The FDA approved Elevar Therapeutics’ Lyrfigtu (lirafugratinib) for previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with FGFR2 fusion or other rearrangement, based on the multicenter REFOCUS phase 1/2 trial. The study reported a 46% objective response rate with a median duration of response of 11.8 months. On the rare-disease front, FDA approved Mirum Pharmaceuticals’ zilurgisertib, branded Atebrioz, as the third treatment for ultra-rare soft tissue disease. The ALK2-blocking therapy targets patients with a genetic disorder that drives progressive muscle and connective tissue turning into bone. Together, the approvals reinforce regulators’ continued focus on biomarker-selected oncology and ultra-rare genetic pathways, while highlighting the growing pace of targeted therapeutics reaching patients through single-group and priority-review-style evidence packages.