Research News

Research News

Research Ezine Past Issues
Wednesday, 22 August 2018
  Check out past issues of the Research e-Zine by clicking on the issue number below. Issue 2: August 2018 Issue 1: May 2018
Research Ezine
Monday, 23 April 2018
  Research Ezine In Cystic Fibrosis Ireland, we realise that health literacy is extremely important for persons with cystic fibrosis (PWCF) and their families who want to learn more about existing and future cystic fibrosis (CF) treatments. With this in mind, we have produced a quarterly e-zine to keep you up to date on the latest clinical research...
article thumbnailLaunch of CFI “Independent Living” Report On Wednesday 4th April 2018 CF Ireland launched a new and groundbreaking report on “Independent Living” which highlights major improvements in the quality and the length of lives of people with CF over two decades (1998-2017). The report shows that the sustained investment in better CF services and...
article thumbnailJoint North-South Rare Disease Conference To celebrate International Rare Disease Day (28th February 2018), a Joint North-South Rare Disease Conference took place in Riddel Hall, Belfast on Monday 5th  March 2018. The Conference offered participants the opportunity to be part of a global call on policy makers, researchers, companies and healthcare...
Drug Iceberg 2.0 Published
Wednesday, 18 April 2018
article thumbnailDrug Iceberg 2.0 Published In February 2018 the Irish platform for patient organisations, science and industry (IPPOSI) and the Medical Research Charities Group (MRCG) announced the publication of the second “Drug Iceberg” Report. This report lay out key recommendations including a call for the development of a new national strategy on access to...
article thumbnail18th National Cystic Fibrosis Clinical Meeting The 18th National Cystic Fibrosis Clinical Meeting took place in Killarney, Co. Kerry on the 1st and 2nd February 2018. Over the course of the two day meeting, leading CF clinicians and researchers from around the world outlined their work to address a particular issue in CF care. Topics discussed...
article thumbnailExtension of Orkambi to children aged 6-11 The recent decision of the European Medicines Agency (EMA) to extend the CF drug “Orkambi” to children aged 6 to 11 years old, on the basis of clinical research emerging from two Phase III studies (NCT02514473 and NCT01897233), means that this ground breaking drug which had previously only been available to...
article thumbnailIntroduction to International Rare Disease Day Conference  On 28 February 2017, Philip Watt in his role as CEO of Cystic Fibrosis Ireland, Chairperson of MRCG and a member of the Rare Disease Taskforce, opened the International Rare Disease Day Conference in the Royal Hospital, Kilmainham, Dublin. Below is his introduction speech from that...
article thumbnailWhitmarsh, R., & Whitmarsh, L (2016) - Click on the image below to read this interesting research poster
article thumbnailCystic Fibrosis Ireland's Conference & Education Grant is open & we are now accepting applications - click on the image below to read the grant policy & to complete an application form   Applications forms should be completed and send back to This email address is being protected from spambots. You need JavaScript enabled to view it. document.getElementById('cloakd87baed5acf31601842841ddf5bd7758').innerHTML =...
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Extension of Orkambi to children aged 6-11

The recent decision of the European Medicines Agency (EMA) to extend the CF drug “Orkambi” to children aged 6 to 11 years old, on the basis of clinical research emerging from two Phase III studies (NCT02514473 and NCT01897233), means that this ground breaking drug which had previously only been available to those aged 12 years and older with two copies of the F508del mutation, will now also be available to children aged 6-11 (subject to the advice of their Consultant).

Orkambi treats the most common CF gene alteration/ mutation in Ireland (and the world) that is people who have two copies of the F508del alteration. Orkambi was approved for reimbursement by the Irish Government in April 2017. As part of that agreement, in a unique “pipeline deal”, it was agreed that future extensions of Orkambi or Kalydeco (including this EMA decision to extend Orkambi to children aged 6-11), would be made available to people with CF in Ireland.

Commenting on the extension of Orkambi to children aged 6-11, the Minister for Health Simon Harris said:
“It’s very positive news that Orkambi is now licenced and available for reimbursement in Ireland for CF patients aged 6 to 11.”