The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended suspending the marketing authorization for Oxbryta (voxelotor), a treatment for sickle cell disease. This suspension is a precautionary measure due to concerns about new safety data, which are currently under review.
The decision comes as EMA evaluates emerging evidence that suggests potential risks associated with the medicine’s use, particularly a possible link to an increased frequency of vaso-occlusive crises (VOC) among patients.
The safety concerns stem from two registry-based studies that showed patients treated with Oxbryta experienced a higher rate of VOC compared to their condition prior to starting the medication. V
OC is a common and severe complication of sickle cell disease, marked by painful episodes that can lead to other serious health problems, including arthritis, kidney failure, and stroke. These findings raised alarms about the drug’s overall safety profile.
The review of Oxbryta was already underway after earlier clinical trial data indicated a higher-than-expected number of deaths among patients taking Oxbryta compared to those on a placebo.
The ongoing review, which began in July 2024, was prompted by this worrying trend. The cumulative evidence from both trials and recent registry data led the CHMP to conclude that the uncertainties surrounding Oxbryta’s safety warranted an immediate suspension.
In response to the CHMP’s recommendation, the company that markets Oxbryta has proactively chosen to recall the drug from all countries where it is distributed and to halt clinical trials, compassionate use programs, and early access initiatives. These steps are being taken as a precaution while the EMA conducts a thorough review of the available data. The company’s decision is aligned with the CHMP’s guidance to stop the use of the drug.
While the review is in progress, EMA has advised healthcare providers not to initiate Oxbryta treatment for new patients and to discontinue its use in current patients. Doctors are also encouraged to offer alternative treatments and monitor patients for any adverse effects after stopping Oxbryta.
Patients are urged to consult their physicians before discontinuing the medication to ensure proper medical guidance during the transition.
The EMA’s recommendation will be passed to the European Commission, which will issue a legally binding decision that will apply across all EU member states. Oxbryta, approved in February 2022 to treat haemolytic anaemia in sickle cell disease patients, is now under further scrutiny, with a final decision expected once all data are reviewed. The ongoing investigation aims to ensure the safety of patients and prevent further adverse outcomes associated with the medicine.
