Section
Trials, approvals, and the drugs behind them.
FDA cleared lirafugratinib (Lyrfigtu) for FGFR2-altered cholangiocarcinoma after ReFocus posted a 46% response rate. The clinical argument is FGFR1 sparing. The cost is months of mandatory retinal monitoring.
Barzolvolimab cleared hives in about half of Phase 3 CSU patients by week 24, including many Xolair failures, but two anaphylaxis cases sent Celldex shares down 13% ahead of a 2027 BLA.
Iambic Therapeutics filed for a Nasdaq IPO the same morning it announced an AbbVie collaboration. Public markets will still judge the company on whether its brain-penetrant HER2 inhibitor holds up against Enhertu and Tukysa.
IntraBio’s Aqneursa cleared the FDA as the first treatment for ataxia-telangiectasia on a -1.88 SARA point crossover win, treating downstream neurodegeneration rather than an intractable 305 kDa kinase defect.
FDA approved Lilly's Inluriyo plus Verzenio for ESR1-mutated ER+/HER2- metastatic breast cancer after endocrine progression, doubling median PFS to 11.1 months in EMBER-3. The clinical gain comes with 86% diarrhea and neutropenia rates, challenging early liquid biopsy switching models.
Ultragenyx's AAV9 gene therapy is the first treatment for Sanfilippo syndrome Type A. Full approval on 17 treated children against a natural history control now collides with a diagnostic window most families still miss.
Curium’s Bexlutry is the first 505(b)(2) radioligand equivalent. The regulatory shortcut is done. Hospital clocks, isotope supply, and a pending $8 billion merger now decide whether Novartis’s NET franchise actually erodes.
Two-year ORIGIN 3 data show weekly atacicept flattened eGFR decline to 0.6 mL/min per year and cut kidney-progression events by 76 percent, with no dialysis or transplants on drug.
Definium's Phase 3 Panorama study met its primary endpoint in generalized anxiety disorder with a 5.1-point HAM-A gap, testing whether an active 50 µg control and zero psychotherapy can clear the FDA hurdle that broke Lykos.
The FDA has granted accelerated approval to Bayer's Hyrnuo as a frontline therapy in HER2-mutant lung cancer, matching rival zongertinib's response rates while carrying twice-daily meal requirements and steeper gastrointestinal toxicity.
FDA approval of Isembyld gives spinal muscular atrophy its first muscle-targeted therapy, moving motor function in patients plateaued on Spinraza or Evrysdi while introducing a 9% fracture warning and a double-biologic payer burden.
Solstice Oncology launched with a $225 million Series A led by RA Capital to advance Harbour BioMed-licensed porustobart into neoadjuvant MSS colon cancer.