For how we live.
The Fit-for-Purpose Rare Disease Framework Initiative launches with a 28 October public meeting and a request for comments. The FDA wants to tailor its review to very small patient populations.
In the 17,701-person E.mbrace trial in adults 60 and older, the nine-valent ExPEC9V vaccine showed 14.63% efficacy, with a confidence interval spanning zero. It stopped early for futility; development is discontinued.
A final question-and-answer guidance tells biosimilar makers what data to file when they change how an approved product is made, fulfilling a 2022 user-fee commitment. It was issued on 13 October 2026.
The agency is describing the trial design it is weighing for ibogaine, a psychedelic with serious cardiac risks, as HHS funds the first federal studies in opioid use disorder and PTSD. Comments close 20 November.
A hybrid public meeting on 26 October will discuss proposed BsUFA IV recommendations. The fees that fund FDA review of biosimilar applications expire in September 2027 and need new legislation to continue.
ZUPREME 1 tested a once-weekly amylin analogue that works differently from GLP-1 drugs, finding up to 9.4% weight loss at 28 weeks and gastrointestinal side-effects close to placebo. Zealand Pharma funded it.
In the industry-funded trial, young responders who kept taking the dopamine D1 blocker relapsed less than those tapered to placebo. The effect was clear in children but not in the small adult group.
In the phase 2 Vibrance-1 trial, alixorexton lifted mean sleep latency from about 2 minutes on placebo to 24 or more on the drug. The six-week study was funded by the manufacturer.
In a single-arm trial, children with vanishing white matter given guanabenz kept walking longer than matched historical controls. The design was uncontrolled and hallucinations were common early on.
Varespladib, given on top of antivenom in a randomised trial in India and the USA, did not beat placebo. But it reached patients seven hours after the bite, and the authors say that timing may be the story.
Open-label follow-up of the phase 3 LUNA3 trial shows rilzabrutinib's platelet responses were durable and sometimes appeared in people who had not responded at first — but a minority reached the mark.
Amycretin, a single molecule that activates both the GLP-1 and amylin receptors, produced up to 24.3% weight loss over 36 weeks in a 125-person study. The primary goal, though, was safety — not weight.
In HIMALAYA, 305 people with ALS took the RIPK1 inhibitor SAR443820 or placebo; function fell almost identically in both, and the sponsor is halting the drug in ALS.
In a 711-patient phase 2b trial, the myeloperoxidase inhibitor mitiperstat did not improve symptoms or walking distance in heart failure with preserved or mildly reduced ejection fraction.
The patented method uses mifepristone for hypercortisolism-related insulin resistance, with dosing capped to avoid over-activating the stress-hormone axis. The public can object before the deal closes.
A draft replacing a 2003 document would extend hepatic-impairment dosing studies to biologic drugs and to drug effects, not just blood levels. It is open for comment before the agency finalises it.
Lorecivivint improved pain modestly and changed nothing structural. Sprifermin grew cartilage and the effect made no difference by year five. Both illustrate the gap between a biomarker and a benefit.
Two 2026 reviews make the case that human-derived models can replace animal testing. A third paper argues the field is checking the biology and the algorithm separately, and calling that assurance.
In a 122-patient South African proof-of-concept study, a four-month all-oral combination of quabodepistat, delamanid and bedaquiline cleared TB from sputum as often as the six-month standard.
Buntanetap showed dose-dependent cognitive benefit in biomarker-positive patients with mild disease. The company's announcement does not say what happened in the full 351-patient population.
Two doses of the long-acting antibody L9LS protected under-fives in a high-transmission area, but researchers say a higher dose is likely needed for stronger cover.
The agency says it has been relaxing chemistry and manufacturing expectations for cell and gene therapies case by case. The change is that every sponsor now knows it.