WHAT YOU NEED TO KNOW | EMA Recommends Suspension of Pfizer’s Sickle Cell Disease Drug Oxbryta
In a significant development for the treatment of sickle cell disease (SCD), the European Medicines Agency (EMA) recommended on September 26, 2024, that the marketing authorization for Pfizer’s drug Oxbryta (voxelotor) be suspended. This announcement came on the heels of Pfizer’s decision to withdraw Oxbryta from all markets where it had been approved, citing severe safety concerns.
Oxbryta, which was designed to treat sickle cell disease, has been a crucial medication for patients with mild-to-moderate symptoms. Approved in the US under an accelerated process in 2019 and later in the EU in February 2022, the drug initially showed promise in alleviating the painful complications associated with SCD. However, recent clinical data has cast doubt on its overall safety and efficacy.
On Wednesday, Pfizer announced that it would be halting the distribution of Oxbryta worldwide. The decision was based on new clinical data that indicated a troubling imbalance of deaths and vaso-occlusive crises (VOCs) among patients taking the medication. VOCs are severe episodes of pain caused by the obstruction of blood vessels, a common and debilitating complication of sickle cell disease.
The EMA’s call to suspend the drug’s use was influenced by emerging safety data from two registry-based studies. These studies revealed that patients experienced a higher occurrence of VOCs during treatment with Oxbryta than they did before starting the medication. Given the severity of these findings, the EMA concluded that the risks associated with Oxbryta now outweigh its benefits.
Clinical Trials and Expanded Access Programs Halted:
In its official statement, Pfizer confirmed that it would stop all clinical trials and expand access programs for Oxbryta globally. The pharmaceutical giant emphasized the need for continued safety analysis and review of the drug’s findings to ensure patient safety.
Alternatives and Future Directions:
The suspension of Oxbryta leaves a significant gap in the treatment landscape for sickle cell disease. While there are alternative therapies, they come with their own challenges. For instance, both the FDA and the EMA approved new SCD and thalassaemia gene-editing treatment Casgevy™ (exa-cel or exagamglogene autotemcel) by Vertex Pharmaceuticals and CRISPR Therapeutics, and the FDA has also authorised Lyfgenia™ (lovo-cel or lovotibeglogene autotemcel) by Bluebird Bio. Lyfgenia has been given orphan drug and priority medicines designations in Europe.
These therapies, which utilize groundbreaking gene-editing technologies, offer hope but also have substantial drawbacks. They are extremely costly and involve a lengthy testing and preparatory process, resulting in slow patient uptake.
What Should Patients Do?
Pfizer has advised patients currently taking Oxbryta to consult with their healthcare providers to explore alternative treatment options. It is crucial for patients to have informed discussions with their doctors to determine the best course of action moving forward.
For additional information or concerns about Oxbryta, patients, physicians, pharmacists, and other healthcare professionals can contact Pfizer Medical Information at 1-800-438-1985.
The suspension of Oxbryta marks a significant moment in the ongoing battle against sickle cell disease. As the medical community continues to seek effective treatments for this debilitating condition, patient safety remains paramount. The recent developments serve as a reminder of the complexities and challenges involved in drug development and the importance of rigorous clinical evaluation.
