Pfizer will voluntarily pull its sickle cell disease (SCD) drug Oxbryta from all markets worldwide, a major blow to a $5.4 billion acquisition the New York pharma made two years ago.
Pfizer will stop distributing Oxbryta, call back all produced lots and discontinue all active clinical trials and expanded access programs for the drug, the company said in a release after market close on Wednesday.
The decision is based on new clinical information that “now indicates the overall benefit of Oxbryta no longer outweighs the risk” in its approved SCD indication.
Oxbryta secured an FDA accelerated approval in late 2019 and a marketing authorization from the European Commission in 2022 based on data showing it was associated with statistically significant improvements in patients’ hemoglobin levels compared with a dummy drug. At the time, the oral therapy was touted as the first approved treatment to directly address the root cause of SCD.
Now, though, Pfizer said newer patient outcomes data didn’t favor Oxbryta. Specifically, the data suggested “an imbalance” in vaso-occlusive crises and deaths that “require further assessment," Pfizer said.
“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, M.D., chief medical officer and head of worldwide medical and safety at Pfizer, said in the press release.
The drug was the centerpiece in Pfizer’s $5.4 billion acquisition of Global Blood Therapeutics in 2022 during a post-COVID M&A spree for the drugmaker. At that time, Pfizer said Oxbryta and GBT’s SCD pipeline had the potential to achieve combined worldwide peak sales of more than $3 billion.
Earlier this year, Pfizer ended one of two late-stage SCD trials of a legacy GBT candidate, a P-selectin inhibitor called inclacumab, because of patient recruitment challenges.
Last year, Oxbryta brought in $328 million in global sales. Pfizer said it does not expect the market removal will impact its 2024 financial guidance.
Pfizer 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.
Oxbryta’s retreat comes after the recent FDA approvals of two gene therapies for SCD: Vertex and CRISPR Therapeutics’ Casgevy and bluebird bio’s Lyfgenia. Those approvals were based on clinical data showing those one-time therapies can reduce or eliminate vaso-occlusive crises for patients with SCD.