Lhe Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended, as a precaution, the suspension of the marketing authorization (MA) for OXBRYTA 500 mg film-coated tablet ( voxelotor – cf. Boxed) [1].
At the same time, the Pfizer laboratory decided to “withdraw and recall this medicine from all countries where it is available, and to stop ongoing clinical trials, compassionate use and early access programs”, indicates the European Agency.
| Treatment of hemolytic anemia caused by sickle cell disease in adults and adolescents aged 12 years and older, as monotherapy or in combination with hydroxyurea. |
EMA reviews OXBRYTA safety data
Both decisions result from new safety data suggesting a higher frequency of vaso-occlusive crises (VOC) during treatment with OXBRYTA, compared to before treatment initiation. “Vaso-occlusive crises are among the most common complications of sickle cell disease; They involve episodes of acute pain and can lead to other health complications, such as arthritis, kidney failure and stroke.”recalls the EMA which indicates that it is continuing to re-evaluate the benefit/risk ratio of this medicine.
This re-evaluation was initiated in July 2024 when data from a clinical trial conducted versus placebo showed that more deaths occurred in patients treated with OXBRYTA, and another trial showed that the total number of deaths was higher than expected.
“Overall, these data raise serious concerns about the safety of OXBRYTA”summarizes the European Agency.
Discontinuation of treatment and patient monitoring
Pending its conclusions, the EMA recommends that doctors:
- not to start administering OXBRYTA to new patients;
- to contact patients currently being treated with OXBRYTA to discontinue treatment and discuss alternative treatment options;
- to continue monitoring patients for possible adverse effects after stopping OXBRYTA.
In France, OXBRYTA was available under an early access authorization (AAP) from 2022 to September 2024. This AAP was stopped on September 21, 2024; a decree published in Official Journal from September 20, 2024 [2] put an end to the reimbursement of OXBRYTA under its AAP, at the request of Pfizer to waive coverage under common law.
