Incyte Announces Japanese Approval of Pemazyre® (pemigatinib) for the Treatment of Patients with Myeloid/Lymphoid Neoplasms (MLNs)
“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
The 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%).
Previously, 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
MLNs with FGFR1 rearrangement are a form of very rare hematological cancers caused by a chromosomal abnormality (translocation) in which the chromosome breaks where the FGFR1 gene is located (position 11 in the short arm of chromosome 8: location 8p11) and fuses with fragments (genes) of other chromosomes. Various partner genes cause a constitutive activation of the FGFR1 tyrosine kinase, impacting cell proliferation and survival. These cancers are largely divided into two phases according to clinical presentation: the chronic phase, if diagnosed as myeloproliferative disorder or myelodysplastic syndrome, or acute phase, if diagnosed as acute leukemia. The prognosis is unfavorable, and while allogenic hematopoietic stem cell transplant is considered to be the only currently available treatment option that may achieve cure or long-term remission, no standard of care has been established.
FIGHT-203 is a Phase 2, multicenter trial that enrolled patients 18 years and older with myeloid/lymphoid neoplasms (MLNs) with a fibroblast growth factor receptor 1 (FGFR1) rearrangement. Sponsored by
Pemazyre is a kinase inhibitor indicated in
Pemazyre is also the first targeted treatment approved for use in
Pemazyre is a potent, selective, oral inhibitor of FGFR isoforms 1, 2 and 3 which, in preclinical studies, has demonstrated selective pharmacologic activity against cancer cells with FGFR alterations.
Pemazyre is marketed by
Pemazyre is a trademark of
For more information on Incyte Biosciences Japan G.K., please visit Incyte.jp.
Except for the historical information set forth herein, the matters set forth in this press release, including whether Pemazyre might provide a successful treatment option for patients with myeloid/lymphoid neoplasms with FGFR1 rearrangement, , contain predictions, estimates and other forward-looking statements.
These forward-looking statements are based on Incyte’s current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA, MHLW and other regulatory authorities; Incyte’s dependence on its relationships with its collaboration partners; the efficacy or safety of Incyte’s products and the products of Incyte’s collaboration partners; the acceptance of Incyte’s products and the products of Incyte’s collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; greater than expected expenses; expenses relating to litigation or strategic activities; and other risks detailed from time to time in Incyte’s reports filed with the
The drug information contained herein is intended for the disclosure of
1 Overview of Orphan Drug/Medical Device Designation System.
2 Pemazyre (pemigatinib) [Package Insert].
+1 302 498 6171
+1 302 274 4773
+81 3 5561 2915