• CF Drugs Update: January 2019

    Orkambi, Kalydeco and Smykevi: More Good News


    Cystic Fibrosis Ireland welcomes the news that Orkambi has been approved by the EMA (European Medicines Agency) for children aged 2-5 years old with 2 copies of the F508del gene alteration. 

    Orkambi was originally approved for reimbursement in Ireland for 12 years and older in 2017. Orkambi was subsequently extended to 6-11 year olds in 2018. The further extension of Orkambi to 2-5 years olds approved by the EMA is consistent with the drugs portfolio deal agreed in April 2017 between the HSE and Vertex Pharmaceuticals. 

    CFI will seek clarification from the HSE on timing of availability of the latest Orkambi extension in Ireland. 


    CF Ireland understands that the introduction of Symkevi (approved on 1 January 2019) is going very well. A small number of patients have been switched to Symkevi who were originally on Orkambi, based on clinical criteria and expert advice from Consultants. However CFI also understands that the vast majority of those on Orkambi will remain on Orkambi as CFI predicted in earlier news updates.

    Symkevi is a CFTR medication that is a possible alternate for those eligible for Orkambi and also for a small number of other linked gene alterations


    The EMA have also recently approved the extension of Kalydeco to 1-2 year olds. 

    Cystic Fibrosis Ireland is also seeking clarification if Kalydeco will be extended to those under 18 years of age with the R117H gene alteration. 

    Kalydeco was the first CFTR drug introduced to Ireland in 2013 of which Orkambi and Symkevi have since followed (2017 and 2019). These are the first drugs that treat the underlying cause of CF. They are personalised drugs in the sense that they are tailored for those with certain gene alterations.

    Kalydeco impacts on those with the G551D gene alteration (15% of the CF population in Ireland) but also other named gene alterations including R117H (6% of the CF population in Ireland). The most common gene alteration in Ireland and worldwide is people with CF with 2 copies of the F508del gene alteration (about 57% in Ireland). This is the group of patients eligible for Orkambi and Symkevi

  • CFI says thanks to the Government and all Political Parties for support for Orkambi and Kalydeco

    Cystic Fibrosis Ireland wishes to acknowledge Government, and all political parties for their support in ensuring Orkambi and Kalydeco was approved

    Among the many we would like to thank are:

      • An Taoiseach, Enda Kenny TD; Minister for Health Simon Harris TD and Finian McGrath Minister for Disabilities 
        Fianna Fail leader, Michael Martin TD; Health spokesperson Billy Kelleher TD; Marc MacSharry TD
        Sinn Fein leader, Gerry Adams TD and Health spokesperson Louise O Reilly 
        Labour spokespersons, Jan O’Sullivan TD and Brendan Ryan TD 
        Solidarity–People Before Profit
        Independents 4 Change, in particular Joan Collins TD 
        Rural Alliance TD’s


    There are many more not included on this this who supported us over the past 10 months – many thanks to all.

  • CFI welcomes Government approval of new CF drug Symkevi

    Cystic Fibrosis Ireland welcomes Irish Government approval of a new Cystic Fibrosis drug Symkevi. This is the third in a series of groundbreaking and innovative drugs for CF in Ireland. These are precision CFTR drugs that treat the underlying cause of CF and are aimed at particular CF gene mutations- hence the need for more than one drug. 

    Symkevi is designed for those with the most common CF gene mutation in Ireland and worldwide, which is people with 2 copies of the F508del mutation (57% of the CF population in Ireland).

    Symkevi is also aimed at those with 1 copy of the F508del mutation and one of 14 mutations.

    The other 2 CFTR drugs that were previously approved by the Irish Government are Kalydeco (2013) and Orkambi (2017). The innovative deal struck in 2017 between Vertex and the HSE provided for both Orkambi but also known drugs that were 'in the pipeline' (i.e. undergoing very positive clinical trials, such as Symkevi). Similar deals using this pipeline approach have since been struck with the Netherlands and Sweden.

    The EMA approved Symkevi in November 2018, the Irish Government approved Symkevi today (20 December) and CFI expects this important to be available to patients in January 2019

    Philip Watt, CEO of CFI stated: '

    We thank again Ministers' Simon Harris TD and Finnian McGrath TD and An Taoiseach, Leo Varadkar TD with the support of all political parties for ensuring new and groundbreaking drugs are provided to patients with CF in Ireland without delay. 

    CFI also wishes to express our support to other patients with rare disease seeking access to drugs such as Alpha 1 and Duchene Muscular Dystrophy and we welcome the recently announced extension of Pembro to Women with Cervical cancer in Ireland'.

  • Decisions on Orkambi and Kalydeco to be made at high level HSE meeting before end of February

    Update 1 February 2017

    Cystic Fibrosis Ireland has received many calls this morning about a headline in the Irish Times (1 February 2017) that states ‘Talks break down over cost of cystic fibrosis drug’.

    We understand after seeking clarification with the HSE and the pharma company (Vertex) that this headline is premature and indeed on closer reading the headline is at variance with the article itself.

    We understand (as stated in the article itself) that discussions within the HSE on Orkambi and the extension of Kalydeco are continuing and that a high level committee at directorate/HSE leader’s level will meet within the next couple of weeks to make a decision.

    Cystic Fibrosis Ireland notes in the Irish Times report the ‘HSE is unlikely to give the go ahead for the drug without a firm indication from Government that extra funding will be available to pay for it’.

    CEO of Cystic Fibrosis Ireland, Philip Watt stated ‘Judging from the amount of calls we received this morning there remains huge anxiety about the forthcoming decision to fund Orkambi and the extension of Kalydeco. This is little wonder as these are the first ever drugs to treat the underlying cause of Cystic Fibrosis (in respect of Orkambi - about 50% of the CF population in Ireland). The increase in lung function of up to 10% and the decrease in hospitalisations of 40% shows how effective Orkambi can be.’

    Philip Watt further stated ‘Cystic Fibrosis Ireland urges the Department of Health and the Department of Finance to avoid an impending row and together agree a mechanism to fund these ground-breaking drugs. We acknowledge the challenges faced by government, but would point out that as well as proven significant health benefits these ground breaking drugs will bring other savings, especially through reduced hospitalisations and less dependency on other expensive medications and treatments.’ 

  • Extension of Kalydeco (Ivacaftor) Update

    CFI have been informed that some news about the extension of Kalydeco (Ivacaftor), which is currently undergoing a Health Technology Assessment (HTA), is due to be posted on the website of the NCPE (National Centre for Pharmacoeconomics) around the 12 October or shortly after. The extension application includes commencing treatment of those already on Kalydeco to 2 years and over (children with CF who have the G551D gene alteration are currently 6 years old when treatment commences). The extension also covers other class 111 gating mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R, as listed on the NCPE website.

    Please note that this news item should not be confused with the Orkambi negotiations. These negotiations are still on going and we are at an important stage.

  • Extension of Kalydeco (Ivacaftor): Update from NCPE

    Today (12 October), the National Centre for Pharmacoeconomics (NCPE) have issued their recommendation following the Health Technology Assessment (HTA) of Kalydeco for children aged 2- 5years. Kalydeco for children aged 2–5 year olds* has not been recommended for reimbursement at the submitted price. (Note: this is not related to the forthcoming Orkambi decision)

    CFI notes that the NCPE ruling leaves a door open to further price negotiations, but the decision will be very disappointing and frustrating for the 18 young children with CF and their families, who could benefit greatly from access to this ground-breaking therapy.

    Philip Watt, CEO of Cystic Fibrosis Ireland says “Accessing this life-changing treatment is essential as eligible patients stand to benefit so much at such a crucial time in their development, fighting a progressive, life-limiting disease. We will be urging the government to enter into price negotiations as soon as possible. We would also urge the pharma company, Vertex, to reduce the price of this important drug during these negotiations”.

    The treatment, which is currently only available for those over six years old, has been shown to significantly increase lung function, slow the progression of lung disease and significantly reduce hospital admissions.

    Research published earlier this year in ‘The Lancet Respiratory Medicine’ journal suggested that pre-school children with one of the nine mutations (G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N and S549R) could also benefit from improved pancreatic function, weight gain and movement of salt between cells.


    *This assessment refers to children aged 2-5years who have one of the following gating (class III) mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P,S549N or S549R.

  • Minister's Statement on Orkambi and Kalydeco

    Cystic Fibrosis Ireland welcomes the statement last night, by Minister for Health Simon Harris TD that progress is being made in relation to the approval of ground breaking CF drugs Orkambi and Kalydeco. CEO of CFI Philip Watt stated 'while this is a positive statement of intent there will remain high anxiety among the CF community in Ireland until these drug therapies are finally approved by the HSE and the Minister.'

    CFI is supporting a dignified protest to highlight the urgency of approving these drug therapies outside the Dail on Wednesday 1 March at 1.15pm.

    Click here to read the Minister's statement.

  • Orkambi and Kalydeco extension update

    CFI understands from reliable sources that the HSE and Vertex met again this week in the latest round of negotiations on the price of Orkambi. We understand that progress is being made, but whether this progress will be sufficient to secure a deal remains unclear. The positive news is that negotiations are continuing on an active basis. CFI has called for these negotiations to be concluded as soon as possible and a fair deal for both parties is reached as this decision is of immense importance to people with CF and their families in Ireland. Cystic Fibrosis Ireland has also made representations to the HSE on the importance of commencing (as soon as possible) negotiations on the extension of Kalydeco to 2-5 year olds which impacts on 18 children with CF in Ireland. We will continue to keep our members updated on both these issues.

  • Orkambi and Kalydeco Update

    The Irish Independent reported on Saturday 14th of January that Vertex has made a revised offer to the HSE for the reimbursement of Orkambi and Kalydeco and this offer is currently under consideration by the HSE (download press report here).

  • Orkambi and Kalydeco: Advocacy and Protest Strategy of Cystic Fibrosis Ireland

    Cystic Fibrosis Ireland (CFI) understands that a decision on Orkambi and the extension of Kalydeco to 2-5 year olds will be decided within the next two weeks by the HSE Directorate. We understand that a revised offer has been made by the manufacturer (Vertex) and this is what is presently being discussed. 

    In other words, negotiations are at an advanced stage and we must be careful on the one hand to keep up lobbying behind the scenes for these vital drugs but at the same time provide a space for the HSE and Vertex to reach an agreement and for the government to have a reasonable time to make a decision.  

    It’s vitally important that the government gets the best deal on price possible, not least because there are other very promising CF ground-breaking drugs in the pipeline in the near future that will need to be resourced. 

    It is therefore the view of the Board of Cystic Fibrosis Ireland which met on 28th of January that it would be better to give space for these negotiations but to support a further protest on 1 March if necessary. 

    CFI understands the high level of anxiety among our members. This was heightened by an incorrect headline in the Irish Times of 1 February which wrongly stated that talks had broken down. Both the HSE and Vertex contacted CFI on the 1 February to say that this was incorrect and that negotiations are continuing and are at an advanced stage.

    We understand that there will be a candlelight vigil held in some parts of the country on the 17th of February or on other dates. CFI wishes the organisers good will and the best of luck with this initiative and hope that it is successful in raising awareness. If you wish to support this initiative, please do so. 

    However on balance, CFI believes it is better to give our official support to the protest that brings a lot of people together at a time when the national media and politicians are more likely to sit up and take notice. In this context we would propose that CFI members get fully behind a protest outside the Dail on the 1 March 2017 at 1.15pm. We very much hope that a positive decision in the meantime will mean that this protest can be cancelled. 

    A small coordination group of all those active in the various Orkambi and Kalydeco groups will be held on Monday 13th of February to ensure a coordinated response. We have no idea as to whether these important therapies will be approved or not, but we do know that progress has been made and we ask for your continuing support for all of us to work together. Thanks for everyone’s contribution to date, it is much appreciated!

    Philip Watt

    CEO on behalf of Cystic Fibrosis Ireland 

    7th February 2017


  • Orkambi and Kalydeco Update

    May 22nd 

    CFI has written to both Vertex and the HSE to urge them to agree the final details that will make Orkambi (and the extension of Kalydeco) available to our patients as soon as possible. CFI understands from both parties they do not envisage any problems at this stage which is good news. 

  • Protest at the Dáil today at 1.15pm

    Cystic Fibrosis Ireland will join the CF Community at a protest today, Wednesday 1 March at 1.15pm outside the Dáil to highlight the urgency of approving Orkambi and Kalydeco. We invite anybody who can, to join the CF Community in the continued effort for access to these vital drug therapies.

    CFI welcomed the recent statement by Minister for Health Simon Harris, TD that progress is being made in relation to the approval of Orkambi and Kalydeco. However, for people living with CF time is precious and they have already been waiting nine months for a decision and almost two months since the revised offer came in from Vertex. We understand this revised offer provides a very significant discount on the original list price.

    Jillian McNulty and Philip Watt (CFI) were invited to a private meeting with the Minister for Health Simon Harris at short notice last Monday. The Taoiseach referred to this meeting in the Dáil, so we can now publicly acknowledge that meeting without breaking confidences. The meeting was courteous and constructive, but fell far short of a ‘yes’ decision on Orkambi and Kalydeco, as can be seen from the subsequent Minister’s statement. The Minister made it clear that while he was against the protest today, it would not jeopardise a possible final positive decision. Once again we welcome the support and efforts by Minister Harris and we will continue to engage with him and the Government to ensure we get a positive decision.CFI will also continue to provide support with the CF community at this difficult and anxious time. 

  • Calling all people with CF, parents, carers & family members - we want to hear your views!!

    Please complete our survey by clicking HERE


    Why is your participation so important?

    As many of you are aware, two new possible treatment options for some people with CF (PWCF) are currently being assessed for both clinical and cost-effectiveness by the National Centre for Pharmacoeconomics (NCPE). 

    1. Orkambi, which seeks to correct the basic underlying genetic defect in 57% of people with CF in Ireland, people with two copies of F508del mutation. 
    2. The expanded use of Kalydeco for mutations:
          • G551D & other non-G551D gating mutations (expanded use to PWCF aged 2-5 years)
          • R117H (for PWCF over 18 years)

    As with all new therapies, they must undergo Health Technology Assessments (HTA) which is

    "A form of research that generates information about the clinical and cost-effectiveness of health technologies. These technologies can include drugs, medical devices, diagnostic techniques, surgical procedures and public health programmes such as cancer screening programmes. A Health Technology Assessment (HTA) may also look at the social, ethical, medicolegal and organisational aspects associated with use of a technology including its resource implications and budget impact.

    The information provided by the HTA is used to inform health policy decisions regarding the investment in (or disinvestment from) these health technologies."

    Cystic Fibrosis Ireland (CFI) are putting together a submission which will be made to the National Centre for Pharmacoeconomics (NCPE). This primary aim of this submission is to ensure the patient & family perspectives are formally taken into consideration during the Health Technology Assessment of the new therapies: Orkambi & Kalydeco (for expanded use).

    The purpose of this survey is to gather information from both people with CF and their families about experiences & views of treatment options for cystic fibrosis & ultimately, 'what matters to you'.

    It is so important that the voice of the CF community is heard loud and clear throughout, so please fill in the survey to have your say today.

    This process and the arrival of similar innovative therapies in the future will challenge the HSE & Irish Government to look at what 'value for money' really means in the context of CF care. The whole community must speak up and make sure these decisions are guided by the unique knowledge and experience of people living with CF.

    This survey has been shared by the Cystic Fibrosis Trust, UK, who have kindly allowed CFI to use & edit to questionnaire to meet the needs of the Irish CF population.

    The survey will take approximately 15-20 minutes to complete & we ask you to provide as much detail as possible.

    The results of this survey will be used to represent the views of the CF community & will primarily be used for advocacy purposes, and to inform policy decisions.

    Your responses will be completely confidential and any published results will be entirely anonymous.

    If you have any questions about the research, please contact Katie, Research & Development Officer, CFI at This email address is being protected from spambots. You need JavaScript enabled to view it. or locall 1890 311 211


  • Statement from Cystic Fibrosis Ireland - Orkambi (and Kalydeco)

    Cystic Fibrosis Ireland (CFI) is concerned that, according to a recent statement by Vertex Pharmaceuticals, there has been no contact between the Health Service Executive and Vertex in recent weeks, despite repeated public assurances by the Minister for Health, Simon Harris TD, and An Taoiseach, Enda Kenny TD, that a deal on important new drug therapies is imminent.

    On behalf of our patients, CFI calls on Minister Harris and the Government to do what they have promised to do – to make an agreement for the provision of Orkambi and Kalydeco for the 600 patients that would benefit from these two vital drug therapies.

    The negotiations for these ground-breaking drugs are now more than nine months old. There have been repeated assurances given in recent weeks which CFI has welcomed. Cystic Fibrosis patients would be very grateful for the promised announcement on a final agreement.

  • Symkevi likely to be available in Ireland in early 2019 

    As predicted in the Autumn issue of Spectrum, the European Medicines Agency have now fully approved the third CFTR drug from Vertex called 'Symkevi'. 

    Those that stand to benefit from this new drug are the same group of patients that benefit from Orkambi (double Delta F508). It is likely that a protocol for deciding which patients will remain on Orkambi and which may switch to Symkevi will be developed.  

    The PR Company for Vertex Pharmaceuticals have informed Cystic Fibrosis Ireland of additional updates, as below:

    • The European Commission has granted Marketing Authorization for SYMKEVI® (tezacaftor/ivacaftor), a new treatment option for patients with two copies of the F508del mutation, the most common mutation in cystic fibrosis.
    • SYMKEVI® is the first medicine in the EU to treat the CFTR protein defect in patients who have one copy of the F508del mutation and one copy of one of 14 mutations that result in residual CFTR activity
    • The agreement that Vertex agreed in 2017 with the HSE included facilitates an expedited review of our reimbursement application for certain treatments and extensions to age cohorts to specific CF mutations, subject to market authorisation by the European Commission.
    • Accordingly, Vertex will work closely with the HSE and relevant officials to secure timely access for these patients, and we hope it will be made available shortly

    As part of the pipe-line/portfolio deal agreed between the HSE and Vertex, CFI predicts that Symkevi will be available in Ireland in early 2019.

    We will keep you updated.

    Statement from Vertex

    Vertex Announces European Authorization for Third Cystic Fibrosis Medicine SYMKEVI® (tezacaftor/ivacaftor), to be used in combination with ivacaftor (KALYDECO®), for People with CF Aged 12 and Older with Certain Mutations in the CFTR gene

    - A new treatment option for patients with two copies of the F508del mutation, the most common mutation in cystic fibrosis -

    - First medicine in the EU to treat the CFTR protein defect in patients who have one copy of the F508del mutation and one copy of one of 14 mutations that result in residual CFTR activity -

    LONDON--(BUSINESS WIRE)--Nov. 1, 2018-- Vertex Pharmaceuticals (Europe) Limited, today announced that the European Commission has granted Marketing Authorization for SYMKEVI® (tezacaftor/ivacaftor) in a combination regimen with ivacaftor (KALYDECO®) for the treatment of people with cystic fibrosis (CF) aged 12 and older who either have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, or one copy of the F508del mutation and a copy of one of the following 14 mutations in which the CFTR protein shows residual activity: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T. In addition, the European Medicines Agency’s Committee for Orphan Medicinal Products recently recommended the maintenance of orphan designation for tezacaftor/ivacaftor in combination with ivacaftor.

    “The authorization of tezacaftor/ivacaftor in combination with ivacaftor is welcome news for European CF patients, their families and everyone involved in their treatment and care. This new medicine is especially important for patients with residual function mutations and those who do not tolerate ORKAMBI® (lumacaftor/ivacaftor),” said Harry Heijerman, Professor and Head of Department of Pulmonology at University Medical Centre Utrecht, The Netherlands.

    The EU Marketing Authorization was based on results from two pivotal Phase 3 studies, EVOLVE and EXPAND, published in the New England Journal of Medicine in November 2017. Results showed treatment with tezacaftor/ivacaftor in combination with ivacaftor provides benefits across different CF populations, including statistically significant improvements in lung function, as determined by absolute change from baseline in percent predicted forced expiratory volume in one second (ppFEV1); with a generally well tolerated safety profile and a lack of increased respiratory adverse events compared to placebo. The improvements in lung function showed a mean absolute change in ppFEV1 compared to placebo of 4.0 percentage points (P<0.0001) and 6.8 percentage points (P<0.0001) in EVOLVE and EXPAND respectively. The most common adverse reactions experienced by patients who received tezacaftor/ivacaftor in combination with ivacaftor in pooled, placebo-controlled Phase 3 studies were headache and nasopharyngitis.

    “Today marks an important milestone for many CF patients in Europe, including those who so far have had no available option to treat the CFTR protein defect responsible for their disease,” said Reshma Kewalramani, MD, Executive Vice President, Global Medicines Development and Medical Affairs and Chief Medical Officer at Vertex. “With today’s Marketing Authorization, we are rapidly moving towards treating 90 percent of CF patients.”

    Tezacaftor/ivacaftor in combination with ivacaftor was approved by the U.S. Food and Drug Administration (FDA) in February 2018 and by Health Canada in June 2018. It is marketed as SYMDEKO™ in the U.S. and Canada.

    About CF
    Cystic fibrosis is a rare, life-shortening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

    CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working or too few CFTR proteins at the cell surface. The defective function or absence of CFTR protein results in poor flow of salt and water into and out of the cell in a number of organs. In the lungs, this leads to the build-up of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the mid-to-late 20s.

    About tezacaftor/ivacaftor and ivacaftor
    Some mutations result in CFTR protein that is not processed or folded normally within the cell, and that generally does not reach the cell surface. Tezacaftor is designed to address the trafficking and processing defect of the CFTR protein to enable it to reach the cell surface where ivacaftor can increase the amount of time the protein stays open.

    For complete product information, please see the Summary of Product Characteristics that can be found on www.ema.europa.eu once posted.

    About EVOLVE and EXPAND
    Data from the two Phase 3 studies EVOLVE and EXPAND were published in the New England Journal of Medicine in November 2017, the studies enrolled approximately 750 people with CF ages 12 and older with two copies of the F508del mutation or with one F508del mutation and a second mutation associated with residual CFTR activity. Across both studies, patients treated with tezacaftor/ivacaftor in combination with ivacaftor experienced statistically significant improvements in lung function, as determined by absolute change from baseline in ppFEV1. The treatment was generally well tolerated; the most common adverse reactions (≥10%) experienced by patients who received tezacaftor/ivacaftor with ivacaftor in the pooled, placebo-controlled Phase 3 studies were headache (14% versus 12% on placebo) and nasopharyngitis (12% versus 10% on placebo).

    About orphan designation for medicines
    Orphan designation is granted by the European Medicines Agency’s Committee for Orphan Medicinal Products to treatments which either address an existing unmet need or can provide significant benefit for people with life-threatening or chronically debilitating diseases, affecting a small number of patients.

    About Vertex
    Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious and life-threatening diseases. In addition to clinical development programs in CF, Vertex has more than a dozen ongoing research programs focused on the underlying mechanisms of other serious diseases.

    Founded in 1989 in Cambridge, Mass., Vertex's headquarters is now located in Boston's Innovation District. Today, the company has research and development sites and commercial offices in the United States, Europe, Canada, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including being named to Science magazine's Top Employers in the life sciences ranking for eight years in a row.

    Special Note Regarding Forward-looking Statements
    This press release contains forward-looking statements, as defined in the Private Securities Litigation Reform Act of 1995, as amended, including the quotes in the second and fourth paragraphs of this press release. While the company believes the forward-looking statements contained in this press release are accurate, there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, risks related to commercializing SYMKEVI in Europe and the other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. (VRTX-GEN)

    Source: Vertex Pharmaceuticals Incorporated

  • Update on Orkambi and Extension of Kalydeco 

    May 30th 2017 

    CFI has been reliably informed that patients will not have to wait much longer to gain access to Orkambi and the extension of Kalydeco. We understand the final arrangements on the roll out of these important drug therapies is nearing completion and has progressed significantly since our last update. Please note that clinicians will also have to make preparations in relation to assessment and monitoring of these drug therapies on a regular basis. With around 600 patients and many specialised centres involved, there is an inevitable lead-in time before all preparations are in place. This is important to ensure the health and safety of CF patients. We will update you further when we get further news but would urge patience and understanding in the mean-time. 


    Very encouraging Late Stage Study Results for forthcoming CF Triple-Combination drugs

    On 27 November Vertex pharmaceuticals released the initial Phase 3 clinical trial data for one of two next-generation, triple-combination drugs that are undergoing late stage clinical trials (Phase 3 trials). These are further part of the CFTR family of drugs that includes Klaydeco; Orkambi and most recently Symkevi.

    ‘The results are very encouraging’  said Philip Watt, CEO Cystic Fibrosis Ireland (CFI). 'They are still at clinical trial stage but they point to the increasingly strong possibility of further drugs coming down the line for those already on a CFTR drug and for some of those that are not. It continues to be a time of hope for people with CF. We are determined to try and ensure that no one gets left behind and that there will be a CFTR drug available to as many patients as possible in Ireland’

    In further, unconnected news - Novartis has sold TOBI Solution and Tobi Pod inhaler to US pharma firm, Mylan which also has a significant presence in Ireland (see information at end of this article).

    The results
    Positive results were announced from late-stage studies of a potential triple-combination CFTR drug for people with cystic fibrosis. Vertex Pharmaceuticals released Phase 3 clinical trial data for two studies of the next-generation modulator VX-659 in combination with ivacaftor and tezacaftor. 

    The first study tested this triple-combination drug on people with CF ages 12 years and older who have one copy of the most common gene alteration (mutation), F508del, and one minimal function mutation

    Results showed that those who received VX-659 combined with ivacaftor and tezacaftor had a 14 percent increase in lung function compared to participants taking a placebo. Researchers believe that anyone with at least one copy of the F508del mutation -- regardless of their second mutation -- could benefit from next-generation modulators.

    The second study included people with CF ages 12 years and older who have two copies of the F508del mutation. The trial compared the effectiveness of Symkevi (tezacaftor/ivacaftor) to VX-659 combined with tezacaftor and ivacaftor. Participants who were given VX-659 combined with tezacaftor and ivacaftor had a 10 percent improvement in lung function over those who were only given Symkevi® (tezacaftor/ivacaftor).

    Just as important are the wider impacts that these drugs will likely have including overall quality of life and reduction in exacerbations and weight gain. It’s too early to be definitive but the signs at this stage are very encouraging.

    Vertex will decide which of the two next-generation drugs they will submit to the U.S. Food and Drug Administration (FDA) for potential approval as a new drug. If approved, it is estimated that triple-combination CFTR modulators could potentially bring the benefits of therapies that treat the underlying cause of the disease to more than 90 percent of people with CF.

    Michael P. Boyle, M.D., senior vice president of therapeutics in the US Cystic Fibrosis Foundation stated:

    ‘It is an exciting time for the CF community, as we approach a milestone that seemed impossible even just a few years ago. Today's announcement represents an important step in our journey to developing treatments for the underlying cause of this disease for all people with cystic fibrosis. We are enthusiastic about the clinical benefit VX-659 demonstrated in these studies and look forward to seeing how these compare to the other next-generation modulator in clinical trials, VX-445.’

    CF Hospital Centres in Ireland have played a key role in supporting clinical trials for these medications. It is unclear yet when they will be submitted for approval to the FDA in the US and the EMA in Europe.

    There are presently 3 Vertex CFTR drugs approved for reimbursement in Ireland. These are ground-breaking drugs that treat the underlying cause of CF and include:

    Kalydeco (2013) primarily for those patients with the G551D gene alteration

    Orkambi (2017)for those patients with two copies of the F508del gene alteration

    Symkevi (2018) for those patients with two copies of the F508del gene alteration (alternate drug to Orkambi)

    In relation to Symkevi, CFI understands that a protocol is being developed. It is likely that those who are doing well on Orkambi (and the feedback to date is generally very positive) will remain on Orkambi.

    The impact of these drugs are closely monitored in Ireland. CFI will continue to provide impartial and informed information about these and future CF Drugs

    Further Drugs News: Novartis sells Tobramycin to Mylan
    In September 2018 Mylan, a Pennsylvania pharma company paid Novartis, the Swiss based pharm-company, 463 million dollars for its CF therapies including TOBI solution and TOBI pod-inhaler. This coincides with TOBI coming off patent. Nebulised Tobramycin was introduced in 1998 and has proven especially effective against Pseudomonos. Meanwhile Teva an international Pharma company has introduced a generic form of Nebulised Tobramycin.  These developments will likely result in significant price reductions in Nebulised Tobramycin to the HSE. 

  • VX-661 news

    Vertex Pharmaceuticals issued a statement on Monday 15th August that it is ending a clinical study testing a two-drug combination therapy on a small group of cystic fibrosis patients after an independent board concluded the experimental treatment wasn’t showing meaningful benefit.

    Executives had previously warned that the late-stage clinical study combining its drug candidate, called VX-661, with its approved drug Kalydeco might not prove effective for about 150 patients enrolled in the study. Each of those patients has two separate genetic mutations linked to CF.

    In a statement, Vertex chief medical officer Jeffrey Chodakewitz said that this CF population has “a form of the disease that is particularly difficult to treat.” But he said Vertex remains hopeful those patients will be able to benefit from a three-drug combination -- including VX-661 and Kalydeco -- that Vertex is also developing.

    Background Note: Vertex have two approved drugs, Kalydeco and Orkambi, which treat CF patients with certain mutations. Kalydeco has been approved and is reimbursed in Ireland. Orkambi is not yet available in Ireland, except on compassionate grounds as the price is currently being negotiated. These are the first drugs that treat the underlying cause of CF