![]() ![]() The decision to authorise Casgevy has been endorsed by the government’s independent scientific advisory committee, the Commission on Human Medicines, after a robust review of the available evidence. ![]() After that, patients may need to spend at least a month in a hospital facility while the treated cells take up residence in the bone marrow and start to make red blood cells with the stable form of haemoglobin. Patients must then undergo conditioning treatment to prepare the bone marrow before the modified cells are infused back into the patient. AdministrationĬasgevy is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory. Safety continues to be closely monitored by the MHRA and the manufacturer.īoth trials are ongoing and further results will be made available in due course. No significant safety concerns were identified during the trials. ![]() Side effects from treatment were similar to those associated with autologous (from a person’s own cells) stem cell transplants, including (but not limited to) nausea, fatigue, fever and increased risk of infection. The remaining three had more than a 70% reduction in the need for red cell transfusions. Of these, 39 (93%) did not need a red blood cell transfusion for at least 12 months after treatment. In the clinical trial for transfusion-dependent β-thalassemia, 54 patients have currently received Casgevy but only 42 patients have been in the trial long enough to be eligible for the primary efficacy interim analysis. Of these eligible patients, 28 (97%) were free of severe pain crises for at least 12 months after treatment. In the clinical trial for sickle-cell disease, 45 patients have currently received Casgevy but only 29 patients have been in the trial long enough to be eligible for the primary efficacy interim analysis. There are limited medicines currently available to patients, so I welcome today’s news that a new treatment has been judged safe and effective, which has the potential to significantly improve the quality of life for so many. ![]() Sickle cell disorder is an incredibly debilitating condition, causing significant pain for the people who live with it and potentially leading to early mortality. John James OBE, Chief Executive of the Sickle Cell Society said: I would like to thank the patients with lived experiences who engaged with us as part of the assessment process and gave us valuable insight into their lives and the challenges of managing their condition. The MHRA will continue to closely monitor the safety and effectiveness of Casgevy, through real-world safety data and post-authorisation safety studies being carried out by the manufacturer. I am pleased to announce that we have authorised an innovative and first-of-its-kind gene-editing treatment called Casgevy, which in trials has been found to restore healthy haemoglobin production in the majority of participants with sickle-cell disease and transfusion-dependent β -thalassaemia, relieving the symptoms of disease. To date, a bone marrow transplant – which must come from a closely matched donor and carries a risk of rejection – has been the only permanent treatment option. Julian Beach, Interim Executive Director of Healthcare Quality and Access at the MHRA said:īoth sickle cell disease and β-thalassemia are painful, life-long conditions that in some cases can be fatal. To do this, stem cells are taken out of bone marrow, edited in a laboratory and then infused back into the patient after which the results have the potential to be life-long. Patients often need a blood transfusion every 3 to 5 weeks, and injections and medicines throughout their lives.Ĭasgevy is designed to work by editing the faulty gene in a patient’s bone marrow stem cells so that the body produces functioning haemoglobin. In people with β-thalassaemia, it can lead to severe anaemia. In people with sickle cell disease, this genetic error can lead to attacks of very severe pain, serious and life-threatening infections, and anaemia (whereby your body has difficulty carrying oxygen). β-thalassemia mainly affects people of Mediterranean, south Asian, southeast Asian and Middle Eastern origin. Sickle cell disease is particularly common in people with an African or Caribbean family background. A new treatment for sickle-cell disease and transfusion-dependent β-thalassemia has been authorised by the Medicines and Healthcare products Regulatory Agency (MHRA) for patients aged 12 and over after a rigorous assessment of its safety, quality and effectiveness.Ĭasgevy is the first medicine to be licensed that uses the innovative gene-editing tool CRISPR, for which its inventors were awarded the Nobel Prize in 2020.īoth sickle cell disease and β-thalassemia are genetic conditions caused by errors in the genes for haemoglobin, which is used by red blood cells to carry oxygen around the body. ![]()
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