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NICE recommends gene therapy for severe sickle cell disease
The committee’s approval of the one-time gene therapy means the healthcare inequalities for patients with this genetic blood disorder can begin to be re-addressed.
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NICE recommends new haemolytic anaemia treatment
The National Institute For Health and Care Excellence (NICE)'s decision follows a reduction in the price of Pfizer’s small molecule treatment for sickle cell disease.
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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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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.
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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.
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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.
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Raman spectroscopy market expected to value $875m by 2028
A report has shown that growing demand for accurate identification of pharmaceutical raw materials is anticipated to boost the global Raman spectroscopy market.
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Stem cell therapy delivers potential for sickle cell disease
A Novartis’ Phase I/II study for sickle cell disease trialling the gene therapy OTQ92 is the first treatment to target a new genetic area and use cryopreserved stem cells, according to new paper published in NEMJ.
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European Commission revokes marketing authorisation for Novartis’ crizanlizumab
Novartis will remove its sickle cell medicine crizanlizumab from the EU/EEA market following a European Commission decision.
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Could novel gene therapy treat sickle cell disease?
Researchers have revealed positive preliminary data from a CRISPR gene therapy trial for sickle cell disease.
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CHMP meeting highlights – May 2023
The Committee for Medicinal Products for Human Use (CHMP) recommended two new medicines for approval in its May meeting, including a neurosteroid for epileptic seizures.
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CHMP meeting highlights – January 2023
Four new medicines were recommended for approval at the Committee for Medicinal Products for Human Use (CHMP)’s January meeting, with positive opinions given to two type 2 diabetes drugs.
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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.
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First patients given lab-grown blood cell transfusion
The second-ever patient has been transfused with lab-grown red blood cells in a clinical trial, promising an effective treatment for patients.
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Pfizer to acquire Global Blood Therapeutics in $5.4bn deal
Through the acquisition of Global Blood Therapeutics, Pfizer aims to enhance its presence in rare haematology and particularly sickle cell disease (SCD).
