Biopharmaceutical company Incyte (Nasdaq:INCY) disclosed on Monday that the Japanese Ministry of Health, Labour and Welfare (MHLW) has approved Pemazyre (pemigatinib), a selective fibroblast growth factor receptor (FGFR) inhibitor used to treat myeloid/lymphoid neoplasms (MLNs) with FGFR1 fusion (also known as 8p11 myeloproliferative syndrome).

The company said that MLNs are a rare, aggressive group of cancers characterised by an over-production of myeloid cells, or bone tissue, that can tend to rapidly progress to an acute myeloid leukemia (AML).

This Japanese approval was based on data from the Phase 2 FIGHT-203 study, a multicentre 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 13.5mg orally once daily continuously or intermittently.

Pemazyre was previously granted Orphan Drug Designation (ODD) by the MHLW. The drug is indicated in the United States for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test. It is also the first targeted treatment approved for use in the United States for treatment of adults with relapsed or refractory myeloid/lymphoid neoplasms (MLNs) with FGFR1 rearrangement.