news
Vertex 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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Vertex Pharmaceuticals
news
Next-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.
news
CHMP 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.
news
FDA 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.
news
Vertex 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.
news
First 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.
article
CPHI 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.
news
European 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.
news
Lonza 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.
podcasts
EPR Podcast Episode 18 – Embracing continuous manufacturing – Trish Hurter, Lyndra Therapeutics
In this episode Dr Patricia Hurter, CEO of Lyndra Therapeutics, discusses how the pharmaceutical sector can embrace continuous manufacturing. She also shares her tips for women looking to progress their careers in pharma.
news
EMA 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.
news
Defining best practice for continuous manufacture of OSD forms
The International Society for Pharmaceutical Engineering (ISPE) has published the Good Practice Guide: Continuous Manufacturing of Oral Solid Dosage Forms, in a bid to increase uptake.
news
Vertex’s Kaftrio approved for use in Europe and made accessible on the NHS
The European Commission and European Medicines Agency have approved the triple combination Kaftrio for use in certain cystic fibrosis patients, triggering the drug to be accessible on the UK’s National Health Service.
article
Radical innovation occurs when pharma looks outside its own world
Pharma executives are committed to bringing drugs to market to improve the lives of patients but is the industry too introspective to be truly innovative?
news
EC approves expanded indication for cystic fibrosis treatment
The European Commission has approved the label extension for KALYDECO® (ivacaftor) to include the treatment of infants with cystic fibrosis between six and 12 months old.
