Lirafugratinib (RLY-4008) is a potent, selective oral FGFR2 inhibitor being developed as a second-line treatment option for cholangiocarcinoma with FGFR2 fusion or rearrangement. The program has reached NDA review stage in the U.S. with priority review. It remains non-approved as of the cited update.
The FDA has granted Priority Review for lirafugratinib, an FGFR2 inhibitor for second-line cholangiocarcinoma treatment, with a PDUFA date of September 27, 2026. Clinical trial data showed a 46.5% objective response rate and 11.8-month median duration of response. The therapy demonstrated a manageable safety profile with lower rates of common FGFR inhibitor side effects.
MoonLake Immunotherapeutics says FDA feedback indicates sufficient data to support a U.S. BLA for hidradenitis suppurativa without additional efficacy trials, while Relay Therapeutics received FDA Breakthrough Therapy designation for zovegalisib in combination with fulvestrant for PIK3CA-mutant breast cancer.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07359820 |
A Study of Lirafugratinib in Non-CCA Solid Tumors With FGFR2 Fusion or Rearrangement |
RECRUITING | PHASE2 |
