Beare–Stevenson cutis gyrata syndrome

Basilea reports pooled efficacy data for derazantinib in iCCA patients with FGFR2 gene mutations and amplifications presented at ESMO MAP Virtual Congress 2020

Retrieved on: 
Monday, October 12, 2020
Clusters of differentiation, Tyrosine kinase receptors, Fibroblast growth factor receptor 2, Organ systems, Syndromes, Clinical medicine, Fibroblast growth factor receptor 1, Beare–Stevenson cutis gyrata syndrome

FGFR inhibitors, including derazantinib, have demonstrated clinical antitumor activity in patients with FGFR2 gene fusion-positive iCCA.

Key Points: 
  • FGFR inhibitors, including derazantinib, have demonstrated clinical antitumor activity in patients with FGFR2 gene fusion-positive iCCA.
  • However, to date there is limited clinical evidence for the benefit of FGFR inhibitors in iCCA patients with FGFR2 gene mutations and amplifications.
  • The data presented at the MAP congress show that derazantinib is active in this group of patients and underscore the broad therapeutic potential of derazantinib in FGFR2-positive iCCA.
  • The following e-poster was presented at the ESMO MAP Virtual Congress 2020:
    Efficacy of derazantinib in intrahepatic cholangiocarcinoma patients with FGFR2 mutations or amplifications: Pooled analysis of clinical trials and early access programs.

Basilea reports at ESMO meeting that drug candidate derazantinib showed clinical benefit in intrahepatic cholangiocarcinoma (iCCA) patients with various FGFR2 genetic aberrations

Retrieved on: 
Monday, September 30, 2019
Tyrosine kinase receptors, Fibroblast growth factor receptor 2, Organ systems, Syndromes, Medical specialties, Beare–Stevenson cutis gyrata syndrome

The analysis shows that derazantinib provided clinically meaningful anti-tumor activity, not only in iCCA patients with FGFR2 gene fusions, but also in iCCA patients with FGFR2 gene mutations and amplifications.

Key Points: 
  • The analysis shows that derazantinib provided clinically meaningful anti-tumor activity, not only in iCCA patients with FGFR2 gene fusions, but also in iCCA patients with FGFR2 gene mutations and amplifications.
  • Activity of derazantinib in patients with FGFR2 gene mutations and amplifications could be an important differentiation factor in this indication and would address an important unmet medical need."
  • DCRs were 67% for patients with FGFR2 gene mutations or amplifications compared to 83% for patients with FGFR2 gene fusions.
  • ICCA patients without any FGFR2 genetic aberration did not respond to treatment with derazantinib, which is consistent with previous reports.