Mouse models of JAK2V617F-driven myeloproliferative disease: survival up to 100% at 42 days; reduced WBC, hematocrit, splenomegaly, fibrosis
“In vivo studies in mouse models of JAK2V617F-driven myeloproliferative disease showed that twice daily oral doses of fedratinib at 60 or 120 mg/kg blocked phosphorylation of STAT3/5, increased survival to 100% at the highest dose after 42 days of treatment, and improved disease-associated symptoms, including reduction of white blood cells, hematocrit, splenomegaly, and fibrosis.”
✓ VerifiedFDA multi-discipline review, p. 8Source ↗
Why that model
Not stated in public reviews
Not reported in the public reviews. Reviewers summarise selectively, so this usually means it wasn't discussed publicly, not that it wasn't studied.
Reviewer's view of the efficacy data
EMA: proof of concept shown, but mouse efficacy required exposures higher than those reached in humans
“Numerous in vivo mouse models were used to evaluate the efficacy of fedratinib. Although proof of concept is shown in these studies, it appears that in mice a higher exposure to fedratinib than the exposure reached in humans is required to result in an effect on tumour cells.”