PEMAZYRE® (pemigatinib) has been provisionally approved by the Therapeutic Goods Administration (TGA) "for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that has progressed after at least one prior line of systemic therapy.

Innovent Biologics...announced that the National Medical Products Administration (NMPA) has approved Pemazyre® (pemigatinib) for the treatment of adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement as confirmed by a validated diagnostic test that have progressed after at least one prior line of systemic therapy.

Innovent Biologics, Inc. (Innovent) (HKEX: 01801)...announced that the Hong Kong Department of Health (DH) has approved Pemazyre® (pemigatinib) for the treatment of adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that have progressed after at least one prior line of systemic therapy.

Innovent Biologics, Inc. (Innovent) (HKEX: 01801), a world-class biopharmaceutical company...announced that the Taiwan market has approved Pemazyre® (pemigatinib) for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement....Among the 107 patients with FGFR 2 fusion/rearrangement, the ORR was 35.5% (95% CI: 27%, 45%), including 3 complete responses.

Pemazyre monotherapy is indicated for the treatment of adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that have progressed after at least one prior line of systemic therapy.

Incyte (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 patients with unresectable biliary tract cancer (BTC) with a fibroblast growth factor receptor 2 (FGFR2) fusion gene, worsening after cancer chemotherapy.

PEMAZYRE is a kinase inhibitor indicated 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.

NICE’s guidance recommends Pemazyre (pemigatinib) for the treatment of CCA with fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that has progressed after at least one prior line of systemic therapy....The NICE decision is based on data from the FIGHT-202 study, which evaluated the FGFR isoform 1,2 and 3 inhibitor in adult patients with previously treated, locally advanced or metastatic CCA with documented FGF/FGFR status.

Biliary Tract Cancer: Useful in Certain Circumstances…For cholangiocarcinoma with FGFR2 fusions or rearrangements: Pemigatinib.

...- Documented FGFR2 rearrangement....

...ORR in Participants FGF/FGFR Alterations Other Than FGFR2 Rearrangements or Fusions`ORR in All Participants With FGF/FGFR Alterations`ORR in Participants Negative for FGF/FGFR Alterations`Progression-free Survival (PFS)`Duration of Response (DOR)`Disease Control Rate (DCR)`Overall Survival`Number of Participants With Any Treatment-emergent Adverse Event (TEAE)`First-order Absorption Rate Constant (ka) of Pemigatinib`CL/F of Pemigatinib`Vc/F of Pemigatinib`Vp/F of Pemigatinib...

...Documentation of FGFR2 rearrangement....

...Documented FGFR2 rearrangement.`...

The objective response rate was 45.3% (3 complete responses and 21 partial responses, n = 24). The median progression-free survival was 9 months (IIQ 6-14) and the median overall survival was 18 months (IIQ 12-NA)….Our study confirms the efficacy of pemigatinib in an unselected population with high response rates and survivals similar to the FIGHT-202 data. It also confirms the good tolerability of pemigatinib and the better prognosis of iCCA with FGFR2 f/r.

In this ongoing, multicenter, single-arm, phase II study, adult patients with locally advanced or metastatic cholangiocarcinoma carrying centrally confirmed FGFR2 fusions or rearrangements who had progressed on ≥1 systemic therapy received 13.5 mg oral pemigatinib once daily (3-week cycle; 2 weeks on, 1 week off) until disease progression...Among 30 patients with FGFR2 fusions or rearrangements evaluated for efficacy, 15 patients achieved partial response (ORR, 50.0%; 95% confidence interval [CI], 31.3-68.7); 15 achieved stable disease, contributing to a disease control rate of 100% (95% CI, 88.4-100).

Median (range) duration of follow-up was 45.4 (19.9–53.7) months. In total, 98.6% of patients discontinued treatment, most commonly for disease progression (n=105/147; 71.4%). In cohort A, ORR (95% CI) was 37.0% (27.9%–46.9%), DCR (95% CI) was 82.4% (73.9%–89.1%), and median DOR (95% CI) was 9.1 (6.0–14.5) months. For cohorts B and C, DCR was 40.0% (19.1%–63.9%) and 17.6% (3.8%–43.4%), respectively. Median (95% CI) PFS was 7.0 (6.1–10.5) months for cohort A, and 2.1 (1.2–4.9) and 1.5 (1.4–1.8) months for cohorts B and C, respectively. For cohorts A–C, median (95% CI) OS was 17.5 (14.4–22.9), 6.7 (2.1–10.6), and 4.0 (2.0–4.6) months...: In this analysis, pemigatinib continued to demonstrate durable response, prolonged OS, and a manageable safety profile in patients with advanced/metastatic CCA with FGFR2 fusions or rearrangements. These results further highlight the need for molecular testing in patients with CCA.

Three FGFR2 f/r patients received an FGFR inhibitor (pemigatinib) as next treatment after testing in 2020. A trend towards longer median overall survival in the f/r vs wt group (24.5 vs 17.8 mo)…

The other 31 subjects with documented FGFR2 fusion or rearrangement were enrolled in Part 2 and received 13.5 mg pemigatinib....Pemigatinib was high response rate and manageable tolerability in Chinese patients with recurrent or metastatic cholangiocarcinoma with FGFR2 fusion or rearrangement.

Patients with locally advanced or metastatic CCA with FGFR2 fusions/rearrangements (n = 107)...documented disease progression after at least one systemic cancer therapy before enrollment in FIGHT-202 were assessed….The median PFS was 7.0 months (4.9 to 11.1) for patients with FGFR2 fusions/rearrangements (n = 65) with second-line pemigatinib received during the FIGHT-202 trial.

...patients with CCA harboring FGFR2 fusions or rearrangements (FGFR2+)...For patients who had progressed after 2 lines of prior therapies and then received pemigatinib third-line during FIGHT-202, median PFS was 8.9 (95% CI: 4.9, 13.1) months (n=30)...Median PFS on second- or third-line pemigatinib for FGFR2+ CCA was longer than second- or third-line systemic therapy received prior to FIGHT-202...

Pts (≥18 y) with known FGF/FGFR alterations and progression after ≥1 prior therapy had FGFR2 rearrangements/fusions (cohort A)... Independent, centrally confirmed ORR was 37.0%; mOS was 17.5 mo….These results reinforce the primary data, showing continued, durable responses and sustained tolerability in pts receiving PEMI for CCA harboring FGFR2 rearrangements/fusions.

Incyte (Nasdaq:INCY) today announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending the conditional marketing authorization of pemigatinib for the treatment of adults with unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement...The CHMP opinion 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.

FGF/FGFR genomic alterations (GAs)... FGFR2 fusions/rearrangement (RE)..Pts with advanced disease…assigned to cohort A (FGFR2 RE), B (other FGF/FGFR GA), or C (no FGF/FGFR GA). Pts received pemigatinib 13.5 mg QD.... In cohort A, ORR (95% confidence interval) was 40.6% (23.7-59.4) with 1 complete response (Figure); mDOR was 7.5 mo (5.5-7.5), DCR was 87.5% (71.0-96.5), mPFS was 6.9 mo (4.8-9.1), and mOS was 14.7 mo (11.7-not reached).

38 (35·5% [95% CI 26·5-45·4]) patients with FGFR2 fusions or rearrangements achieved an objective response (three complete responses and 35 partial responses)….These data support the therapeutic potential of pemigatinib in previously treated patients with cholangiocarcinoma who have FGFR2 fusions or rearrangements.

ORR in cohort A was 35.5% (95% CI, 26.5%–45.4%), with 3 complete responses; median (m) DOR was 7.5 (95% CI, 5.7–14.5) months (mo), DCR was 82% (95% CI, 74%–89%), mPFS and mOS were 6.9 (95% CI, 6.2–9.6) and 21.1 (14.8–not reached) mo (OS not mature at cutoff)....These data support pemigatinib as a potential treatment option for previously treated pts with CCA harboring FGFR2 gene rearrangements/fusions.

In this analysis, pemigatinib continued to demonstrate durable response, prolonged survival, and a manageable safety profile in patients with advanced/metastatic CCA with FGFR2 fusions or rearrangements.