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Pfizer has withdrawn voxelotor (Oxbryta) worldwide for the treatment of sickle cell disease (SCD), owing to the emergence of clinical data that links “fatal events” to the drug.
In a statement published on 25 September 2024, the company said that its decision was “based on the totality of clinical data that now indicates the overall benefit of Oxbryta no longer outweighs the risk in the approved sickle cell patient population”.
“The data suggest an imbalance in vaso-occlusive crises and fatal events, which require further assessment,” the statement added.
Vaso-occlusive crises occur when red blood cells block blood flow to tissue in SCD patients.
“Pfizer has notified regulatory authorities about these findings and its decision to voluntarily withdraw Oxbryta from the market and discontinue distribution and clinical studies, while further reviewing the available data and investigating the findings.”
The Medicines and Healthcare products Regulatory Agency published a recall notice on 30 September 2024 for voxelotor, which it said would “reinforce action already being undertaken by Pfizer Ltd. and ensures that healthcare professionals in NHS trusts and other care settings take appropriate action to recall all batches from patients as soon as possible”.
The notice said that healthcare professionals should stop supplying voxelotor immediately and contact all patients undergoing treatment and advise them to discontinue treatment.
Where appropriate, the MHRA advised healthcare professionals to discuss alternative treatment options.
The MHRA said patients should be instructed to return the product to the hospital pharmacy or homecare company that dispensed it.
Patients should also continue to be monitored for adverse events after their treatment with voxelotor is discontinued, it said.
The decision follows the National Institute for Health and Care Excellence’s (NICE) recommendation of voxelotor as a treatment for SCD in May 2024, reversing its decision in draft guidance published earlier in the year.
At the time, NICE said it was able to make the positive recommendation on the new treatment following a price reduction provided by the manufacturer after the initial consultation.
Commenting on the drug’s withdrawal, Aida Habtezion, chief medical officer and head of worldwide medical and safety at Pfizer, said: “The safety and wellbeing of patients is of the utmost importance to Pfizer, and we believe this action is in the best interest of patients.
“Our primary concern is for patients who suffer from SCD, which remains a very serious and difficult-to-treat disease with limited treatment options. We advise patients to contact their physicians to discuss alternative treatment while we continue to investigate the findings from our review of the data.”
In a statement published on 26 September 2024, the Sickle Cell Society said: “We are deeply shocked and extremely disappointed by Pfizer’s sudden decision to withdraw voxelotor (Oxbryta) from the NHS in the UK, and across the world. We were given no warning.
“The withdrawal of voxelotor (Oxbryta) is a significant blow, particularly given that it follows the removal of crizanlizumab earlier this year, leaving patients with even fewer options to manage this painful, life-altering and debilitating condition.
“Today’s news effectively wipes out 20 years of progress in developing new treatments for sickle cell disorder.
“We understand that patient safety is paramount, but the lack of new, effective treatments for SCD over the past two decades has made it incredibly difficult for patients to manage their condition.
“Crucially, along with our clinical colleagues, we have not been afforded the opportunity to fully understand and discuss exactly what the safety issues are in this case.”
