For how we live.
GSK's PD-1 blocker won accelerated US approval on 19 October for previously untreated stage II/III dMMR rectal cancer. In the single-arm AZUR-1 trial, most patients avoided surgery, radiation and chemotherapy.
The FDA approved Tecentriq on 8 October, with chemotherapy, for Stage III mismatch repair deficient colon cancer after resection. In ATOMIC, adding it cut disease-free survival events, hazard ratio 0.50.
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.
On 13 October the FDA extended Velphoro (sucroferric oxyhydroxide) to children aged 6 to 9 on dialysis and added a 250 mg chewable tablet, widening an iron-based binder previously limited to patients 9 and older.
Hovilpri (pritelivir), a helicase-primase inhibitor, was approved on 9 October for immunocompromised adults whose herpes lesions resist aciclovir. In its trial, 63% healed by day 28 against 34% on comparator therapy.
On 14 October the FDA extended nogapendekin alfa inbakicept-pmln, with BCG, to BCG-unresponsive papillary-only disease - a first for that group - without a new trial, by extrapolating from its carcinoma-in-situ data.
Teva's subcutaneous depot is built to avoid the post-injection sedation that hobbled the first olanzapine shot. It cleared the FDA on 8 October; in the phase 3 SOLARIS trial it beat placebo on symptoms at eight weeks.
The pill joins trastuzumab and pertuzumab as maintenance after induction. In the 654-patient HER2CLIMB-05 trial it stretched median progression-free survival to 24.9 months from 16.3, a hazard ratio of 0.64.
Rhapsido becomes the first drug labelled for the itchy welts raised by scratching or pressure. In the RemIND trial, 29.3% on the pill cleared the provocation test at week 12 versus 14.0% on placebo.
The agency issued the tradable voucher to Ultragenyx for FAYUVI, an AAV gene therapy for the neurologic features of MPS IIIA that it approved on 17 September 2026. Such vouchers can be redeemed or sold.
An FDA advisory committee will meet on 30 October to review Sydnexis's atropine sulfate 0.01% eye drops, proposed to slow myopia progression in children aged 3 and older. The public docket closes 29 October.
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.
Merck's sotatercept gained an FDA efficacy supplement on 21 September. In the phase 3 ZENITH trial, a composite of death, transplant or hospitalisation hit 17.4% on the drug versus 54.7% on placebo.
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.
Eli Lilly's oral Inluriyo gained an FDA efficacy supplement on 18 September. In the phase 3 EMBER-3 trial, adding abemaciclib lifted median progression-free survival to 9.4 months from 5.5.
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.
Emcitate cleared the FDA on 28 September as a new molecular entity. In an open-label trial of 45 patients, the drug lowered the high serum T3 that drives MCT8 deficiency by about two-thirds.
Bristol Myers Squibb's mavacamten gained an FDA efficacy supplement on 30 September. In the pivotal EXPLORER-HCM trial, 37% on the drug met the primary endpoint versus 17% on placebo.
Rezzayo's once-weekly echinocandin can now be used in adolescents with candidaemia who have few other options. The expansion rests on adult data and modelling, not on studies in children.
BeiGene's PD-1 antibody, added to the HER2 drug zanidatamab and chemotherapy, lifted median overall survival to 26.4 months from 19.2 in the HERIZON-GEA-01 trial. The gain concentrated in the strongest HER2-expressers.
Cleared on 10 July under NDA 218579, the Provepharm agent fluoresces in near-infrared light to map vessels, perfusion and lymph nodes during surgery. Efficacy rests on studies of an earlier formulation.
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.
Regeneron's activin A antibody, brand name Pasatru, cleared the FDA on 19 August. In the pivotal 63-patient trial, new abnormal-bone lesions fell by 90% or more against placebo over 56 weeks.
The Scholar Rock antibody cleared the FDA on 11 September. In the phase 3 SAPPHIRE trial, apitegromab plus standard therapy beat placebo by 1.8 points on a motor-function scale at 12 months.
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.
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.
LITESPARK-022 cut the risk of recurrence by 28% when belzutifan was added to pembrolizumab after surgery. Survival was no different, and severe side effects nearly doubled.
Adding the immunotherapy to perioperative chemotherapy improved overall survival in resectable gastric and junction cancer — crossing the significance line the interim analysis had missed.
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.
The only FDA-approved drug for the penile-curvature disorder straightens the average man's bend only modestly more than placebo. Surgery is more definitive, and most oral remedies are unsupported.
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.
Two trials of oveporexton, an orexin receptor agonist for narcolepsy type 1, met their wakefulness endpoint and cut cataplexy sharply. Side effects were near-universal.
In the SKYLARK trial, a 14-day course improved depression scores faster than placebo. The effect was real but modest, and the label carries a driving-impairment warning.
In LIBERTY 1 and 2, more than 70% of women with fibroid-related heavy bleeding responded to the once-daily pill, versus under 20% on placebo. The label caps use at 24 months over bone loss.
EECOH-1 randomised 211 adults and found the weekly GLP-1 cut HbA1c up to 1.56 points more than placebo over 24 weeks — competitive numbers from a short, single-country, company-run trial.
In a 92-patient Greek cohort, the long-acting PTH replacement normalised calcium and phosphate and freed 58% of patients from calcium and vitamin D pills — echoing the trials, over a short follow-up.
In HERIZON-GEA-01, zanidatamab plus chemotherapy raised median progression-free survival to 12.4 months from 8.1 with trastuzumab. A clear survival gain came only when tislelizumab was added.
Orzeyful replaces signaling from the brain chemical narcolepsy patients lack, rather than stimulating wakefulness around the gap. Two-thirds of patients on the higher dose had trouble sleeping.
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.
The radioligand cut the risk of radiographic progression or death by 28% when added to standard therapy in a phase 3 trial — a real but incremental gain, bought with more side-effects.
A comparison of 2020–2025 approvals finds 94 novel oncology drugs from the NMPA against 87 from the FDA, with China's volume overtaking from 2023. The speed and first-in-class figures run the other way.
Simtriyo works on a different neurotransmitter pathway than existing stimulants. But its own manufacturer classifies it as a CNS stimulant, so a scheduling review stands before pharmacy shelves.
Lipfendra cut LDL cholesterol by more than half in two trials. Its significance is less the number than the format: a once-daily tablet where every prior PCSK9 inhibitor required a needle.
A comparison of FDA, EMA and ANVISA decisions from 2020 to 2024 finds Brazilian approvals arriving about a year later. A second study clocks the wait for public-system financing in years, not months.
In the MajesTEC-9 trial, the bispecific antibody teclistamab cut the risk of progression or death by 71% against standard three- and two-drug regimens. Serious infections were common.
Xocova is taken after a known exposure, not after symptoms appear. In a 2,041-person trial it cut symptomatic infection by 67% through day 10.
Beqalzi (sonrotoclax) is approved for relapsed mantle-cell lymphoma after a BTK inhibitor. Just over half of 103 patients responded in a single-arm trial with no survival data.
In VERITAC-2 the oral degrader more than doubled progression-free survival over fulvestrant in patients with an ESR1 mutation — 5.0 months versus 2.1 — but not in the trial as a whole.
About one in five adults with generalized myasthenia gravis test negative for the antibody Vyvgart was built around. A new trial, and a new approval, brings them into its label.
Bizengri now covers NRG1-fusion cholangiocarcinoma, a mutation found in a fraction of a fraction of cases. The evidence is 19 patients, of whom about a third responded.
The CHMP backed tolebrutinib for non-relapsing secondary progressive multiple sclerosis, a population with no prior disability-targeting treatment. The trial showed a 31% delay in progression.
Sparsentan cut proteinuria nearly in half in the DUPLEX trial's FSGS patients. Its effect on the eGFR measure that tracks actual kidney function decline was smaller, and comes with a liver-monitoring requirement.
The European Medicines Agency's March meeting produced five positive opinions, three of them for orphan-designated cancer and rare-disease treatments already available in some other markets.
Awiqli's approval for adults with type 2 diabetes rests on four trials comparing it with daily basal insulin. A pediatric dosing study is still due by June.
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.
A three-year update of RATIONALE-309 found tislelizumab plus chemotherapy lengthened median progression-free survival to 9.6 months from 7.4. The overall-survival gain was not statistically significant on its own.
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.
Voyxact cut urinary protein by about half at nine months in the VISIONARY trial. Whether that slows kidney decline is the question the accelerated approval leaves open until 2027.
The CHMP's November meeting produced ten positive opinions. Two of them — teplizumab and a Telethon-sponsored gene therapy — address conditions the EU has had no authorised treatment for.
Redemplo (plozasiran) is dosed once every three months. The approval carries required studies on liver injury, dysglycaemia and carcinogenicity, and skipped an advisory committee.
Kygevvi is a pair of oral nucleosides for an ultra-rare mitochondrial myopathy. There was no randomised trial — survival in treated patients was compared with untreated patients drawn from the literature.
Lynkuet is the second neurokinin-targeted drug cleared in the US for vasomotor symptoms. The trials found no hepatotoxic effects; FDA still asked for three years of enhanced liver-injury reporting.
Brensocatib cleared CHMP on an accelerated timetable with a 19.4% cut in exacerbation rate. The same October meeting issued a negative opinion on belumosudil and kept a sickle cell drug suspended.
Jascayd cleared on 7 October under priority review with orphan designation. The phase 3 trial it rests on measured lung function decline, not survival, and most patients were already on an antifibrotic.
Palsonify is cleared for adults whose acromegaly did not respond adequately to surgery. In the phase 3 switching trial, 83.3% on the pill kept biochemical control versus 3.6% on placebo.
Brensocatib blocks an enzyme that arms neutrophils rather than killing bacteria. In a 1,721-patient trial it cut yearly flare-ups by about a fifth — a modest effect in a disease with nothing else approved.
Sebetralstat was approved on 3 July for patients aged 12 and older. In its pivotal crossover trial, 76% of attacks treated with the 600 mg dose began to ease within 12 hours, against 49% on placebo.