The European Medicines Agency (EMA) has urged the suspension of marketing authorization for Pfizer’s drug Oxbryta, a treatment for sickle cell disease (SCD), following the pharmaceutical company’s decision to voluntarily withdraw the medication from global markets.
The announcement from the EMA came shortly after Pfizer declared its intention to discontinue Oxbryta due to safety concerns. The agency’s human medicines committee (CHMP) recommended the immediate suspension, emphasizing that this precautionary measure is necessary as they continue to evaluate emerging safety data related to the drug.
Oxbryta, designed to treat SCD—a serious genetic blood disorder predominantly affecting individuals of African, Middle Eastern, and South Asian descent—has come under scrutiny after two studies indicated an increased incidence of vaso-occlusive crises among patients using the medication. These crises can lead to severe pain and potential complications such as arthritis, kidney failure, and stroke.
The EMA’s ongoing investigation into Oxbryta began in July after troubling clinical trial data revealed a concerning trend: the number of deaths in patients receiving Oxbryta exceeded that of those receiving a placebo. The findings prompted the EMA to further assess the drug’s safety profile.
In its statement, the EMA advised healthcare professionals to refrain from initiating any new treatments with Oxbryta and to inform patients currently using the medication to discontinue its use. Physicians are also advised to monitor patients for any adverse effects after stopping the treatment.
Sickle cell disease results from inherited mutations that lead to abnormal hemoglobin production, causing red blood cells to become rigid and shaped like a sickle. This abnormality impairs blood flow, leading to pain and a variety of serious complications, including infections and acute chest syndrome.
In light of these developments, Pfizer stated that its decision to withdraw Oxbryta was based on a comprehensive review of the clinical data, which indicated that the overall benefits of the drug no longer outweighed the associated risks for patients with sickle cell disease.
