v3 · current
C. Laurent (Regulatory Affairs Lead) · 08 Apr 2024 · a8770da6cf07…
ATC code confirmed (removed "provisional"); added OS not-yet-reached statement from updated data cut
Pharmacotherapeutic group: Antineoplastic agents, protein kinase inhibitors, ATC code: L01EN05. Mechanism of action Veraxanib is a selective, potent, reversible inhibitor of fibroblast growth factor receptor 2 (FGFR2) kinase. It inhibits FGFR2 autophosphorylation and downstream signalling through the MAPK and PI3K/AKT pathways in tumour cells harbouring FGFR2 gene fusions or activating mutations, leading to inhibition of cell proliferation and induction of apoptosis. Clinical efficacy Efficacy was established in study VRXB-101, an open-label, single-arm trial in 112 patients with advanced FGFR2-altered solid tumours who had received at least one prior line of systemic therapy. The primary endpoint was overall response rate (ORR) by independent radiological review. The ORR was 38% (95% CI: 29–47%), with a median duration of response of 9.3 months (95% CI: 7.2–12.1 months). Median progression-free survival was 7.1 months (95% CI: 5.8–8.6 months). Median overall survival was not yet reached at the time of analysis.
v2
Dr. A. Müller (MAH Medical Affairs) · 12 Sept 2023 · a361acca661b…
Added apoptosis mechanism; filled in trial enrolment n=112 and efficacy endpoints from final CSR
Pharmacotherapeutic group: Antineoplastic agents, protein kinase inhibitors, ATC code: L01EN05 (provisional). Mechanism of action Veraxanib is a selective, potent, reversible inhibitor of fibroblast growth factor receptor 2 (FGFR2) kinase. It inhibits FGFR2 autophosphorylation and downstream signalling through the MAPK and PI3K/AKT pathways in tumour cells harbouring FGFR2 gene fusions or activating mutations, leading to inhibition of cell proliferation and induction of apoptosis. Clinical efficacy Efficacy was established in study VRXB-101, an open-label, single-arm trial in 112 patients with advanced FGFR2-altered solid tumours who had received at least one prior line of systemic therapy. The primary endpoint was overall response rate (ORR) by independent radiological review. The ORR was 38% (95% CI: 29–47%), with a median duration of response of 9.3 months (95% CI: 7.2–12.1 months). Median progression-free survival was 7.1 months (95% CI: 5.8–8.6 months).
v1
Dr. A. Müller (MAH Medical Affairs) · 15 Mar 2023 · 08179008ac27…
Initial submission
Pharmacotherapeutic group: Antineoplastic agents, protein kinase inhibitors, ATC code: L01EN05 (provisional). Mechanism of action Veraxanib is a selective, potent, reversible inhibitor of fibroblast growth factor receptor 2 (FGFR2) kinase. It inhibits FGFR2 autophosphorylation and downstream signalling through the MAPK and PI3K/AKT pathways in tumour cells harbouring FGFR2 gene fusions or activating mutations. Clinical efficacy Efficacy was established in study VRXB-101, an open-label, single-arm trial in patients with advanced FGFR2-altered solid tumours. The primary endpoint was overall response rate (ORR) by independent radiological review.