European Medicines Agency (EMA)

European Medicines Agency (EMA)

  • 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.”


  • Further promising news from Vertex for those with F508del and one minimal function gene alteration.

     24 July 2017

    Further promising news from Vertex for those with F508del and one minimal function gene alteration.

    This news release covers the VX-152; VX-440 and the VX659 therapies presently undergoing clinical trials. These show very exciting outcomes for those with F508del and one minimal function gene alteration. Note: they still have to complete further phases of research before they go for approval to the European Medicines Agency (EMA) - likely timeframe for approval, if all continues to go well, is 2018/2019

    The results were from two phase 2, or midstage, clinical trials, and one phase 1 study evaluating three different triple-combination regimens. The studies tested three different experimental drugs, each on top of a regimen of two other drugs, called tezacaftor and ivacaftor.

    The primary goal of the study was improvement in FEV1, ‘forced expiratory volume’: This measures how much air someone can expel in one second.

    In the two phase 2 studies, one drug, called VX-152, showed a very exciting 9.7 percentage point average improvement in that measure, on top of the two-drug cocktail. Another, called VX-440, showed an even better 12 percentage point average improvement. A third, VX-659, in a phase 1 study, improved FEV1 by an average of 9.6 percentage points, also very encouraging.

    The next step is for Vertex to decide which regimen(s) to move into a late-stage clinical trial, expected to begin in the first half of 2018.

    ‘The results are a further important step for the treatment of cystic fibrosis but still at a relatively early stage’ said Philip Watt, CEO of CFI.

    Background Note:
    Vertex was the first company to develop a drug therapy targeting the root genetic cause of the disease; called Kalydeco, the treatment was approved by the Irish Government in 2013 and targets gene alterations accounting for about 4,500 cases of CF worldwide and 120 in Ireland. Ireland has the highest percentage of patients who benefitted from Kalydeco in the world. Around 11% of the CF population in Ireland have the G551D gene alteration and in the south west region, this rises to 25% of the total CF population. Kalydeco was expanded to 2-5 year olds and the R 117h gene alteration group as part of the April 2017 agreement in Ireland.  

    The company received approval for its second CF treatment, Orkambi, in Ireland in April 2017. A combination of Kalydeco (whose chemical name is ivacaftor and another drug, lumacaftor, potentially impacts on 25,000 patients worldwide and 550 patients in Ireland.


    ‘Minimal function alterations’ are gene changes that leave the CFTR protein minimally functional or unable to function at all.