Sickle cell drug is withdrawn over safety concerns just months after rollout

The sickle cell treatment voxelotor (Oxbryta) has been withdrawn from all markets by the manufacturer Pfizer after a “higher than anticipated” number of deaths were reported and clinical evidence showed that the drug’s benefits no longer outweighed the risks.

In a recall notice issued on 30 September the UK Medicines and Healthcare Products Regulatory Agency (MHRA) told clinicians and patients that “emerging data” suggested an “unfavourable imbalance in vaso-occlusive crises (acute painful crises) and fatal events (deaths)” in patients treated with the drug.

The agency shared a letter from Pfizer with UK healthcare professionals, which said that all patients currently having voxelotor prescribed must be contacted and advised to discontinue treatment and, where appropriate, discuss alternative treatment options. Patients should be instructed to return the product to the hospital pharmacy or homecare company that dispensed it.

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Source: BMJ, 1 October 2024