare Conference.


CFUnite Introductory Video

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

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