August 21, 2025
Source: drugdu
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Recently, Pfizer announced that its key sickle cell disease drug inclacumab failed to reach the primary endpoint in the phase III THRIVE-131 study. This news casts a shadow over Pfizer's layout in this field.
The Phase III THRIVE-131 study was designed to evaluate the effect of inclacumab in reducing vaso-occlusive crises. The results of the study showed that there was no significant difference in the incidence of vascular occlusive crisis between the inclacumab group and the placebo group, and inclacumab failed to achieve a statistically significant effect.
Sickle cell disease (SCD) is a hereditary blood disorder in which red blood cells are sickle-shaped due to abnormalities in hemoglobin molecules, affecting blood flow and oxygen delivery. Patients often have symptoms such as pain and anemia, and may face many difficulties in life due to sudden illness, which brings a heavy burden to patients and families.
Looking back at Pfizer's layout of SCD treatment, in August 2022, Pfizer acquired Global Blood Therapeutics (GBT) with a total transaction volume of US $5.4 billion. It is GBT's three sickle cell disease drugs including inclacumab that it is interested in.
Unfortunately, the other two drugs, oxbryta and osivelotor, also failed to achieve the desired results.
Oxbryta (voxelotor) is a hemoglobin S allosteric modifier that was approved by the FDA for the treatment of SCD in 2019. It was an important breakthrough in the field of SCD at that time. However, in September 2024, Pfizer announced the global withdrawal of oxbryta. The reason is that relevant clinical studies and registration data show that the mortality rate of patients in the oxbryta group is 3 times higher than that in the placebo group, which makes the drug terminate its clinical application due to poor risk-benefit ratio.
Another drug, osivelotor (GBT60110), is a next-generation hemoglobin S polymerization inhibitor and an asset acquired through Pfizer'S acquisition of GBT. Osivelotor showed some potential in early studies, but in December 2024, Phase III studies of the drug were partially suspended as the FDA raised concerns about its safety. In May 2025, news confirmed that its Phase III study had been completely suspended.
These setbacks encountered by Pfizer in the field of SCD treatment highlight the complexity and challenges of SCD drug development.
Regarding the current status of the development of sickle cell disease treatment drugs, although the development of inclacumab and other drugs has encountered setbacks, the global research and development of sickle cell disease treatment drugs is still continuing, which may bring a new dawn to patients.
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