Incyte 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 patients with unresectable biliary tract cancer (BTC) with a fibroblast growth factor receptor 2 (FGFR2) fusion gene1, worsening after cancer chemotherapy.
“The MHLW approval of Pemazyre is an important milestone for the BTC community, and underscores our commitment to finding and delivering solutions for patients with significant unmet medical needs,” said Lothar Finke, M.D., Ph.D., General Manager, Incyte Asia. “BTC is a rare and serious condition, and we are proud that with the support of the MHLW we will be able to bring a new targeted treatment to more patients around the world.”
BTC is a rare cancer that forms in the bile duct. Cholangiocarcinoma, a subtype of BTC, is classified based on its origin: intrahepatic, which occurs in the bile duct inside the liver and extrahepatic, which occurs in the bile duct outside the liver. Patients with BTC are often diagnosed at a late or advanced stage when the prognosis is poor2,3. FGFR2 fusions or rearrangements, which occur almost exclusively in intrahepatic cholangiocarcinoma, are observed in a small percentage of Japanese patients with BTC4,5,6,7.
The approval is based on data from the FIGHT-202 study evaluating the safety and efficacy of pemigatinib in adult patients with previously treated, locally advanced or metastatic cholangiocarcinoma with documented FGF/FGFR status. In FIGHT-202 patients harboring FGFR2 fusions or rearrangements (Cohort A), Pemazyre monotherapy resulted in an overall response rate of 36% (primary endpoint), and median DOR of 7.49 months (secondary endpoint). The most frequent treatment-emergent adverse event (TEAE) were grade ≤2 hyperphosphatemia (58.2%). Other frequent TEAEs (all grades) observed in ≥30% of patients were alopecia, diarrhea, fatigue, dysgeusia, nausea, constipation, stomatitis, dry mouth and decreased appetite. The majority of these TEAEs were grade ≤2. The TEAE with grade ≥3 that occurred in ≥10% of patients was hypophosphatemia.
Previously, the MHLW granted Orphan Drug Designation 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 need8. Designated orphan drugs are eligible for priority review for marketing authorizations to ensure supply to clinical settings at the earliest opportunity8.
1 In the approved Japanese indication, “fusion gene” includes both fusion genes and rearrangements.
2 Banales JM, et al. Nat Rev Gastroenterol Hepatol. 2016;13:261‒280.
3 Uhlig J, et al. Ann Surg Oncol. 2019;26:1993–2000.
4 Liver Cancer White Paper 2015. Japan Society of Hepatology.
5 Nakamura H, et al. Nat Genet. 2015;47:1003‒1010.
6 Jang H, et al. J Gastroenterol. 2020;26:6207–6223.
7 Japan Liver Cancer Study Group Follow-Up Survey Committee. The 20th National Primary Liver Cancer Follow-up Report (2008-2009). Liver. 2019;60: 258-293.
8 Overview of Orphan Drug/Medical Device Designation System. Ministry of Health, Labour and Welfare. Available at: https://www.mhlw.go.jp/stf/seisakunitsuite/bunya/0000068484.html.