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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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sickle cell disease
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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).
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Global Blood Therapeutics: driving innovation in sickle cell disease
Sebastian Stachowiak, Head of Europe & GCC at Global Blood Therapeutics (GBT), explains how, while the company establishes its presence in Europe, it continues its commitment to advance the care of people living with sickle cell disease by collaborating with the sickle cell community to develop breakthrough therapies, raise awareness…
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Drug Development In-Depth Focus 2022
Features in this in-depth focus highlight the opportunities presented by targeted protein degradation therapeutics, as well as the breakthroughs in developing treatments for sickle cell disease.
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Experimental gene therapy reverses sickle cell disease for three years
The gene therapy corrected the shape of some sickle cell patients' red blood cells and eliminated episodes of severe pain.
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NICE recommends first sickle cell disease treatment in 20 years
Crizanlizumab recommended as a treatment option for preventing recurrent sickle cell crises for people aged 16 and over in the UK.
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WHO publishes recommendations on human genome editing
To promote the safe and ethical use of genome editing for the treatment of disease and genetic disorders WHO has published the first global recommendations.
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Oxbryta® significantly improves haemoglobin levels in paediatric sickle cell disease patients
Data shows treating sickle cell disease patients aged four to 11 years with Oxbryta® (voxelotor) results in increases in haemoglobin associated with improved clinical status.
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CRISPR therapies could be one of the most transformative in human history, says GlobalData
Adam Pearson, Senior Oncology Analyst at GlobalData, comments on the development of CRISPR-based therapies and outlines potential opportunities and challenges in the space.
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Gates Foundation to fund Novartis’ development of a sickle cell disease gene therapy
The Gates Foundation will fund Novartis’ discovery and development of a gene therapy for sickle cell disease that is accessible for low- and middle-income countries.
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DECODR app identifies unintentional DNA mutations from CRISPR gene therapies
The DECODR app was developed to accelerate the development of CRISPR gene therapies, producing similar data as deep sequencing processes in a shorter timeframe and at less cost.
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EMA accepts MAA for Oxbryta to treat haemolytic anaemia in sickle cell disease
The EMA has accepted Global Blood Therapeutics' marketing authorisation application for Oxbryta (voxelotor) to treat haemolytic anaemia in sickle cell disease.
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EMA recommends 11 new medicines for approval following July meeting
Dapivirine Vaginal Ring (dapivirine) and Blenrep (belantamab mafodotin) have been recommended for approval by the EMA's CHMP, along with nine other medicines.
