For how we live.
I-SPY2.2 tested trastuzumab-deruxtecan plus rilvegostomig before surgery in high-risk HER2-negative breast cancer. In immune-positive tumours 54% had a complete response, and most of them avoided anthracyclines.
A final FDA guidance treats the on-screen output of software sent with a prescription drug as a type of drug labelling, and says how the prescribing information should describe it and when sponsors submit it for review.
In a 98-patient French trial, six-monthly rituximab did not extend remission beyond daily azathioprine in this rare eosinophilic vasculitis — and recipients had more severe infections.
In a 17-person phase 2a trial, once-weekly inhaled SPL84 lifted lung function in up to 70% of treated patients with the 3849+10 kb C→T mutation — a first reported clinical signal for an RNA-targeted inhaled therapy.
LOTIS-9 tested loncastuximab tesirine plus rituximab in unfit and frail older adults with untreated DLBCL. Complete responses reached 53% and 42%, but severe side effects, including 11 deaths, ended the trial early.
The agency authorised emergency use of four veterinary products to prevent or treat New World screwworm infestations, acting on a 2025 declaration that the parasite poses a potential public-health emergency.
A 60-woman phase 2A trial found the dual PPAR agonist reduced liver fat by nearly 15 percentage points more than placebo over 24 weeks — an early, small signal in a group usually left out of fatty-liver drug trials.
A 9 October labelling supplement folds results from two post-marketing rheumatoid-arthritis safety studies into the JAK inhibitor baricitinib's boxed warning, and revises its infection and cardiovascular sections.
A detailed safety analysis of the 12,254-patient OCEANIC-STROKE trial found no significant rise in major bleeding with the factor XI blocker, though lower gastrointestinal bleeding was more frequent.
In the phase 3 VERIFY trial, 77% of patients on the hepcidin-mimic drug avoided phlebotomy over weeks 20–32 versus 33% on placebo, and far more kept their haematocrit under control.
Among 7,579 pregnancies in people with migraine, anti-CGRP antibody use early in pregnancy carried a 42% higher adjusted risk of spontaneous abortion; propranolol showed no increase. The data are observational.
The phase 2 ROXAMI trial gave the oral HIF stabiliser roxadustat to 158 STEMI patients after stenting. Infarct size at 30 days was no smaller than with standard care. Later remodelling findings were exploratory.
RINGSIDE phase 2 tested three doses of oral varegacestat in 42 adults with progressing desmoid tumours. The lowest daily dose gave the deepest shrinkage and was carried into phase 3. No comparator arm.
In a 28-patient single-arm phase 2 study, disitamab vedotin with tislelizumab produced a complete response in 73% of evaluable HER2-positive bladder cancers that could not be resected. There was no comparator.
In a 134-patient phase 3 crossover trial, deucrictibant — the first oral bradykinin B2 receptor blocker — brought symptom relief in a median 1.28 hours, against more than 12 hours on placebo.
ESO-T01 builds cancer-killing T cells inside the body, skipping the cell harvest and chemo prep normal CAR-T needs. All four responded — but the cells faded, and median progression-free survival was 4 months.
In a 348-patient trial, patients in sustained low disease activity who stepped down their biologic kept it almost as often as those who stayed on full dose over 24 weeks — but some measures still favoured continuing.
A single-arm phase 2 trial added glofitamab to pola-R-CHP in 41 people with newly diagnosed B-cell lymphoma. The best complete-response rate was 95%, cytokine release stayed grade 1, and no neurotoxicity was reported.
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.
In the MONEAD cohort, six-year-olds whose mothers breastfed while taking antiseizure medicines scored no worse on language or adaptive behaviour — and longer breastfeeding tracked with slightly better scores.
In the industry-funded evERA trial, giredestrant plus everolimus delayed progression of ER-positive breast cancer — but the clearest benefit was in ESR1-mutated tumours, and the endpoint was a surrogate, not survival.
AbbVie's once-daily pill cleared the FDA on 25 September as a new molecular entity. Across three phase 3 TEMPO trials it beat placebo in early Parkinson's and as an add-on to levodopa.
Two phase 3 trials put the oral drug safiglipron ahead of placebo and non-inferior to dapagliflozin on HbA1c. Both ran only in China, were maker-funded, and showed dose-dependent gut side effects.
In the phase 3 UKALL14 trial, one cycle of the T-cell drug nelarabine after induction was well tolerated but left event-free survival essentially unchanged in adults aged 25 to 65.
A randomised Asian trial in 130 patients found no efficacy gap between the two drug regimens for active lupus nephritis, but serious infections and all three deaths fell in the mycophenolate group.
The PLUSS trial put a steroid directly into the lungs of 1,059 infants born before 28 weeks. Survival free of chronic lung disease barely differed from surfactant alone.
A single-centre, placebo-controlled trial found targeted onabotulinumtoxinA injections cut dystonia-related foot pain without harming movement. The effect was large, but the trial randomised only 33 people.
Final results of the phase 3 IMpassion050 trial show adding the immunotherapy atezolizumab to standard HER2-targeted treatment did not improve event-free or disease-free survival.
The phase 3 CLL6 RESIDUUM trial extended progression-free survival by about 22 months in patients left with residual disease, but overall survival did not differ and the drug caused more infections.
In the Scandinavian phase 3 ASAC trial, daily aspirin failed to improve disease-free survival after treatment of colorectal cancer liver metastases — and overall survival was worse on the drug.
ACHIEVE-4 found the non-peptide GLP-1 pill non-inferior to insulin glargine for major cardiac events over two years, with less severe hypoglycaemia but far more gastrointestinal upset. Lilly funded it.
Ketamine cuts suicidal thinking fast but briefly. A 50-person trial gave low-dose buprenorphine afterward and saw the effect hold — an early, single-centre result in a high-risk group.
A population-linked Australian cohort followed children to school entry after antenatal metformin. The adjusted relative risk of developmental vulnerability was 0.97 — no different from unexposed children.
In a 366-child phase 2b trial, the live attenuated nasal vaccine BPZE1 raised the mucosal antibody current shots miss, and did not blunt a co-given Tdap. The maker funded the study.
The FDA approved PTC Therapeutics' sepiapterin in July 2025. In the phase 3 APHENITY trial, responders saw a 63% fall in blood phenylalanine over six weeks against roughly no change on placebo.
In the phase 2 KRAKEN trial, muvalaplin lowered lipoprotein(a) by 47.6% to 85.8% over 12 weeks on an intact-particle assay. Eli Lilly funded it, and the trial was not built to measure heart attacks or strokes.
In the GSK-funded phase 3 MATINEE trial, the anti-IL-5 antibody lowered moderate or severe exacerbations from 1.01 to 0.80 a year — but symptom and quality-of-life endpoints showed no significant benefit.
In a phase 2 trial in people whose hidradenitis suppurativa had already failed anti-TNF drugs, two lutikizumab doses beat placebo at 16 weeks. A third did not, and the placebo response was high.
In the phase 3 LIBerate-HR trial, lerodalcibep lowered LDL cholesterol 56.2% more than placebo at 52 weeks on top of statins. It won approval in 2026; no outcome trial has yet reported.
The oral histone deacetylase inhibitor cleared the FDA in March 2024. In the phase 3 EPIDYS trial, boys on givinostat still lost function over 72 weeks — just less of it than those on placebo.
AMPLIFIED enriched for early rheumatoid arthritis patients thought to respond best to abatacept, then compared it head-to-head with adalimumab. At week 24 the two drugs were indistinguishable.
One lot of Vitruvias's 1/2-grain Thyroid Tablets — 1,955 bottles sold nationwide — was pulled after testing showed the tablets carry more hormone than the label states. The FDA calls it its most serious recall class.
Two 2026 Class I recalls cover Omnipod 5 insulin pods whose soft cannula can be damaged in manufacturing, letting insulin leak instead of reaching the user. The FDA ties under-delivery to a risk of DKA.
The publicly funded E4512 trial found two years of crizotinib after surgery did not prolong disease-free survival against observation, with a hazard ratio of 1.08 and no safety upside.
In the investigator-led Danish DALIAH trial, pegylated interferon-alfa and hydroxyurea produced similar molecular responses by intention-to-treat, but far more patients stopped interferon.
The FDA logged a Class I recall of 35,883 boxes of the simethicone antigas product on 17 June, after softgels were contaminated with a propylene glycol-based coolant that leaked from packaging machinery.
Approved on 24 June under NDA 220436, the Avyxa Holdings product is a ready-to-infuse form of the NK-1 blocker fosaprepitant, cleared on studies of an earlier intravenous version of the drug.
A Spanish registry followed 166 patients with treatment-resistant epileptic encephalopathies for a year. Median seizure frequency fell 68.8%, though the design cannot match a placebo-controlled trial.
The FDA logged the recall of 145,464 bags of 0.9% sodium chloride in 500 mL Viaflex containers on 9 September, after fiberglass particulate matter was found. It is the agency's most serious recall tier.
In the phase 3 Bax24 trial, the aldosterone-synthase inhibitor cut 24-hour ambulatory systolic pressure 14.0 mm Hg more than placebo over 12 weeks. AstraZeneca funded it, and three patients developed high potassium.
Gilead's phase 3 trial in 539 patients was terminated after the CD47 antibody failed to improve remission or survival and caused more fatal adverse events than azacitidine alone.
There is no approved drug for methamphetamine use disorder. In 339 patients treated in ordinary clinics, a generic antidepressant cut use by about two days a month more than placebo, with more dropouts from side-effects.
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.
The Class I recall covers 625,500 prefilled syringes. Cartons labelled morphine sulfate 2 mg/mL hold a Dilaudid (hydromorphone) syringe of 0.5 mg/0.5 mL instead — the wrong opioid at the wrong dose.
The FDA classified the nationwide recall Class I, its most serious tier. About 31,935 multi-dose vials are affected, with particulate identified as nylon, glass and other materials, plus broken and leaking units.
Two-year blinded data and open-label follow-up from the ELATIVE trial show the drug kept improving liver blood tests in primary biliary cholangitis — but the endpoint is still a surrogate, and the trial is industry-run.
In the company-run CONDOR trial, brolucizumab injections outperformed panretinal laser for proliferative diabetic retinopathy over 54 weeks — while intraocular inflammation was far more common in the drug arm.
The TRACK trial was stopped early for futility. The blood thinner failed to reduce a composite of cardiovascular events, while major bleeding rose by half. Bayer, which makes the drug, was a listed collaborator.
In the phase 3 TenCRAOS trial, intravenous tenecteplase given within 4.5 hours did no better than aspirin at recovering sight, and carried more bleeding — including one fatal brain haemorrhage.
The NIH-funded STArT trial in children and young adults was halted for futility: time to crisis resolution was 60.8 hours with arginine versus 65.8 with placebo, well within the range of chance.
A double-blind Danish trial gave the drug or placebo during resuscitation at 21 hospitals. Restored circulation occurred in 39% versus 37% — no meaningful difference — and bicarbonate raised blood alkalosis and sodium.
In the industry-funded ADVANCE OUTCOMES trial, the once-daily prostacyclin-pathway drug more than halved the risk of a first worsening event, but drove far more treatment discontinuations for side-effects.
Ceperognastat, an oral OGA inhibitor, showed no benefit on the primary cognitive endpoint at 100 weeks. The higher dose progressed 32% faster than placebo and had more serious adverse events.
VICTORY randomised 238 adults with severe burns to intravenous vitamin C or placebo. It was stopped early: 28-day mortality was 15.0% versus 7.6%, and the trial crossed a prespecified harm threshold.
REPLENISH randomised 381 patients with relapsed polymyalgia rheumatica. Sustained remission at 52 weeks reached about 41% on secukinumab versus 20.4% on placebo. Novartis funded the trial.
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 the SOLARIO trial, patients who got local antibiotics at surgery did no worse on a short systemic course than a long one, and reported far fewer side effects. The comparison was independently funded.
In midlife women the usual cause is the menopause transition, where hot flushes and night sweats last a median of seven years. In older adults they are common, mostly benign, and rarely a mortality signal.
For most adults who bruise more than they used to, the cause is thinner skin from ageing, sun or steroids, plus the aspirin and anticoagulants many older people take. A few patterns point elsewhere.
TEMPLE, an AbbVie-funded phase 3b trial, is the first direct test of the CGRP blocker atogepant against topiramate — a long-used preventive many patients cannot tolerate.
SINGLE-AF randomised 1,803 South Koreans with borderline stroke risk to a direct oral anticoagulant or nothing. The drug lowered a composite of stroke, bleeding and death at two years, from 1.5% to 0.5%.
In PHOENYCS GO, dapirolizumab pegol lifted the share of lupus patients reaching a composite response at 48 weeks to 50% from 35% on placebo — a real but modest 14.6-point gain.
A direct final rule swaps 'animal' for 'nonclinical' across five parts of FDA's drug regulations. It adds no requirements, but signals a shift the agency began with monoclonal antibodies.
An analysis of 50 top-selling 'T booster' products found only 24.8% contained an ingredient with data showing it raises testosterone — and 10.1% contained something shown to lower it. Two herbs are partial exceptions.
Short courses look well tolerated, but trials rarely run past a few weeks, so long-term safety is untested. Two clearer problems: variable supplement content, and a surge in children's accidental ingestions.
A Cochrane review found no evidence that antibiotics ease the pain of an inflamed tooth nerve. The best trial evidence points to ibuprofen, with paracetamol added — but only a dentist fixes the cause.
The 2024 American Academy of Sleep Medicine guideline strongly backs intravenous iron and gabapentinoids, and now suggests against the dopamine agonists that were first-line for two decades.
Gout affects about one in twenty American men, driven by high uric acid. Guidelines and randomised trials back long-term urate-lowering to a target below 6 mg/dL, yet only a third of patients receive it.
The TRAVERSE trial cleared its cardiovascular safety in 5,246 high-risk men; the Testosterone Trials found a real but moderate sexual-function benefit. Guidelines reserve it for genuine, twice-confirmed deficiency.
In the TESEO trial, benznidazole for 30 days matched the 60-day standard for clearing the parasite and caused fewer side effects, pointing to a gentler regimen for a neglected disease.
Regulators say benzocaine and lidocaine gels, homeopathic tablets and amber necklaces carry real risks — a rare blood disorder, seizures, choking deaths — for little proven benefit in teething.
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.
In people with no heart disease, aspirin's benefit was small or absent and bleeding rose across three large trials. US guidance now advises against starting it at 60 or older.
A French study of 11,558 patients without heart failure linked atenolol to fewer cardiovascular events than bisoprolol. It is observational, so it shows an association, not proof one is better.
A network meta-analysis of 118 trials and 31,195 men found every oral PDE5 inhibitor beat placebo, with tadalafil ranked most effective. Guidelines make the tablets, after a health check, the first-line choice.
The trials that licensed minoxidil ran for 24 to 48 weeks, and a five-year follow-up shows why the honest answer to 'when can I stop' is: you probably can't without losing the gain.
At one year, 79% of newly diagnosed patients given early infliximab plus an immunomodulator were in steroid-free, surgery-free remission, versus 15% treated conventionally.
An independent 2026 meta-analysis of AAV micro-dystrophin trials found small, mostly uncontrolled functional benefits — largest in the youngest boys — set against serious harms including rare deaths.
Pooled trials put the herb roughly on a par with standard antidepressants for mild-to-moderate depression, with fewer dropouts for side effects. The catches are dangerous drug interactions and wildly variable products.
A meta-analysis of four small trials found zilebesiran lowered office systolic pressure by 7.16 mmHg versus placebo, with wide confidence intervals, a hint the effect wanes by six months, and more hyperkalaemia.
The first randomised head-to-head trial of the two obesity drugs gave 751 adults the maximum tolerated dose of each for 72 weeks. Tirzepatide cut weight by 20.2%, semaglutide by 13.7%.
Two large trials, ETHOS and IMPACT, found adding an inhaled steroid to a two-bronchodilator inhaler lowered exacerbation rates in COPD — at the cost of more pneumonia.
As RTS,S reached more than 2 million children in Ghana, Kenya and Malawi, a WHO-coordinated pilot trained about 6,000 health workers to detect and report side-effects that most rollouts assume can already be caught.
A double-blind trial gave 17,598 people pantoprazole or placebo for three years and tracked pneumonia, fractures, kidney disease and dementia. Only gut infections rose, and only modestly.
In FINEARTS-HF, 6,001 patients with an ejection fraction of 40% or more had fewer worsening-heart-failure events on finerenone. Cardiovascular deaths were not lower.
Cold sores heal on their own in about a week, and nothing clears the virus for good. Creams and pills shave hours to roughly a day off an episode — but only if started at the first tingle.
Nocturia is usually blamed on the prostate, but the commonest driver is the kidneys making too much urine overnight. In one trial, low-dose desmopressin trimmed nightly voids and bought about 40 extra minutes of sleep.
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.
Older meta-analyses linked lithium to fewer suicides. A randomised trial in 519 US veterans, stopped early for futility, found no such benefit when it was added to usual care.
In a 142-patient phase 3 trial, 37% reached complete remission on the antibody obinutuzumab versus 6% on tacrolimus at two years. A key kidney-function endpoint was not met, tempering the result.
In ESSENCE, the first 800 patients with biopsy-confirmed fatty-liver disease were analysed at week 72. Steatohepatitis resolved in 62.9% on semaglutide versus 34.3% on placebo — an interim look at an ongoing trial.
Two trials found low-dose colchicine cut cardiovascular events, and the FDA approved it for heart risk in 2023. Then the largest trial found nothing, leaving the question genuinely unsettled.
The 2024 AASM guideline now advises against routinely using the dopamine agonists that were once standard, because over time they often make the condition worse — and puts anticonvulsants and iron first.
Cobenfy, approved in the US in September 2024, cut symptom scores more than placebo in two five-week trials — without the weight gain and movement effects of older drugs, but with cholinergic side effects.
A US trial found ketamine at least as good as electroconvulsive therapy; a Swedish trial found ECT clearly better. The gap lies in whom they treated and how they scored success.
A matched cohort of 173,910 people with dementia in England tied the drugs to raised risks of pneumonia, stroke, clots, heart attack, fracture and kidney injury — highest just after starting.
In the phase 3 THRIVE trial, veligrotug shrank bulging eyes in 70% of patients versus 5% on placebo. It is the first serious challenger to the only drug approved for the condition.
Swollen ankles are common with age. Chronic venous disease rises steeply over the years, and a very ordinary culprit is medication: calcium channel blockers caused ankle swelling in 10.7% of users versus 3.2% on placebo.
Sold as natural, safer and tailored by hormone testing, custom-compounded menopausal hormones are untested against approved options — and expert panels advise restricting their use.
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.
A daily testosterone-and-progestin gel is in a 52-week efficacy trial in 462 couples. A non-hormonal pill cleared its first safety study in 16 men. Neither is approved.
A single-centre randomised trial found that three cycles of intravenous immunoglobulin on top of high-dose steroids improved muscle disease faster and further — in just 44 patients, at one Dutch hospital.
One drug, dapoxetine, is purpose-approved for it, and not in the US. An umbrella review of 65 trials found the SSRIs and anaesthetics that lengthen latency rest on weak evidence.
A 4,153-woman trial found progesterone did not raise live births overall. The benefit concentrated in women who were bleeding and had had three or more previous miscarriages.
The old antidepressant is prescribed at low doses for insomnia far more than its evidence supports. A Cochrane review found only a modest, low-certainty effect on subjective sleep and none on measured sleep efficiency.
In a phase 3 trial, tambotatug pelitecan extended median survival to 13.3 months versus 9.4 on standard chemotherapy — but the trial was open-label and run entirely in China.
In PROPEL 3, children on once-daily infigratinib grew 1.74 cm/year faster than those on placebo over 52 weeks. It is a pill, not an injection — but the endpoint is growth speed, not long-term health.
A 1,695-patient trial tested whether metoprolol helps people with COPD and no cardiovascular indication. It missed its primary endpoint — but, unlike an earlier trial, showed no signal of harm.
In QWINT-1, fixed-dose weekly efsitora cut HbA1c about as much as daily glargine in 795 insulin-naive adults — with a lower rate of significant hypoglycaemia and a quarter of the dose changes.
Alpha-blockers ease symptoms within weeks; 5-alpha-reductase inhibitors shrink the gland over months. In a 3,047-man trial, combining them cut progression risk by 66%. Newer office procedures spare sexual function.
The oral decongestant in most cold and sinus tablets — phenylephrine — works no better than placebo, US regulators now say. Saline and short-term sprays do more.
The familiar explanation — dehydration and lost salts — is the weakest part of the evidence. The better-supported idea is that cramping is a failure of nerve control, which is why a mouthful of pickle juice can stop one.
In a 258-patient phase 3 trial, the oral drug reduced transfusion needs in about 30% of adults with transfusion-dependent thalassaemia, versus 13% on placebo.
A pandemic waiver letting clinicians prescribe stimulants and other controlled medicines without an in-person visit has been extended a fourth time, to end-2026, with the permanent framework still a proposal.
In an 81-child trial, vosoritide raised annualised growth velocity by 2.33 cm a year against placebo in hypochondroplasia, which has no targeted treatment. The endpoint is growth speed, not final height.
In the LAIS trial, more patients given intravenous loberamisal recovered with little or no disability at 90 days — a rare positive result in a field littered with failed neuroprotectants.
Self-testing and self-managing warfarin cut clot events by roughly 40%, and self-management also lowered deaths — one of the few at-home tests with randomised evidence of real benefit.
A British Columbia study emulated a trial across 41,000 patients. Those started on take-home methadone within weeks to months of induction stayed in treatment longer and died less than those on daily witnessed dosing.
Enlarged male breast gland tissue is common and usually benign. In a study of 19,640 young males, about three-quarters of cases were idiopathic and only 3.4% were linked to underlying disease.
Tozorakimab, which blocks interleukin-33, lowered the rate of moderate or severe COPD exacerbations by about a third against placebo, without an eosinophil requirement to qualify.
AD109 cut the apnoea-hypopnoea index by four events an hour more than placebo over 26 weeks. It did not improve fatigue, and one in five participants stopped because of side effects.
Zenagamtide was tested as a weekly injection and a daily pill in type 2 diabetes. Both cut HbA1c against placebo at 36 weeks, and the oral version's side effects rose steeply with dose.
Most sore throats clear on their own within a week. Painkillers and honey ease the pain; antibiotics shorten symptoms by only about 16 hours and are rarely worth it.
Satralizumab reduced eye-bulging and inflammation in thyroid eye disease across the twin SatraGO phase 3 trials, but the primary proptosis endpoint reached statistical significance in only one of them.
Tranexamic acid cut postpartum haemorrhage in placenta praevia. Dapagliflozin nearly halved acute kidney injury after cardiac surgery. The effect sizes are very different, and so is how much to trust them.
MariTide antagonises the GIP receptor that tirzepatide switches on, yet in 592 adults it drove 12–16% weight loss over a year — and it is dosed once a month, not once a week.
A small single-arm trial of the PD-1/CTLA-4 antibody cadonilimab with lenvatinib reported a 40.6% response rate in previously treated advanced endometrial cancer.
A section 564 declaration, prompted by a July determination from the Secretary of War, opens the door to authorising unapproved drugs for acute pain in combat and CBRN casualties.
A 411,000-patient claims study found adults on GLP-1 drugs who started oxycodone had more severe constipation and bowel obstruction at 30 days than those starting hydrocodone or tramadol.
Stargardt disease, the most common inherited macular dystrophy, has no approved treatment. In the 50-patient TEASE-1 trial, gildeuretinol slowed atrophic lesion growth by a fraction of a millimetre a year.
In an early-phase trial of 136 previously treated patients, daraxonrasib produced responses in 31–37% depending on dose — but 54% had a serious side effect, and this is a phase 1–2 study.
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.
Reviewing and stopping unnecessary drugs in older people is safe in trials and reduces inappropriate prescriptions. Whether it makes them live longer or feel better is far less settled than the enthusiasm implies.
At recommended doses it is one of the best-tolerated painkillers. The liver harm concentrates at high doses, and half of the failure cases are unintentional, often from combining products.
The phase 3 VALOR trial met its primary endpoint at the higher dose of the oral TYK2-JAK1 inhibitor, with more serious infections — a benefit and a safety signal that arrive together.
Matched against DPP-4 inhibitors, GLP-1 users had slightly more diabetic retinopathy and more retinopathy procedures. Vision impairment and blindness did not differ — and the coding for those is poor.
The glucagon and GLP-1 dual agonist, already approved in China, cut bodyweight 18.1% at 16 mg and 15.6% at 10 mg over 32 weeks versus 0.9% on placebo in a US phase 2 trial.
In the phase 3 Launch-HTN trial, adults already on two to five drugs fell 9.1 mm Hg further on lorundrostat than placebo — the second aldosterone-synthase inhibitor to post positive data after baxdrostat.
Users were slightly less likely than non-users to attempt suicide in the two to four years after treatment. The FDA's 2005 boxed warning stands, and the analysis cannot explain the direction of the finding.
Observational studies had linked proton pump inhibitors to fractures, dementia, kidney disease and more. A randomised trial collected all of those outcomes and found only enteric infections.
A once-daily experimental pill beat placebo in a phase 3 trial and is under regulatory review — yet no study has tested its claimed edge over older dopamine agonists head-to-head.
Across studies, current NSAID use was tied to about a 73% higher odds of acute kidney injury, more in older people and those with existing kidney disease. The combination with two blood-pressure drugs is the real hazard.
A European task force argued the first-generation drugs sold over the counter should not be. On symptom relief the newer ones differ from each other too — and the most familiar performed worst.
Two Cochrane reviews back scopolamine and first-generation antihistamines for preventing motion sickness. Both found the same gap: not a single randomised trial has looked at relieving symptoms already under way.
Expert guidelines back antibiotics for moderate-to-severe cases and simple measures for mild ones. The popular Pepto-Bismol prevention habit rests on 1980s data — and a 2025 trial could not confirm it worked.
The older oral preventives managed 0.44 to 1.4. The larger change is that the new drugs work in people the old ones failed, and are tolerated well enough to stay taken.
GLORY-2 randomised 461 Chinese adults with obesity to a weekly GLP-1/glucagon dual agonist or placebo; weight fell 16.65% versus 1.50% at 60 weeks, alongside high rates of nausea and vomiting.
A Cochrane review of 64 trials found acetazolamide cut the risk of acute mountain sickness by about half. It is a genuine drug effect, not a folk remedy — and it is a prescription decision, not a supplement.
Dazukibart eased skin and muscle disease within weeks and held up for nearly two years — but the trials were tiny, manufacturer-run, and the makers say a phase 3 is needed to confirm it.
A prespecified analysis of the ATTRibute-CM trial found the TTR-stabilising pill slowed the decline in how patients rated their own health, with a number-needed-to-treat of six to keep one 'not worse'.
Patients in remission who stayed on the JAK inhibitor through a second year flared far less than those switched to placebo — 7.4% versus 59.5% — in an extension of the SELECT-GCA trial.
In February, FDA said it intended to act against compounded GLP-1 supply without saying how. The answer arrived in May: exclude semaglutide, tirzepatide and liraglutide from the list.
The SMART trial tested clesrovimab against palivizumab in premature and heart- and lung-affected infants. It was built to show safety, not to prove one prevents more RSV than the other.
In the phase 3 CORALreef AddOn head-to-head, enlicitide dropped LDL cholesterol 64.6% at day 56 — far past ezetimibe, bempedoic acid, or the two combined.
A randomised-withdrawal trial showed the antibody-lowering drug kept CIDP patients in remission. A 2026 extension reports lasting benefit, but with no placebo group to measure it against.
Petrelintide, a long-acting amylin analogue given without a GLP-1 drug, reduced bodyweight by up to 8.6% at 16 weeks across two small phase 1 trials and was well tolerated. The finding is early.
Adding the antipsychotic to an antidepressant beat placebo in two phase 3 trials. But the dose that worked in one failed in the other — a wrinkle worth carrying into the prescription.
In 328 patients with third-generation cephalosporin-resistant gut bacteria in the blood, an old, narrow-spectrum antibiotic met its non-inferiority mark, offering a way to spare carbapenems.
Oral paltusotine kept median IGF-I near the normal ceiling through week 207 in an open-label extension, but only 20 of 43 patients remained by then and the study had no comparison group.
In a 500-patient ICU trial across seven countries, bicarbonate infusions for metabolic acidosis in patients on vasopressors did not lower major kidney events or death at 30 days.
Final ALIGN results show the endothelin blocker preserved kidney function over 2.5 years in a rare kidney disease, yet the difference at week 136 just missed statistical significance (p=0.057).
In a UK trial of 1,956 critically ill adults, neither carbocisteine nor nebulised hypertonic saline shortened time on a ventilator, and each was tied to a specific harm.
In two industry-funded phase 3 studies, every child followed at least 16 months met the goal — but all had a grade 3 or 4 event, and one died of a conditioning-drug complication.
In the FIND-CKD trial, non-diabetic patients on finerenone lost kidney function at 3.3 versus 4.0 ml/min/1.73 m² a year — a small edge that extends a diabetes drug to a new group.
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.
In the phase 3 GCAptAIN trial the interleukin-17A blocker did not significantly beat placebo for sustained remission at one year, leaving the IL-6 inhibitor tocilizumab as the proven steroid-sparing option.
The anti-alpha-synuclein antibody did not significantly delay motor progression in the phase 2b PADOVA study, though a non-significant trend and secondary signals keep a phase 3 trial alive.
Two GSK-funded phase 3 trials found roughly 20% of patients on bepirovirsen reached a functional cure at 72 weeks versus none on placebo — alongside more adverse events and liver-enzyme rises.
Two large trials of the anti-ST2 antibody gave discordant results: a modest signal in the phase 2b study, and a missed primary endpoint for the same dose in the confirmatory phase 3.
In the phase 3 Integral-1 trial, cefepime plus the new β-lactamase blocker nacubactam cured 82% of complicated urinary infections versus 61% for imipenem, offering a way to spare carbapenems.
Cemdisiran, a gene-silencing drug given under the skin once every three months, cut daily-living scores more than placebo in antibody-positive generalised myasthenia gravis, a rare complement-driven disease.
Of 1,363 evaluable patients matched to drugs by tumor genetics, 7% became exceptional responders. Activity across all 37 drugs was modest, and severe side effects hit over a quarter.
Relacorilant kept blood pressure under control when patients who had responded were kept on it rather than switched to placebo. The pivotal trial randomised just 62 people.
In the phase 3 PATCH trial, oestrogen skin patches were non-inferior to LHRH injections for three-year metastasis-free survival, with fewer hot flushes but more breast swelling.
OCULUS was halted at interim analysis: continuing a GLP-1 or GIP agonist before endoscopy left 25.0% of patients with clinically significant retained stomach contents, versus 3.1% who held one dose.
For years the disorder was treated off-label. A phase 3 randomised-withdrawal trial won lower-sodium oxybate the first US approval for it — but its design measures what is lost on stopping.
In the open-label extension of the VIVID programme, remission rates stayed high at 104 weeks — but no placebo group ran alongside, so durability is described, not proven, against nature.
In the COSPIRIT-JIA trial, most children with two stubborn forms of juvenile idiopathic arthritis responded to ixekizumab by week 16 — but the main result had no control group.
In the ALLEGORY trial, 76.7% of patients with active SLE reached the SRI-4 response at 52 weeks on the B-cell–depleting antibody versus 53.5% on placebo, extending a drug already cleared for lupus nephritis.
In the LATITUDE trial, monthly cabotegravir–rilpivirine injections cut regimen failure to 22.8% from 41.2% on oral therapy among people with a history of missed doses, prompting an early stop.
Efimosfermin, an FGF21 analogue given every four weeks, was well tolerated over 24 weeks in 84 patients with fatty-liver disease — but this trial was designed to test safety, not whether it heals the liver.
The agency announced an intent to restrict GLP-1 active ingredients destined for non-approved compounded drugs, and warned that seizure and injunction are on the table without further notice.
An Israeli case-control study found an apparent fourfold risk that vanished once the year before diagnosis was excluded. A Nordic study of 17,232 cancers found no association at all.
Pooling 151 studies and 58,534 participants across 30 drugs, researchers ranked antidepressants by physiological side effects rather than efficacy. Some drugs raised cholesterol while reducing body weight.
Two trials showed more deaths on voxelotor than placebo. The registry data that triggered the 2024 precautionary suspension did not confirm the original signal — the trials did something worse.