22 Dec
2019

Health Minister: Cystic Fibrosis drug Orkambi will be made available to younger children

first_imgHealth Minister Simon Harris has today stated that Cystic Fibrosis sufferers aged between six and eleven will be able to avail of Orkambi.The drug hit headlines last year after the National Centre for Pharmacoeconomics deemed the drug “unjustifiably expensive and not sufficiently cost-effective”, despite Cystic Fibrosis Ireland stressing the necessity of taking the drug.Last November, the HSE and producers Vertex Pharmaceuticals engaged in negotiations over the price of the drug, which stood at €159,000 per patient per year in 2016. They reached an agreement in April that allowed the availability/reimbursement of the following:Orkambi for the treatment of cystic fibrosis (CF) in patients aged 12 years and older who are homozygous for the F508del mutation in the CFTR geneKalydeco film-coated tablets for the treatment of patients with cystic fibrosis (CF) aged 18 years and older who have an R117H mutation in the CFTR geneKalydeco granules for the treatment of children with cystic fibrosis (CF) aged 2 years and older and weighing less than 25 kg who have one of the following gating (class III) mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549RThe HSE also said in April that this would be rolled out to include other age brackets subject to market authorisation in Europe.Sharing the announcement on Twitter, Minister Harris says: “Good news today for CF patients that Orkambi will be available to younger children (aged 6-11) too after EU approval. We ensured this age group was included in our deal reached last year so looking forward to younger Irish children having access soon very soon!”Cystic Fibrosis Ireland has welcomed the news today, and hopes that the treatment will be made available soon. In a statement, Chief Executive of CF Ireland Philip Watt says: “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).”The chronic disease affects 1,200 children and adults in Ireland, and 70,000 worldwide. Approximately 50 new cases of CF are diagnosed each year in Ireland.Health Minister: Cystic Fibrosis drug Orkambi will be made available to younger children was last modified: January 10th, 2018 by Elaine McCalligShare this:Click to share on Facebook (Opens in new window)Click to share on Twitter (Opens in new window)Click to share on LinkedIn (Opens in new window)Click to share on Reddit (Opens in new window)Click to share on Pocket (Opens in new window)Click to share on Telegram (Opens in new window)Click to share on WhatsApp (Opens in new window)Click to share on Skype (Opens in new window)Click to print (Opens in new window) Tags:cfcystic fibrosisorkambiSimon Harrislast_img

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