Following reports of serious adverse reactions, and pending full evaluation of the efficacy and safety data, the European Commission has suspended the marketing authorization (MA) of the medicine Oxbryta (voxelotor) in in charge of hemolytic anemia (by excessive destruction of red blood cells) in patients with sickle cell disease. It is up to doctors to plan how to stop treatment and to consider available therapeutic alternatives. Knowing that no initiation of treatment is any longer possible, the National Medicines Safety Agency (ANSM) has established the course of action to follow. [1].
Increased risk of death
Oxbryta has had European Marketing Authorization since February 14, 2022 for the treatment of hemolytic anemia (by excessive destruction of red blood cells) in patients with sickle cell disease. In France, it is available via derogatory access (early post-MA access), as well as as part of a clinical trial.
Since the end of July 2024, Oxbryta has been the subject of a European re-evaluation of the benefit/risk balance of this medicine. Indeed, clinical trials carried out in several countries outside Europe have shown an increased risk of death during treatment with Oxbryta. Additionally, new data from the United States have demonstrated an increased frequency of vaso-occlusive crises in patients treated with Oxbryta.
In this context, the European Commission suspended the Marketing Authorization of Oxbryta on October 4, 2024, pending the conclusion of the CHMP re-evaluation.
A letter to the healthcare professionals concerned was sent by the Pfizer laboratory, holder of the Marketing Authorisation. All patients treated with Oxbryta in France have been contacted and informed or will be informed shortly. It is up to their doctor to plan with them how to stop treatment and consider available therapeutic alternatives.
All batches of this medication still in stock in hospital pharmacies (PUI) will be recalled.
To deal with this suspension, the ANSM has developed procedures to follow in consultation with the specialist doctors concerned and patient associations (see box below).
Guidelines for prescribing doctors
No further treatment of a patient with Oxbryta can be initiated due to the suspension of Marketing Authorization.
If some of your patients are being treated with Oxbryta, contact them to explain how to stop their treatment, consider a therapeutic alternative as well as close therapeutic monitoring.
It cannot be ruled out that abrupt cessation of Oxbryta could lead to a rebound of the disease and increase the risk of vaso-occlusive crises (VOC): remind your patients of the main triggering factors. Encourage them to see you immediately or contact an emergency service if the first signs appear.
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