cystic fibrosis
Vertex presents promising new data from cystic fibrosis trials
June 9, 2025Medical Communications, Research and Development European Cystic Fibrosis Conference, Rare Diseases, Vertex, clinical trial, cystic fibrosis
Vertex Pharmaceuticals has presented new data showing the clinical and quality-of-life benefits of its cystic fibrosis (CF) therapies, particularly Alyftrek …
Arcturus Therapeutics provides update on OTC deficiency and CF programmes
July 2, 2024Research and Development Arcturus Therapeutics, OTC, Pharmacy, clinical trials, cystic fibrosis
Arcturus Therapeutics has announced mid-year updates for ARCT-810, an investigational mRNA therapeutic to treat ornithine transcarbamylase (OTC) deficiency, and ARCT-032, …
Vertex announces positive results from trial for cystic fibrosis combination treatment
February 6, 2024Research and Development Chronic Diseases, Vertex pharmaceuticals, clinical trial, combination treatment, cystic fibrosis
Vertex Pharmaceuticals has announced positive results from its once-daily vanzacaftor/tezacaftor/deutivacaftor programme, the company’s phase 3 programme for the treatment of …
Arcturus Therapeutics gains FDA ODD for cystic fibrosis treatment
November 28, 2023Research and Development Arcturus Therapeutics, Chronic Diseases, FDA, ODD, cystic fibrosis
Arcturus Therapeutics has announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to ARCT-032, …
Vertex applies to expand indication of cystic fibrosis drug
May 20, 2021EMA, MHRA, cystic fibrosis
Vertex has announced that the EMA and MHRA have validated the post marketing applications for an expanded indication of Kaftiro …
EC approves Kaftrio as combination treatment for cystic fibrosis
April 29, 2021EU, European Commission, cystic fibrosis
Vertex Pharmaceuticals has been granted European Commission approval for the label extension of Kaftrio (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with …
Polyphor granted Clinical Trial Authorisation for inhaled antibiotic
December 22, 2020Manufacturing and Production Polyphor, cystic fibrosis
Polyphor, a Swiss clinical-stage biopharmaceutical company, has announced that the MHRA has granted a Clinical Trial Authorisation (CTA) to begin …
Vertex scores EU approval for cystic fibrosis drug Kalydeco in R117H+ children and adolescents
June 10, 2020Research and Development, Sales and Marketing Kalydeco, Vertex, cystic fibrosis, ivacaftor, pharma
Vertex has revealed that it has secured a new approval from the European Commission for its therapy Kalydeco (ivacaftor) in …
Vertex’s Kalydeco seizes EU CHMP recommendation for Kalydeco in R117H+ cystic fibrosis
May 1, 2020Medical Communications, Sales and Marketing Europe, Kalydeco, Vertex, cystic fibrosis
The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has given its recommendation for the approval …
FDA approves Vertex’s Trikafta in new indication, now potentially covering 90% of cystic fibrosis patients
October 23, 2019Research and Development, Sales and Marketing FDA, Trikafta, Vertex, cystic fibrosis, pharma
The FDA has granted marketing authorisation to Vertex’s Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for the treatment of cystic fibrosis (CF), it …
Vertex cystic fibrosis drugs become available in Scotland after NHS rejection
September 12, 2019Sales and Marketing SMC, Vertex, cystic fibrosis
Two potentially life-changing cystic fibrosis drugs are to become available for a potential pool of 400 patients in Scotland just …
Scotland turns down Vertex’s Orkambi
August 13, 2019Manufacturing and Production Orkambi, SMC, Scotland, Vertex, cystic fibrosis, pharma
The Scottish Medicines Consortium (SMC) has turned down Vertex’ cystic fibrosis drug Orkambi on the grounds that it is not …
FDA expands Vertex’s cystic fibrosis drug Symdeko in paediatric patients
June 24, 2019Sales and Marketing FDA, Symdeko, Vertex, cystic fibrosis, pharma
Vertex has announced that the FDA has chosen to expand the existing authorisation on its cystic fibrosis (CF) therapy Symdeko …
Parents of children with cystic fibrosis start Orkambi buyers club
June 5, 2019Research and Development NHS, Orkambi, Vertex, cystic fibrosis
Parents of children with cystic fibrosis have started an Orkambi buyers’ club to import generic versions of Vertex’s drug from …
The price of access: Can the NHS afford Orkambi?
April 29, 2019Business Services, Manufacturing and Production, Medical Communications, Research and Development, Sales and Marketing Drug pricing, NHS, NICE, Orkambi, UK, Vertex, access, cystic fibrosis, feature, pharma, pricing
The impasse over access to Vertex’s Orkambi for cystic fibrosis patients in England and Wales has revealed the cracks in …
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