ARROW-BTC

NCT03037385 📎

Regimen

Experimental
Pralsetinib 400 mg PO QD (continuous) — selective RET inhibitor, Blueprint Medicines/Genentech (Gavreto)
Control
single-arm / no comparator

Population

RET fusion-positive solid tumors (non-lung, non-thyroid, or post-standard therapy) in a pan-cancer ARROW expansion cohort; n=29 (Subbiah V, Nat Med 2022). BTC/CCA: subset of the pan-cancer cohort; RET fusions in BTC <1%. RET fusions detected by central RNA-based NGS or DNA panel. Pralsetinib received FDA tumor-agnostic accelerated approval for RET fusion-positive solid tumors (August 2022).

Key finding

Pralsetinib demonstrated 57% ORR in RET fusion-positive pan-cancer basket with durable responses (12-month DoR rate 81%), supporting FDA tumor-agnostic accelerated approval. Like selpercatinib (LIBRETTO-001), BTC cohort data are too sparse to report separately due to ultra-low RET fusion prevalence in BTC. Both pralsetinib and selpercatinib are clinically regarded as equivalent precision options for RET fusion-positive BTC, though no head-to-head comparison exists. Pneumonitis and hypertension are class-effect toxicities.

Source: PMID 35962206

Timeline

    Guideline citations

    • NCCN BTC (p.4)