Incyte Announces Japanese Approval of Pemazyre® (pemigatinib) for the Treatment of Patients with Myeloid/Lymphoid Neoplasms (MLNs)

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[{"type":"text","content":" WILMINGTON, Del. & TOKYO--(BUSINESS WIRE)--\nIncyte (Nasdaq:INCY) today announced that the Japanese Ministry of Health, Labour and Welfare (MHLW) has approved Pemazyre® (pemigatinib), a selective fibroblast growth factor receptor (FGFR) inhibitor, for the treatment of myeloid/lymphoid neoplasms (MLNs) with FGFR1 fusion (also known as 8p11 myeloproliferative syndrome). 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).\n\n“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.”\n\nThe approval was based on data from the Phase 2 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%).\n\nPreviously, the MHLW granted Orphan Drug Designation (ODD) for Pemazyre – a designation granted to investigational compounds intended to treat rare diseases that affect fewer than 50,000 people in Japan, and for which there is a high medical need1. Designated orphan drugs are also eligible for priority review for marketing authorizations to ensure supply to clinical settings at the earliest opportunity1. The MLN ODD is the second such designation granted to Pemazyre by the MHLW, having also received ODD for cholangiocarcinoma, a type of bili...

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