The first trial of a drug built to lower inherited Lp(a) cut the particle by 78 percent and left event rates unchanged. At least four trials are still running, and none of them repeats the experiment that just failed.
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Its $1.275 billion upfront commitment to a Phase 1 program puts the spotlight on oral peptide developers, including Entera as it approaches Phase 3 in osteoporosis.
The FDA accepted uniQure's BLA for ifezuntirgene inilparvovec with Priority Review and a May 1, 2027 PDUFA date. The accelerated-approval bid rests on an external natural-history control, not a placebo arm.
Inovio's Voceryq is the first plasmid DNA immunotherapy to win FDA approval. It cleared on a 27% complete response in a small open-label cohort, and a confirmatory trial is still ahead.
In depth
Bepirovirsen is the first therapy to offer people with chronic hepatitis B a shot at functional cure after a six-month course. It works in about one in five, and only in a narrow, already-treated population.
Read the storyAHEAD-MERIT missed its progression-free survival endpoint. In this disease, pembrolizumab itself never won PFS either. The trial now turns to overall survival, the endpoint where this cancer actually rewards immunotherapy.
Viatris is paying $1.65 billion for Pacira, roughly 2.2 times trailing revenue. The price only makes sense if you count EXPAREL's early 2030 licensed generic cliff and the four years of runway before it.
With only about 5% of rare conditions carrying an FDA-approved treatment, the agency is trying to turn case-by-case flexibility into written, predictable defaults. Whether that produces certainty or new rigidity is the open question.
A Nature paper finds that one arginine transfer RNA, tRNA1 Arg(UCU), keeps prostate tumors on androgen receptor signaling. When it falls, cells slip into a lineage-plastic, AR-inhibitor-resistant state.
