Pfizer withdraws all lots of OXBRYTA (voxelotor)

Pfizer has voluntarily withdrawn all lots of OXBRYTA (voxelotor), a treatment for sickle cell disease (SCD), from the market due to concerns over the safety data, which suggests an increased risk of vaso-occlusive crises and fatal events. Pfizer also discontinued all clinical trials and expanded access programs for the drug. The company advises patients to consult their physicians for alternative treatments while they further investigate these findings. The financial impact is not expected to affect Pfizer’s 2024 guidance.

At this time, there do not appear to be any widely reported lawsuits directly related to Pfizer’s voluntary withdrawal of Oxbryta (voxelotor) for sickle cell disease (SCD). However, this recent withdrawal, driven by concerns over safety data—including a higher-than-expected number of deaths and vaso-occlusive crises in clinical trials—has raised significant concern among patients and healthcare providers. The European Medicines Agency disclosed that 16 out of 18 deaths in two trials were among patients who received Oxbryta, though there’s no definitive proof linking the drug directly to these deaths?(GEN News)?(The Pharmaceutical Journal).

While no lawsuits have been widely publicized yet, given the nature of the drug’s withdrawal and the clinical trial outcomes, litigation could potentially emerge, particularly from affected patients or their families. For now, Pfizer has emphasized patient safety as its top priority and has advised SCD patients to seek alternative treatments?(

Voxelotor (Oxbryta) was approved in several countries, primarily for the treatment of sickle cell disease (SCD) in patients 12 years and older. The countries where it was approved include:

• United States: The FDA granted approval in November 2019 as a treatment for sickle cell disease?(Pharmacy Times).
• European Union: The European Medicines Agency (EMA) approved voxelotor for use in the EU?(The Pharmaceutical Journal).
• United Kingdom: Approval followed EU standards due to its initial approval under EMA regulations?(harmacy Times).
• Middle Eastern and African countries: The drug was tested in several Phase III trials in countries including Egypt, Ghana, Kenya, Nigeria, Oman, and Saudi rabia?(GEN News).

These approvals were granted based on the drug’s ability to inhibit hemoglobin S polymerization, a central mechanism of sickle cell disease. However, the recent safety concerns and the voluntary withdrawal of Oxbryta from global markets, including these regions, reflect a reevaluation of its risk-benefit profile.

There is limited publicly available data on the exact number of people who took voxelotor (Oxbryta) in the United States. However, since its approval in 2019, voxelotor was widely prescribed for sickle cell disease, particularly in patients aged 12 years and older. It was considered a groundbreaking therapy because it targeted the underlying cause of sickle cell disease by inhibiting hemoglobin S polymerization.

Although no precise patient count has been officially reported by Pfizer, the drug’s approval under the FDA’s Priority Review status and its role as one of the few available treatments for SCD suggest that a significant number of patients likely received voxelotor between 2019 and 2024.

According to multiple reports, a total of 16 deaths were associated with the use of voxelotor (Oxbryta) in clinical trials for sickle cell disease. This was disclosed by the European Medicines Agency (EMA). These deaths occurred across two Phase III trials, and most of the patients who died had been receiving voxelotor:

• In the GBT440-032 (HOPE Kids 2) trial, there were 8 deaths in patients treated with voxelotor, compared to 2 deaths in the placebo group.
• In the GBT440-042 (RESOLVE) trial, there were 8 additional deaths in the open-label portion of the study, with most of the fatalities involving patients on voxelotor?(GEN News)?(The Pharmaceutical Journal)?(Pharmacy Times).

It’s important to note that the EMA has indicated that there is no definitive evidence that voxelotor directly caused these deaths, and some of the deaths were potentially linked to infections like malaria?(GEN News)?(Pharmacy Times). The imbalance in adverse events, including vaso-occlusive crises, led to the voluntary withdrawal of the drug.