MHLW (Japan) approval of Pemazyre for the treatment of patients with myeloid/lymphoid neoplasms

MLNs are a rare, aggressive group of cancers characterized by an over-production of myeloid cells, or bone tissue, with the tendency to rapidly progress to an acute myeloid leukemia (AML).

“The MHLW approval of Pemazyre in MLNs is an important step toward potentially providing a therapeutic option for Japanese patients with this rare condition,” said Lothar Finke, M.D., Ph.D., Group Vice President and General Manager, Incyte Asia. “Bringing this first and only approved therapy to MLN patients in Japan demonstrates Incyte’s commitment to finding solutions for critical unmet medical needs regardless of the size of the patient population – as is the case with MLNs, an extremely rare disease that is estimated to affect fewer than 100 patients worldwide.”

The approval was based on data from the Phase II FIGHT-203 study, a multicenter open-label, single-arm trial that evaluated the safety and efficacy of Pemazyre in 41 patients in myeloid or lymphoid neoplasms with FGFR1 fusion gene positive who received Pemazyre 13.5 mg orally once daily continuously or intermittently. The primary endpoint, investigator-assessed complete response rate, was 62.5% (95% CI: 45.8 - 77.3). The complete response rate in the continuous dosing population was 66.7% (95% CI: 46.0 - 83.5). The most common adverse reactions observed in patients receiving Pemazyre were hyperphosphatemia (70.7%), alopecia (56.1%), diarrhea (43.9%) and stomatitis (43.9%).