Prix Galien

CCA is an aggressive cancer that accounts for 10–20% of all primary hepatic cancers and 3% of all gastrointestinal tumours1 CCAs are diverse biliary epithelial tumors involving the intrahepatic, perihilar and distal parts of the biliary tree1,2 Although rare (0.4–1.8 cases per 100,000 people across Europe), the incidence of CCA, in particular intrahepatic CCA (iCCA), has been generally increasing worldwide over the past few decades1,3 Patients with advanced CCA have a poor prognosis, limited treatment options and symptoms that substantially impact their quality of life1,3,4; The 5-year survival rate for patients with CCA is low, ranging from 5% to 15%5 For patients who experience disease progression after first-line (1L) therapy, second-line (2L) chemotherapy provides only a modest survival benefit6-8: Based on a systematic literature review of 25 studies evaluating the use of 2L chemotherapy for patients with advanced biliary cancer (N=761), median overall survival (OS) was 7.2 months (95% CI, 6.2–8.2) and response rate was 7.7% (95% CI, 4.6–10.9)7,8 FGFR2 fusions are known oncogenic drivers in CCA9,10; FGFR2 fusions are the most common FGFR alteration occurring, almost exclusively, in ~10–16% of iCCA cases11-14. In the Netherlands, approximately 9 patients will have iCCA with an FGFR2 fusion or translocation. PEMAZYRE® is the first approved, molecularly targeted, oral treatment for FGFR2 fusion-positive. CCA PEMAZYRE® monotherapy is indicated for the treatment of adults with locally advanced or metastatic CCA with an FGFR2 fusion or rearrangement that have progressed after at least one prior line of systemic therapy15 The efficacy of PEMAZYRE® was investigated in FIGHT-202, a multicentre, open-label, single-arm study in patients with previously treated, locally advanced/metastatic or surgically unresectable CCA15 • ORR (primary endpoint): 37.0% (95% CI, 27.94–46.86)15 • Median time to response: 2.7 months (range, 0.7–6.9 months)15 • Disease control rate: 82% (95% CI, 74–89)17 • Median overall survival of 17.48 months (95% CI: 14.42, 22.93)20 • Median OS in responders vs non-responders: 30.1 (95% CI, 21.5–not evaluable) vs 13.7 months (95% CI, 9.6–16.1)18 Serious adverse reactions were reported in 46.3% (n=68) of patients. The most common serious adverse reactions were hyponatraemia (2.0% [n=3]) and blood creatinine increase (1.4% [n=2]). No serious adverse reaction led to PEMAZYRE dose reduction. One serious adverse reaction of hyponatraemia (0.7%) led to dose interruption. One serious adverse reaction of blood creatinine increase (0.7%) led to dose discontinuation. 15 Based on these unprecedented clinical results, the high medical need, the innovative character that was also supported in Germany, where PEMAZYRE® (pemigatinib) was awarded21 as one of most innovative therapies in 2021, Incyte is convinced that PEMAZYRE® ( pemigatinib) is a leading candidate for the Galenus Medicines Prize in 2021. • PEMAZYRE®: First Personalized medicine enables patients with FGFR2+ CCA extend median overall survival to 17.48 months20 PHARMACEUTICAL AWARD 2022 PRIX GALIEN NEDERLAND 2022 De referenties en verkorte productinformatie vindt u elders in deze uitgave. 25