For how we live.
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 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.
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.
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.
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.
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.