Table 3 Summary of key features and efficacy outcomes of completed fedratinib phase II and III clinical trials.
Trial | Arms | Line of therapy | Number of patients | Comparator | Duration of disease or ruxolitinib treatment prior to fedratinib treatment (months, median and inter-quartile range) | SVR35% at 24 weeks | TSS > 50% at 24 weeks | Adverse events | Reference |
|---|---|---|---|---|---|---|---|---|---|
JAKARTA | Fed 400 mg Fed 500 mg Placebo | First | 289 | None | Not relevant | Fed 400 mg 35% Fed 500 mg 40% Placebo 1% | Fed 400 mg 35% Fed 500 mg 34% Placebo 7% | Fed 400 mg: GI – diarrhea, vomiting, nausea ~55% Hematologic (commonest) – anemia 99% any grade, 43% grade 3–4 14% of patients discontinued treatment because of AE | [6] |
JAKARTA2 | Fed 400 mg | Second | 97 | None | Duration of disease = 49 (35.2–62.7) Duration of ruxolitinib treatment = 10.25 (5.75–14.75) | Per protocol cohort 55% ITT cohort 31% Stringent cohort 30% | Per protocol cohort 26% ITT cohort 27% | GI – diahrrea, vomiting, nausea ~50% Hematologic (commonest) – anemia 48% any grade, 38% grade 3–4 19% of patients discontinued treatment because of AE, mostly thrombocytopenia | |
FREEDOM | Fed 400 mg | Second | 38 | None | Duration of disease = 40.2 (14.4–75.6) | 26% | 44% | GI – 89% any grade (mostly constipation) Hematologic (commonest) – anemia 60% any grade, 40% grade 3–4 | [11] |
FREEDOM2 | Fed 400 mg | Second | 201 | BAT | Duration of disease = 43.4(13.5–84.5) Duration of ruxolitinib treatment = 21.1 (10.0–53.5) | Fed 400 mg 36% BAT 6% | Fed 400 mg 34% BAT 17% | GI - 56% any grade in Fed vs 9% BAT, mainly diarrhea Hematologic (mainly anemia and thrombocytopenia) – 26% in Fed vs 18% in BAT 10% of patients in Fed group vs 6% in BAT discontinued treatment because of AE | [12] |
