In a news release published yesterday, Pfizer announced it will voluntarily withdraw its sickle cell disease (SCD) therapy Oxbryta (voxelotor) from all markets worldwide.
The company said it is also discontinuing all active clinical trials for voxelotor and expanded access programs around the world.
Pfizer explained that it made its decision based on a full review of clinical data that indicates the “overall benefit of Oxbryta no longer outweighs the risk in the approved sickle cell patient population.”
According to the data, there appears to be an imbalance in vaso-occlusive crises and fatal events that “require further assessment.”
Pfizer said it has notified regulatory authorities about these findings and its decision to voluntarily withdraw Oxbryta from the market, as well as “discontinue distribution and clinical studies while further reviewing the available data and investigating the findings.”
This is a significant turn of events as Pfizer acquired the drug in the $5.4 billion acquisition of Global Blood Therapeutics (GBT) in 2022. At the time, Pfizer projected that Oxbryta and GBT’s SCD pipeline could hit combined worldwide sales of more than $3 billion.
Oxbryta is currently authorized in more than 35 countries around the world.
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“The safety and well-being of patients is of the utmost importance to Pfizer, and we believe this action is in the best interest of patients,” Aida Habtezion, MD, chief medical officer and head of worldwide medical and safety at Pfizer, said in the news release.
Oxbryta is an oral, once-daily therapy in tablet form for the treatment of SCD. The drug works by increasing hemoglobin’s affinity for oxygen. It inhibits sickle hemoglobin polymerization and the resultant sickling and destruction of red blood cells leading to hemolysis and hemolytic anemia, which are primary pathologies faced by people with SCD.
Oxbryta received accelerated approval in 2019 from the US Food and Drug Administration (FDA) for the treatment of SCD in adults and children ages 12 years and older. In December 2021, the agency approved expanded use of the treatment for SCD patients four years of age and older.
It also received marketing authorization from the European Commission in February 2022 based on data that showed it could significantly improve hemoglobin levels compared with a placebo. It was indicated for pediatric patients 12 years of age and older either for use as a monotherapy or in combination with hydroxycarbamide (hydroxyurea).
The therapy was hailed to be the first approved treatment to address the root cause of SCD at the time of the approval.
Pfizer said it has notified regulatory authorities of the market withdrawal. The company is still investigating the new clinical findings, but it advises patients to contact their physicians to discuss alternative treatment options, Habtezion said.
The FDA recently approved two gene therapies for SCD: Vertex and CRISPR Therapeutics’ Casgevy and bluebird bio’s Lyfgenia. Their approvals were based on clinical data that showed the therapies can reduce or eliminate vaso-occlusive crises in SCD patients.
In 2023, Oxbryta earned $328 million in global sales. Pfizer said it does not expect pulling Oxbryta from the market will affect its “full-year 2024 financial guidance.”
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