Vertex

Vertex

  • ‘Government to sign off on CF drug deal next month’ (Sunday Business Post)

    Please see below an article from the Sunday Business Post which predicts the approval of Orkambi ‘next month’. This follows on from recent positive comments by the Minister for Health Simon Harris TD on both Orkambi and the extension of Kalydeco on RTE radio and his comments about the likelihood of a deal on CF pipeline drugs to be included in the forthcoming agreement with Vertex.

    While CFI will remain cautious until final approval is given, it is clear that all the signs are indicating a yes decision in coming weeks, which will be fantastic news for CF patients in Ireland who are waiting for both Orkambi and the extension of Kalydeco. CFI wishes to acknowledge the continuing commitment of Minister Harris and the Taoiseach in respect of new and innovative therapies and to the party leaders and health spokespersons of opposition political parties who have highlighted this issue in the Dáil in recent months with very significant effect.

     

    CFI in particular wishes to acknowledge the parents and people with CF who have spoken so courageously to the national media in support of these new therapies and the ongoing support of the Board of CFI led by Patricia Duffy Barber. While many have been involved and we thank you all, we would like to single out Jillian McNulty in particular for her work in this regard. Thanks also to the Whitmarsh family (Ronan and Linda) and to Hazel Robinson too and all on the coordination group. ‘Your voice and support has made all the difference’. 

  • EU Approval for Orkambi in Children with Cystic Fibrosis Ages 6-11 with Two Copies of the F508del Mutation 

    Cystic Fibrosis Ireland welcomes the decision of the European Medicines Agency (EMA) to extend the Cystic Fibrosis drug ‘Orkambi’ to children aged 6 to 11 years old. Previously the drug had been available to young people and adults aged 12 years and over in Ireland and the rest of the European Union, where approved for reimbursement.

    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 (or future drugs that improve on Orkambi or Kalydeco from Vertex pharmaceuticals), would be made available to people with CF in Ireland.

    Cystic Fibrosis Ireland calls on the Minister for Health to ensure that children with CF who have the potential to benefit from this extension of Orkambi are given the choice of accessing this groundbreaking and innovative drug as soon as possible (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 2 copies of the F508del alteration. It is difficult to make a precise forecast of the number of additional children that will benefit from the extension of Orkambi, but we would estimate that up to 50 children in Ireland will  ultimately benefit from this new decision. 

    A statement from Vertex on the Approval of Orkambi in children with CF, ages 6 - 11, with two copies of the F508del Mutation can be viewed by clicking here.

    Philip Watt
    Chief Executive
    Cystic Fibrosis Ireland


  • 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.

  • Germany approves award winning Cystic Fibrosis drug ‘Orkambi’

    Philip Watt CEO of Cystic Fibrosis Ireland welcomes today’s news (19 December 2016) that a pricing and reimbursement agreement for the ground breaking Cystic Fibrosis drug Orkambi has been reached with the German Statutory Health Insurances (GKV-SV).

    According to a press release issued by Vertex today, ‘the German Federal Joint Committee (G-BA) recognized the “considerable additional benefit” of Orkambi for people with CF who have two copies of the F508del mutation’.

    Orkambi has been available to eligible patients in Germany under their approval system (different to Ireland) that allows a drug to be made available on a one year trial basis once it has been approved by the European Medicines Authority (EMA). The EMA approved Orkambi in November 2015. Under the German system, authorities have a 12 month opportunity to assess the effectiveness and proposed pricing of a drug.

    Orkambi is now available to eligible people with CF in Germany, the United States and Austria. France has a similar system to Germany and is due to consider Orkambi in March 2017. CFI further notes that Orkambi has recently received the ‘Drug Discovery of the Year Award’ award from the British Pharmacological Society and the French ‘Prix Galien’ award for the most promising rare disease medicine in 2016.

    Cystic Fibrosis Ireland recently welcomed the news that the HSE and Vertex have entered into renewed negotiations on the potential of making Orkambi available in Ireland. CFI has urged that these negotiations are carried out in private and not over the airwaves and a fair agreement is reached as soon as possible. Philip Watt stated ‘both sides should make compromises in order to reach a fair agreement, including a considerable reduction in price from the manufacturer, Vertex and a greater acknowledgment by the HSE of the very high costs and risks involved in developing a drug for rarer diseases such as CF’.

    Philip Watt further stated: ‘CFI calls for an overhaul of the system of assessing drugs for orphan diseases (rare diseases) in Ireland, including consideration of adopting the French or German drug approval systems and/or adopting a similar approach to the way cancer drugs are assessed and approved in Ireland.’

  • Good News on Orkambi and Kalydeco

    CFI has confirmation from both Vertex and the HSE that the final contracts for Orkambi and Kalydeco extension and pipeline drugs have been signed and it is likely that some CF centres will be beginning to get the drugs as early as next week.

    Please note: It will still take a while for all eligible patients to receive the drugs as there are plus 600 patients involved and multiple CF centres. Individual assessments are important for health and safety reasons. In this context we continue to urge reasonable patience.

    CFI will continue to work to ensure access to proven pipeline therapies when they become available, including for those who are not presently eligible for a bespoke CFTR drug therapy.

    Click here to read the Vertex press statement in full.

  • Good News on Forthcoming Triple Combination Therapies from Vertex 

    Cystic Fibrosis Ireland welcomes the announcement from Vertex Pharmaceuticals that two next-generations correctors, VX-659 and VX-445 have been selected to advance into Phase 3 Development as part of two different Triple Combination Regimens for people with CF. See the statement from Vertex below;

     

    Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the selection of two next-generation correctors, VX-659 and VX-445, to advance into Phase 3 development as part of two different triple combination regimens for people with cystic fibrosis (CF). The decision to advance VX-659 and VX-445 into Phase 3 development was based on initial Phase 2 data, including new data from ongoing Phase 2 studies that showed mean absolute improvements in percent predicted forced expiratory volume in one second (ppFEV1) of up to 13.3 and 13.8 percentage points from baseline through four weeks of treatment for the triple combination regimens with VX-659 (400mg QD) or VX-445 (200mg QD), respectively, in people who have one F508del mutation and one minimal function mutation (F508del/Min). Regulatory discussions are ongoing to finalize the design of Phase 3 programs for VX-659 and VX-445. Upon completion of these discussions, Vertex plans to initiate a Phase 3 program in the first half of 2018 to evaluate VX-659 in triple combination with tezacaftor and ivacaftor. In addition, the company plans to initiate a Phase 3 program in mid-2018 to evaluate VX-445 in triple combination with tezacaftor and VX-561 as a once-daily regimen, pending additional data in the first half of 2018, including the Phase 2 data on the combination of VX-445, tezacaftor and VX-561. Vertex will discuss the Phase 2 data and Phase 3 development strategy during the company's fourth-quarter and full-year 2017 financial results call for investors today, January 31, 2018 at 4:30 p.m. ET.

    The triple combination regimens were generally well tolerated across both studies, and the majority of adverse events were mild to moderate in severity. Across the studies, the discontinuation rate due to adverse events was low.

    "These results support the selection of both the VX-659 and VX-445 triple combination regimens and underscore the potential for these regimens to provide significant clinical benefits for up to 90 percent of people with CF," said Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at Vertex. "We look forward to concluding our discussions with regulators and initiating Phase 3 development in the first half of the year, with the goal of bringing a triple combination regimen to patients as quickly as possible."

    "The triple combination data demonstrate the rapid advances we are making in treating the underlying cause of CF," said Jennifer Taylor-Cousar, M.D., M.S.C.S., Associate Professor of Medicine and Pediatrics at National Jewish Health, Colorado, and co-chair of Vertex's Triple Combination Steering Committee. "Together, all the Phase 2 data to date provide further evidence that the addition of a next-generation corrector to tezacaftor and ivacaftor has the potential to provide substantial clinical benefits to patients with one F508del and one minimal function mutation who don't currently have a medicine to treat the underlying cause of their CF, as well as to provide additional benefits to patients with at least one F508del mutation who are already eligible for CFTR modulator therapies."

    About the Phase 3 Programs
    Vertex is in the process of finalizing Phase 3 study designs for VX-659 and VX-445 with regulatory agencies, with the goal of bringing a potential triple combination regimen to patients as quickly as possible. Upon completion of these discussions, the company plans to begin the first Phase 3 program in the first half of 2018, which will initially evaluate VX-659 in triple combination with tezacaftor and ivacaftor in F508del/Min patients. A second study, also to begin in the first half of 2018, will evaluate this triple combination in people with two copies of the F508del mutation (F508del/F508del). Pending data from the ongoing Phase 2 study and completion of regulatory discussions, Vertex plans to initiate a Phase 3 program in mid-2018 to evaluate VX-445 with tezacaftor and VX-561 as a once-daily regimen in F508del/Min and F508del/F508del patients.

    The VX-659 and VX-445 Phase 2 studies are both ongoing to evaluate triple combination regimens with tezacaftor and ivacaftor in F508del/F508del patients and triple combination regimens with tezacaftor and VX-561 as a once-daily regimen in F508del/Min patients. Vertex expects these data in the first half of 2018.

    The decision to advance VX-659 and VX-445 into Phase 3 development was based on initial data from the Phase 2 studies of the company's four next-generation correctors - VX-659, VX-445, VX-152 and VX-440, each in triple combination with tezacaftor and ivacaftor. Initial data from the ongoing VX-659 and VX-445 studies were announced today and are below. In July 2017, Vertex reported results from the VX-440 study as well as results from the 100mg and 200mg arms of the VX-152 study.

    About the VX-659 Phase 2 Study
    This ongoing randomized, double-blind Phase 2 study is evaluating VX-659 (80mg, 240mg and 400mg QD) in combination with tezacaftor and ivacaftor in two different groups of people with CF ages 18 and older - those who have one F508del mutation and one minimal function mutation (Part 1), and in those who have two copies of the F508del mutation (Part 2). Minimal function mutations are those that result in little-to-no functioning CFTR protein and are not responsive to ivacaftor, tezacaftor or the combination of tezacaftor and ivacaftor. Part 3 of the study is evaluating VX-659 in combination with tezacaftor and VX-561 as a potential once-daily triple combination regimen in F508del/Min patients. The primary objectives of the study are safety, tolerability and efficacy as assessed by mean absolute change in ppFEV1 from baseline. Secondary endpoints include change in sweat chloride and Cystic Fibrosis Questionnaire-Revised (CFQ-R). Data reported today are from Part 1 of the study. Parts 2 and 3 of the study are ongoing with data expected in the first half of 2018.

    Safety Data: In Part 1 of the study, the triple combination regimen was generally well tolerated. The majority of adverse events were mild or moderate. Serious adverse events were reported in seven patients: three patients in the placebo group (2 with infective pulmonary exacerbations and 1 with decreased pulmonary function test) and four in the triple combination groups (3 with infective pulmonary exacerbations and 1 with influenza). None of these serious adverse events were considered related to treatment and none resulted in treatment discontinuation. The most common adverse events ( > 10%), regardless of treatment group, were cough, headache, oropharyngeal (throat) pain and sputum increased. There were no discontinuations due to adverse events. One patient interrupted treatment due to an adverse event in the triple combination treatment groups (rash). The rash resolved following interruption of treatment and the patient subsequently restarted and completed triple combination treatment without any further incidence.

    Efficacy Data: Part 1 of the study evaluated the triple combination for four weeks in 63 patients who have one F508del mutation and one minimal function mutation (10 in placebo, 11 in VX-659 80mg, 20 in VX-659 240mg and 22 in VX-659 400mg). A summary of the within-group lung function and sweat chloride data is provided below:

    VX-659 in F508del/Min Patients

     

     

     

    Mean Absolute Within-Group
    Change From Baseline Through Day
    29*

     

     

    Mean Absolute Within-
    Group Change in ppFEV1
    (percentage points)

     

     

    Mean Absolute Within-
    Group Change in Sweat
    Chloride (mmol/L)

    Triple placebo

     

     

    +0.3
    (p=0.9053)

     

     

     

    +2.9
    (p=0.5338)

     

    VX-659 (80mg QD) + tezacaftor
    (100mg QD) + ivacaftor (150mg q12h)

     

     

    +10.2
    (p=0.0004)

     

     

     

    -45.8
    (p < 0.0001)

     

    VX-659 (240mg QD) + tezacaftor
    (100mg QD) + ivacaftor (150mg q12h)

     

     

    +11.6
    (p < 0.0001)

     

     

     

    -43.7
    (p < 0.0001)

     

    VX-659 (400mg QD) + tezacaftor

    (100mg q12h) + ivacaftor (150mg q12h)

     

     

    +13.3
    (p < 0.0001)

     

     

     

    -51.4
    (p < 0.0001)

     

    * all p-values are within group p-values based on mixed effect models; values expressed as
    ‘Through Day 29' are the average of Day 15 and Day 29 measures

    A secondary endpoint in the study measured mean absolute change in the respiratory domain of CFQ-R,1 a validated patient-reported outcome measure, at Day 29. The mean absolute improvements for patients who received the triple combination were 24.6 points (VX-659 80mg), 19.8 points (VX-659 240mg) and 21.8 points (VX-659 400mg). The improvement for those who received placebo was 4.7 points.

    About the VX-445 Phase 2 Study
    This ongoing Phase 2 randomized, double-blind study evaluated the safety and tolerability of single and multiple ascending doses of VX-445 alone and in triple combination with tezacaftor and ivacaftor in healthy volunteers (Parts A, B and C). It is also evaluating the safety, tolerability and efficacy of VX-445 (50mg, 100mg and 200mg QD) in triple combination with tezacaftor and ivacaftor for four weeks in people with CF ages 18 and older who have one F508del mutation and one minimal function mutation (Part D) and in people who have two copies of the F508del mutation (Part E). Part F of the study is evaluating VX-445 in combination with tezacaftor and VX-561 as a potential once-daily triple combination regimen in F508del/Min patients. The primary objectives of the parts of the study in CF patients are safety, tolerability and efficacy as assessed by mean absolute change in ppFEV1 from baseline. Secondary endpoints include change in sweat chloride and CFQ-R. Data reported today are from Part D of the study. Parts E and F of the study are ongoing with data expected in the first half of 2018.

    Safety Data: In Part D of the study, the triple combination regimen was generally well tolerated. The majority of adverse events were mild or moderate. Serious adverse events were reported in five patients: two patients in the placebo group (1 with hemoptysis and 1 with infective pulmonary exacerbation) and three patients in the triple combination groups (1 patient with infective pulmonary exacerbation, jugular vein thrombosis related to a central line, and distal intestinal obstruction syndrome; 1 patient with infective pulmonary exacerbation and influenza; and 1 patient with infective pulmonary exacerbation). None of these serious adverse events were considered related to treatment and none resulted in treatment discontinuation. The most common adverse events ( > 10%), regardless of treatment group, were cough, sputum increased, infective pulmonary exacerbation, hemoptysis, headache, nasal congestion, nausea, oropharyngeal pain and pyrexia. Two patients discontinued treatment due to adverse events in the triple combination treatment groups (1 patient with rash and 1 patient with increased bilirubin without associated elevations in transaminases) and none in the placebo group. Following treatment discontinuation, the rash resolved and the increased bilirubin returned to baseline. Two patients interrupted treatment due to adverse events in the triple combination groups (1 with constipation and 1 with increased bilirubin without associated elevations in transaminases); both events resolved when treatment was interrupted and both patients subsequently restarted and completed triple combination treatment without further incident.

    Efficacy Data: Part D of the study evaluated the triple combination for four weeks in 65 patients who have one F508del mutation and one minimal function mutation (12 in combined placebo, 10 in VX-445 50mg, 22 in VX-445 100mg and 21 in VX-445 200mg). A summary of the within-group lung function and sweat chloride data is provided below:

    VX-445 in F508del/Min Patients
    Mean Absolute Within-GroupChange From Baseline Through Day29*     Mean Absolute Within-Group Change in ppFEV1(percentage points)     Mean Absolute Within-Group Change in SweatChloride (mmol/L)

    Triple placebo

     

     

    0.0

    (p=0.9943)

     

     

     

    -2.2

    (p=0.5804)

     

    VX-445 (50mg QD) + tezacaftor

    (100mg QD) + ivacaftor (150mg q12h)

     

     

    +11.1

    (p < 0.0001)

     

     

     

    -38.2

    (p < 0.0001)

     

    VX-445 (200mg QD) + tezacaftor

    (100mg q12h) + ivacaftor (150mg q12h)

     

     

    +13.8

    (p < 0.0001)

     

     

     

    -39.1

    (p < 0.0001)

     

    * all p-values are within group p-values based on mixed effect models; values expressed as

    ‘Through Day 29' are the average of Day 15 and Day 29 measures

    A secondary endpoint in the study measured mean absolute change in the respiratory domain of CFQ-R1 at Day 29. The mean absolute improvements for patients who received the triple combination were 20.8 points (VX-445 50mg), 15.4 points (VX-445 100mg) and 25.7 points (VX-445 200mg). The improvement for those who received placebo was 4.2 points.

    About the VX-152 Phase 2 Study
    In July 2017, Vertex reported results for the 100mg and 200mg dose arms of the ongoing VX-152 Phase 2 study, which was also evaluating a 300mg dose of VX-152 in combination with tezacaftor and ivacaftor for two weeks in people who have one F508del mutation and one minimal function mutation and for four weeks in people who have two copies of the F508del mutation. Results from the 300mg arms of the VX-152 study were consistent with previously reported results, which showed a favorable safety profile, and rapid and significant increases in ppFEV1.

    About CF
    CF 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 buildup 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 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 and Australia. 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. For additional information and the latest updates from the company, please visit www.vrtx.com.

    Collaborative History with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT)

    Vertex initiated its CF research program in 2000 as part of a collaboration with CFFT, the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation. KALYDECO® (ivacaftor), ORKAMBI® (lumacaftor/ivacaftor), tezacaftor, VX-440, VX-152, VX-659 and VX-445 were discovered by Vertex as part of this collaboration.

    Special Note Regarding Forward-looking Statements
    This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, Dr. Chodakewitz's and Dr. Taylor-Cousar's statements in the third and fourth paragraphs, respectively, and the information provided regarding (i) Vertex's selection of VX-659 and VX-445, (ii) additional data Vertex expects from its ongoing Phase 2 next-generation clinical trials, (iii) Vertex's regulatory discussions to finalize the design of the Phase 3 development programs and (iv) the timing and design of Vertex's Phase 3 development programs for VX-659 and VX-445. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release, and 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: (i) that Vertex could experience unforeseen delays in initiating its Phase 3 development programs to evaluate VX-659 and/or VX-445 as part of triple combination regimens, (ii) that the data set forth in this press release from the ongoing Phase 2 clinical trials may differ from data from additional parts of these Phase 2 clinical trials, (iii) that data from the Phase 3 development programs may not support approval of the company's triple-combination regimens due to safety, efficacy or other reasons, and (iv) other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

    (VRTX-GEN)
    1 CFQ-R results reported are based on a mixed effect model not adjusted for baseline CFQ-R


  • Statement from Cystic Fibrosis Ireland
    Continuing lack of deal for Orkambi in England for people with cystic fibrosis will also impact on Northern Ireland

    Date of issue: July 5, 2018

    Cystic Fibrosis Ireland (CFI) is very disappointed to learn today (5 July 2018) that there continues to be no agreement for the provision of Orkambi and future cystic fibrosis (CF) drugs in England as negotiations have once again broken down without agreement between NHS England and Vertex Pharmaceuticals, despite a new deal being put on the table.

    The failure to find an agreement also impacts on people with CF in Northern Ireland as it has been made clear by the Northern Ireland Secretary of State that they will be guided by decisions on new medications by NHS England. 

    Philip Watt, CEO of CFI, stated:

    “The absence of an Assembly in Northern Ireland means that there is very limited opportunity for regional pressure to be brought to bear on behalf of patients in Northern Ireland, except by individual politicians and the fantastic work of our colleagues in the UK-based patient group Cystic Fibrosis Trust, including its CEO David Ramsden.

    “We know from our experience in Ireland, that CF patients in the UK and their families will feel devastated today by this cruel and harsh decision.

    “The decision not to fund Orkambi appears to be final and is all the more inexplicable given the fact that the drug was recently approved in the Netherlands and Sweden. Both of these countries adopted the innovative ‘Irish solution’ of providing Orkambi and providing rapid access to future drugs that are already showing excellent results in advanced clinical trials, as part of the one deal. Sweden, in particular, has a very similar drug approval system to the UK.

    “We would call on the Taoiseach, Minister for Health and politicians in the Republic of Ireland and Northern Ireland to speak out and show solidarity on this issue. While this decision will be devastating news throughout the UK for those with CF and their families, it will be particularly devastating for those in Northern Ireland who know that these innovative and ground-breaking drugs are available only a few miles south of the border and our thoughts and support are with them today.”

    ENDS

    For more information:

    Philip Watt, CEO, Cystic Fibrosis Ireland, email This email address is being protected from spambots. You need JavaScript enabled to view it.  / telephone 087 637 0557

    Issued on behalf of Cystic Fibrosis Ireland by Don Delaney, d2 communications, email This email address is being protected from spambots. You need JavaScript enabled to view it. / telephone 087 793 3249


    Click here to read the statement issued by CF Trust.

     

  • Letter to Tony O Brien, Chief Executive HSE, Re: Orkambi negotiations

    Yesterday Philip Watt, CEO of Cystic Fibrosis Ireland sent this letter to Tony O Brien in the HSE regarding the negotiations between the HSE and Vertex. A copy of this letter was also sent to the Minister for Health Simon Harris, John Hennessy, National Director, Primary Care in the HSE and Michael Barry, Director of the National Centre for Pharmacoeconomics.

     

    15 December 2017  

    Dear Mr O Brien,

    In relation to Orkambi, we greatly welcome the fact that negotiations have recommenced between the HSE and Vertex and we urge both parties to take this important opportunity to negotiate with a view to reaching a compromise that is fair to both parties and most important of all is fair to patients with CF in Ireland.

    We would further urge both parties ensure that these negotiations are held in camera and not in public to give the negotiations a real chance of success. We have written to Vertex along similar lines with the hope that a sustainable compromise can be reached.

    As the national patient body for patients with CF we are not party to the negotiations but we would offer the following observations arising from recent public discourse

    1. All of the positive benefits of Orkambi need to be considered when assessing the impact of Orkambi. These include the improvements to lung function; the reduction in the rate of exacerbations; improvements in weight and improvements in overall quality of life. CFI recognizes that these impacts will vary from one patient to another because of the nature of CF.

    2. Preventing the inevitable rate of lung function decline needs to be full taken into account. Recent long-term data published at the North American CF conference demonstrates that Orkambi slows this decline by 42% over a 2 year period.

    3. CFI has and will continue to speak out against inequities in the international pharmaceutical industry including the level of CEO salaries, however our patients cannot afford to wait for such major systemic reforms, as their health needs are in more critical and immediate. We note that these inequities are not confined to Vertex.

    4. We note that the Government’s National Plan on Rare Disease recommended the consideration of a new system of assessing orphan drugs (drugs for rare diseases) we would urge that this recommendation 30 is implemented as soon as possible.

    Yours Sincerely,

    Philip Watt

    CEO CFI 

  • Open letter to Minister for Health, Simon Harris TD

    29th November 2016

    Dear Minister,

    On behalf of Cystic Fibrosis Ireland we would ask to meet with you as soon as possible.

    We know from previous contact and your recent letter to us, that you are a compassionate and caring person, and we know your task as Minster for Health is a difficult one, including in respect of approving high cost innovative therapies.

    However, there is no disguising the reality that there has been considerable upset within the CF community in Ireland over the last couple of days which has not been helped by how the Orkambi 'decision' has been conveyed to patients. We note that you share some of our concern in this regard. This heartless form of communication to ill patients has since been compounded by the dearth of information that has been provided by the HSE to our patients since the 'decision' was leaked late on Saturday evening to a well-respected journalist.

    We note in your letter to CFI that you do not see this as the end of the process. We hope this means there is light at the end of this tunnel. However we remain very unclear to what you mean in practice. We would wish to discuss this further with you.

    In the absence of any communication from the HSE to us, we remain concerned that the impact of Orkambi appears to be 'talked down'. We would point out the following:

    Why would the HSE negotiate for 25 weeks/6 rounds of negotiations, if the HSE believed that Orkambi was not effective in the first place?

    In this context, we are further concerned that there appears to be no public acknowledgement by the HSE of the long term research data that was published on the 28th of October 2016 that confirmed:

    • A 40% decrease in hospitalisations
    • The retardation of the progression of CF for those on Orkambi
    • The reduction in other costs resulting from less hospitalisations and less dependency on other CF drugs

    We note that the highly respected Professor Stuart Elborn CBE formerly of Belfast City Hospital and now lead clinician in the lead CF hospital in the UK (Brompton Hospital in London) and Principal Investigator for the clinical trials for Orkambi stated on the 28th October 2016:

    "Long-term follow-up data has indicated that this treatment can prevent disease progression.

    "Initially, we were able to show that you can make people a bit better. Now we're seeing exciting and reassuring long-term improvement."

    "We hope this will lead to a further rethink about the long-term benefits," said Prof Elborn. "There are likely to be reductions in the cost of hospital visits and other treatments."

    "I'm really excited by the therapy and also the pipeline of other powerful drugs that could get us closer to a cure."

    (Source: 28.10.16 Press Association)


    To conclude, we would urge you as Minister to intervene and make Orkambi available for the 550 people who stand to benefit as soon as possible, even if this involves some further negotiations. We would also urge that negotiations for the extension of Kalydeco for 2-5 year olds commences as soon as possible. We will continue to lobby Vertex to significantly lower the cost of this important drug.

    Yours sincerely

    Philip Watt

    CEO Cystic Fibrosis Ireland 

  • Open Letter to Vertex Pharmaceuticals from CFI

    29th November 2016

    President, and Vice President
    Regional General Manager
    Europe North
    Vertex Pharmaceuticals (Europe) Ltd

    Dear Sirs,

    On behalf of Cystic Fibrosis Ireland (CFI) we would strongly urge, even at this late hour, that further negotiations are undertaken between Vertex Pharmaceuticals and the HSE to find a fair price for the reimbursement of Orkambi for people with CF in Ireland.

    We would urge you to reduce/further reduce the price of Orkambi and if possible enter into a ‘shared risk’ agreement to enable this important drug to be made available in Ireland – if these are the sticking points in the negotiations.

    Through a failure to communicate from the HSE, we have not been privy to the reasons why negotiations have been seemingly concluded/suspended except from snippets gleaned from the media in relation to concerns about the price and efficacy of Orkambi. We would hope the negotiations will continue.

    We have written to the Minister for Health, Simon Harris TD to seek a meeting to discuss these issues and to be reassured that as he has stated this is not the end of the process. We would equally urge Vertex to seek further compromises from which a fair decision on price can be reached and we would also urge a meeting with you on the same to convey these concerns.

    Yours Sincerely

    Philip Watt
    CEO CFI

  • 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 negotiations: latest update

    The Sunday Business Post reported at the weekend that negotiations between the state and the manufacturer of cystic fibrosis drug Orkambi have finally begun.

    You can download the article here. (pdf)

    SundayBPost

  • Orkambi Update - 30th November 2016

    The following are points made by Cystic Fibrosis Ireland in response to the public debate on Orkambi over the past few days.

    orkambi

    1. The Urgent need For Ministerial Intervention and Positive Response by Vertex

    2. The Efficacy of Orkambi

    Cystic Fibrosis Ireland urges the HSE to provide a more comprehensive and fairer public statement on the efficacy of Orkambi, particularly in relation to recent research that has shown that CF progression is slowed down by Orkambi and the impact of the 40% reduction in exacerbations.

    Orkambi (also known as ‘lumacaftor/ivacaftor’) is for people aged over 12 with two copies of F508-del gene alteration, which about 550 people in Ireland (compared with around 3,200 people across the whole of the UK).

    Declining lung function and exacerbations of Cystic Fibrosis is ultimately the main cause of death among people with cystic fibrosis. By reducing this decline, precision medicines like Orkambi can help people with cystic fibrosis stay healthier for longer. The gap in lung function widens for every year that someone eligible is not on Orkambi. This is particularly poignant where one sibling is on Orkambi and one is not.

    Orkambi reduces exacerbations by up to 40%- that is worsening of a condition that results in hospitalisations. Every time a person with CF experiences an exacerbation research has shown it can reduce their survival expectancy from 3-6 months.

    Cystic Fibrosis Ireland urges the Department of Health/HSE to take into account a recent study (28th October 2016) carried out over 96 weeks.

    Analysis of the 96-week PROGRESS extension study of the pivotal phase 3 TRAFFIC and TRANSPORT studies of Orkambi confirmed the safety profile, and mean lung function was maintained above baseline for up to 120 weeks and the 40% reduction in exacerbations.

    Professor Stuart Elborn formerly of Belfast City Hospital and now lead Cf adult consultant in the Brompton hospital commented:
    "Long-term follow-up data has indicated that this treatment can prevent disease progression.

    "Initially, we were able to show that you can make people a bit better. Now we're seeing exciting and reassuring long-term improvement."

    "We hope this will lead to a further rethink about the long-term benefits," said Professor Elborn. "There are likely to be reductions in the cost of hospital visits and other treatments."

    "I'm really excited by the therapy and also the pipeline of other powerful drugs that could get us closer to a cure."

    3. Meeting the Cost of Orkambi from Savings in the Drugs Budget

    CFI has acted responsibly at all times. We did not engage in advocacy over the 25 weeks that the HSE negotiated with Vertex, the pharmaceutical company. This was a deliberate strategy to support the HSE to obtain the best price possible for this important drug. We have called on Vertex to make significant reductions in price and to enter into shared risk agreements with the HSE, if these are the sticking points on which the negotiations have foundered.

    We would not expect the HSE to pay for a drug that does not work on some patients. We would question recent assertions that Orkambi only works on about one quarter of those taking part in clinical trials and we have sought expert opinion on this.

    IPHA agreement and related savings

    CFI notes from the Minister’s statement to the Joint Oireachtas Committee meeting in September 2016 re the IPHA agreement
    ‘The States team objective was to improve the assessment and reimbursement process for new drugs and to secure significant price reduction so as to enable continued access to new and existing drugs for Irish patients while reducing growth in the HSE’s overall drugs bill…..The new Agreement, which runs to the middle of 2020, is projected to result in savings – that is, expenditure foregone – of some €600 million from IPHA companies, with a further €150 million in savings anticipated from non-IPHA companies over the lifetime of the deal’.

    The Fight for Orkambi will continue

    CFI will continue to lobby the HSE/Department of Health and Vertex to reach a fair price on Orkambi.


    Philip Watt

    CEO, Cystic Fibrosis Ireland

  • Orkambi Update - Ongoing Negotiations

    Cystic Fibrosis Ireland have been advised that Vertex met with the HSE yesterday (05.01.17) and negotiations on the re-imbursement of Orkambi are ongoing.

  • Progress on Potential new CF drug

    The Food and Drug Administration in the United States (FDA) advisory committee, made up of independent medical experts and community representatives, voted 12 to 1 to recommend that the FDA approve the ivacaftor/lumacaftor combination therapy (Orkambi). 

    While the committee’s recommendation is an important part of the FDA’s review process, the decision is not binding. The FDA has said it will make its final decision by July 5, 2015.

    Orkambi targets the underlying cause of the disease in people with two copies of the F508del mutation. Currently, the FDA is considering whether to approve the drug for people with CF ages 12 and older who have two copies of the F508del. 

    New-Drug-for-Cystic Fibrosis

    In clinical trials, Orkambi was shown to improve lung function in people with CF and significantly reduce the rate of pulmonary exacerbations, which can lead to frequent hospitalizations and worsening lung function.

    Orkambi has still to be approved by the FDA (July 2015) and then it will be considered by the European medicine’s Agency (EMA) and then by individual countries have to consider reimbursement of the drug, should it be approved by the EMA. CFI will continue to keep our members updated on the progress of this and other potential therapies developed for use in CF care.

  • Protest and March

    Wednesday 12th April Kildare Street, Dublin at 1pm

    To support access to Orkambi and Kalydeco Cystic Fibrosis drug therapies

     

    Assemble 1pm sharp outside the Dail

    Wear something purple and bring a whistle

    In support of our member’s wishes and in partnership with CF advocates, Cystic Fibrosis Ireland is urging all our members and supporters to take part in a protest and march commencing outside the Dail in Kildare Street, Dublin on Wednesday the 12 April 2017 at 1pm sharp.

    It has now been 10 months since the assessments of the CF ground-breaking Orkambi and Kalydeco have concluded. There is heightened anxiety within the CF community of continued delays in approving these drug therapies, despite some recent positive statements.

    CFI calls on both the HSE and Vertex to reach an agreement to provide these ground-breaking drugs without further delay.

    The protest will also include a short march.

    Note: Negotiations are likely to be concluded very shortly. If an agreement is reached before Wednesday 12th of April we will be happy to call off the protest. 

  • Response from Vertex

    VertexOn Tuesday 29th November, CEO Philip Watt wrote an open letter to Vertex Pharmaceuticals regarding the negotiations around Orkambi. You can download Vertex's response below.

     

  • 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.

  • Sweden adapts Irish drug therapy deal to make Orkambi available

    Philip Watt, CEO of Cystic Fibrosis Ireland welcomes the decision to make the ground-breaking CF drug therapy Orkambi available to CF patients in Sweden. In doing so, the Swedish authorities and Vertex (pharmaceuticals) have adapted the pioneering long term access programme that was approved in Ireland in April 2017. This long term access programme not only provides patients access to Orkambi but also CF drugs not yet approved but which are in at advanced clinical trial stage and which may be even better than Orkambi.

    The CF drug Orkambi is now available in Austria, Germany, Ireland, Italy, the Netherlands, Sweden and the U.S.

    Read the full statement from Vertex below:


    Vertex Announces Long-Term Access Agreement in Sweden for Cystic Fibrosis Medicine ORKAMBI® (lumacaftor/ivacaftor) 

    June 18, 2018

    - The agreement allows for reimbursement of ORKAMBI for people who have two copies of the F508del mutation from July 1 -

    - A framework for assessment and access to our future cystic fibrosis medicines is included as part of the agreement -

    LONDON--(BUSINESS WIRE)--Jun. 18, 2018--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that ORKAMBI® (lumacaftor/ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis (CF) in people with two copies of theF508del mutation, ages six and older, will be reimbursed in Sweden after concluding the three-party negotiations with TLV and the county councils. Reimbursement is effective from July 1. The innovative, long-term access agreement also provides a framework for the assessment and access of our future CF medicines.

    “We are delighted that people with CF in Sweden will join the thousands of others around the world who are already benefitting from our CF medicines,” said Simon Bedson, International General Manager at Vertex. “We commend the Swedish authorities for partnering with us on an innovative, long-term access agreement. In countries where Vertex remains actively involved in reimbursement discussions, we encourage these health authorities and governments to match the commitment to innovation shown in Sweden to secure access for all patients who may benefit.”

    CF is a devastating rare disease that causes continuous damage to multiple organs from birth. In the lungs, a build-up of sticky mucus causes progressive and permanent damage, severe infections and ultimately premature death. In addition to Sweden, countries where lumacaftor/ivacaftor is available to all eligible patients include Austria, Germany, Ireland, Italy, the Netherlands and the U.S.

    About Cystic Fibrosis 

    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 ORKAMBI® (lumacaftor/ivacaftor) and the F508del mutation

    In people with two copies of theF508del mutation, the CFTR protein is not processed and trafficked normally within the cell, resulting in little-to-no CFTR protein at the cell surface. Patients with two copies of theF508del mutation are easily identified by a simple genetic test.

    ORKAMBI is a combination of lumacaftor, which is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del-CFTR protein, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface. Lumacaftor/ivacaftor is available as tablets and is typically taken twice per day.

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

    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 and Australia. 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. 

    For additional information and the latest updates from the company, please visitwww.vrtx.com.

    (VRTX-GEN)

    View source version on businesswire.com: https://www.businesswire.com/news/home/20180618005495/en/

    Source: Vertex Pharmaceuticals Incorporated