All Vertex Pharmaceuticals articles
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NewsNICE recommends innovative cystic fibrosis therapy
Recommendation of the once-daily triple therapy increases the number of eligible cystic fibrosis patients who can access treatments in the UK.
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NewsVertex secures European approval for CRISPR cell therapy
Now conditionally approved in Europe for sickle cell disease and transfusion-dependent beta thalassemia, the CRISPR therapy offers eligible patients a functional cure.
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NewsNext-in-class combination treatment shows potential in cystic fibrosis
Phase III trials have found CFTR modulator vanzacaftor/tezacaftor/deutivacaftor (vanza triple) to be non-inferior to TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in improving cystic fibrosis lung function.
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NewsCHMP issues positive option for first gene-editing medicine
If approved, Vertex’s Casgevy (exagamglogene autotemcel) would be the first gene-editing medicine authorised in the European Union.
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NewsFDA approves first cell-based gene therapies for sickle cell disease
The US FDA has approved Vertex’s Casgevy and bluebird bio’s Lyfgenia, for one-time treatment of sickle cell disease in patients 12 years and older.
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NewsVertex scores European cystic fibrosis medicine approval
Following a milestone approval by the European Commission in July 2023, Vertex Pharmaceuticals has been granted a label expansion for its cystic fibrosis medicine KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) with ivacaftor.
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NewsFirst CRISPR-based gene-editing therapy authorised
The UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted a world-first regulatory authorisation to a medicine based on Nobel Prize-winning technology.
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ArticleCPHI Barcelona 2023 - Event preview
CPHI Barcelona takes place in October 2023, covering a wide range of topics from next generation biomanufacturing and formulation to sustainability.
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NewsEuropean Commission extends approval of cystic fibrosis medicine
For the first time, a medicine to treat the most common form of cystic fibrosis has been approved in paediatric patients aged one to under two years old.
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NewsLonza and Vertex to partner to manufacture diabetes cell therapies
The proposed collaboration between Lonza and Vertex Pharmaceuticals will support manufacture of stem cell-derived, fully differentiated insulin-producing islet diabetes cell therapies.
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NewsEMA validates first regulatory submission for CRISPR-based medicine
The first regulatory submission for a CRISPR-based therapy has been validated by the European Medicines Agency, to treat sickle cell disease and transfusion-dependent beta thalassemia.