NEW YORK – Incyte said on Monday that that the Japanese Ministry of Health, Labor, and Welfare approved Pemazyre (pemigatinib) for treatment of patients with myeloid or lymphoid neoplasms (MLNs) bearing FGFR1 fusions.
The agency's decision was based on the Phase II FIGHT-203 trial, in which Wilmington, Delaware-based Incyte established a complete response rate of 62.5 percent for patients with FGFR1-positive MLN treated with 13.5 mg of Pemazyre once daily. For patients who were dosed continuously, rather than intermittently, the complete response rate was 66.7 percent. Patients had to have an 8p11 translocation based on conventional cytogenetics or an FGFR1 rearrangement on break-apart fluorescence in situ hybridization testing.
Pemazyre inhibits FGFR 1, 2, and 3. The US Food and Drug Administration approved the drug in the same setting in August 2022. Pemazyre is also approved in the US and Europe for patients with locally advanced or metastatic cholangiocarcinoma with FGFR2 fusion or rearrangements that have progressed after at least one prior line of therapy. In the US, Foundation Medicine's FoundationOne CDx test is approved as a companion diagnostic to identify patients with bile duct cancer who are eligible for treatment with Pemazyre.
MLNs with FGFR1 rearrangements result from a chromosomal abnormality in which chromosome 8 breaks at position 11, where the FGFR1 gene is located, and fuses with fragments of other chromosomes, leading to constitutive activation of FGFR1 tyrosine kinase. These cancers are rare and typically have an unfavorable prognosis. Allogeneic hematopoietic stem cell transplant is the most common currently available treatment.