The European Medicines Agency (EMA) has recommended suspending the approval of Pfizer’s sickle cell disease (SCD) treatment, Oxbryta, advising healthcare professionals to cease its use following emerging safety concerns. This decision aligns with Pfizer’s recent announcement to voluntarily withdraw the drug from all global markets.
Oxbryta, used to treat the life-threatening blood disorder that disproportionately affects individuals of African, Middle Eastern, and South Asian descent, is under investigation after clinical trials raised alarms over its safety profile. The EMA’s Human Medicines Committee (CHMP) initiated a review in July after trial data indicated a higher-than-expected number of deaths among Oxbryta users compared to those receiving a placebo.
The EMA’s statement on Thursday outlined its concerns: “This measure is taken as a precaution while a review of emerging data is ongoing.” The primary worry stems from two separate studies, which highlighted an increased occurrence of “vaso-occlusive crises” — painful episodes caused by blocked blood vessels that can lead to severe complications like kidney failure, stroke, and arthritis. These findings prompted regulators to act swiftly to prevent further risks to patients.
Pfizer, a leading global pharmaceutical company, confirmed on Wednesday that it would withdraw Oxbryta from all markets where it has received approval. The company emphasized that its decision was made after considering the cumulative clinical data, which now shows that the risks of using Oxbryta outweigh its benefits for patients with SCD.
Sickle cell disease is a group of inherited blood disorders that result in abnormal hemoglobin, the protein responsible for carrying oxygen throughout the body. This abnormality causes red blood cells to take on a sickle shape, which restricts normal blood flow, leading to significant health complications such as pain, infections, acute chest syndrome, and stroke.
As part of the EMA’s latest recommendation, doctors are urged to halt new treatments involving Oxbryta and advise patients currently on the medication to stop its use. “Healthcare professionals should monitor patients for adverse events after discontinuation of the treatment,” the EMA stated.
The EMA’s investigation into the safety risks associated with Oxbryta is still underway, and further findings are expected as the regulatory body continues to assess the drug’s long-term impact.
For those affected by sickle cell disease, this decision underscores the importance of ongoing research and the need for safe and effective treatment options. As the medical community continues to evaluate the best approach for managing SCD, patients are advised to consult their healthcare providers for guidance on alternative treatments.
Pfizer’s voluntary withdrawal marks a significant shift in the landscape of sickle cell treatment, raising questions about the future of therapies targeting this debilitating disorder.
