On September 25, 2024, Pfizer Inc. announced the voluntary withdrawal of all lots of its sickle cell disease (SCD) treatment from global markets. Pfizer is halting the distribution and discontinuing all clinical trials and expanded access programs involving the treatment. This decision follows a thorough review of clinical data indicating that the risks, including an imbalance in vaso-occlusive crises and fatal events, now outweigh the benefits for patients.

Key Findings and Pfizer’s Response
Recent patient outcome data revealed concerning risks, prompting Pfizer to notify regulatory authorities and take precautionary action. “The safety and well-being of patients is of the utmost importance to Pfizer,” stated Aida Habtezion, Chief Medical Officer at Pfizer. While the treatment had been seen as a promising therapy for SCD patients, Pfizer believes that this withdrawal is in the best interest of patients and advises them to consult their physicians for alternative treatments.
Background on SCD and the Treatment
The treatment, approved by the FDA in 2019, was designed to inhibit sickle hemoglobin polymerization and reduce red blood cell sickling, aiming to address hemolytic anemia in SCD patients. The drug had shown promising results in increasing hemoglobin levels, with approvals in over 35 countries. However, the latest clinical findings have shifted the risk-benefit balance, prompting this worldwide withdrawal.
Sickle cell disease is a serious genetic disorder characterized by abnormal red blood cells, leading to inflammation, anemia, and potentially life-threatening complications like strokes and acute pain crises. With approximately 4.5 million people globally affected by SCD, effective treatments remain a critical need.
Despite this setback, Pfizer has stated that it does not expect the withdrawal of this treatment to impact its 2024 financial guidance. The company will continue investigating the clinical data and work closely with patients, physicians, and regulators to determine the next steps.

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