The Aleth Briefs trace each story to its original source and show how the week unfolded.
The week in five lines
The FDA cleared Novo Nordisk‘s Wegovy obesity pill.
BioMarin bought Amicus Therapeutics for $4.8bn.
The White House struck nine more drug-pricing deals.
CMS proposed mandatory international pricing for Medicare.
A jury hit Johnson & Johnson with $1.56bn over talc.
In this edition
Weekend catch-up (20 December-21 December)
BioMarin agreed to buy Amicus Therapeutics for $4.8bn in cash.
BioMarin will pay $14.50 a share, a 33% premium to last close and 58% to 60-day average, using cash and $3.7bn of new debt. Amicus brings Galafold in Fabry disease and Pombiliti with Opfolda in Pompe disease, which generated $599m over the past 12 months, plus US rights to phase 3 kidney candidate DMX-200. Expected to close in Q2 2026. BioMarin also disclosed that it had discontinued BMN 349, its oral candidate for alpha-1 antitrypsin deficiency (AATD) liver disease.
BioMarin, Amicus strike a $4.8 billion rare disease deal - BioPharma Dive
The White House announced MFN pricing deals with nine more drugmakers.
Nine major drugmakers agreed to extend most-favoured-nation prices to every state Medicaid programme and sell direct through TrumpRx below list price. The fact sheet cites Repatha falling from $573 to $239 and Epclusa from $24,920 to $2,425. The companies guaranteed MFN pricing for new launches and committed >$150bn in US manufacturing and research investments. The agreements bring the administration’s total to 14 since 30 September. Trump said insurers were next.
Fact sheet: President Donald J Trump announces the largest developments to date in bringing most favored nation pricing to American patients - The White House
Administration reaches deals with nine drug companies to lower drug costs - AHA
CMS proposed two mandatory Medicare models that tie rebates to what other rich countries pay.
GLOBE would cover Part B from 1 October 2026 and GUARD Part D from 1 January 2027, each reaching areas representing about a quarter of relevant patients. Both would replace the domestic inflation-rebate benchmark with one based on 19 OECD countries, including the UK, adjusted for purchasing power, and would be compulsory for drugs in scope. CMS estimates savings of $11.9bn and $14.1bn. PhRMA warned the proposals would divert billions from US drug R&D as China closes the gap.
Global Benchmark for Efficient Drug Pricing (GLOBE) Model - Federal Register
Guarding US Medicare Against Rising Drug Costs (GUARD) Model - Federal Register
Cytokinetics won FDA approval for its first product Myqorzo.
Myqorzo (aficamten), a once-daily cardiac myosin inhibitor, was cleared for adults with symptomatic obstructive hypertrophic cardiomyopathy ahead of its 26 December deadline. In phase 3 SEQUOIA-HCM, peak oxygen uptake rose 1.8 mL/kg/min at 24 weeks versus no change on placebo. A boxed warning and REMS requiring echocardiograms make monitoring a key issue against Camzyos. US launch is planned for the second half of January. Both China and the EU CHMP recently backed the drug.
Cytokinetics wins first approval, setting up a fight with BMS - BioPharma Dive
The FDA approved Boehringer’s Jascayd for progressive pulmonary fibrosis.
Jascayd (nerandomilast), a preferential PDE4B inhibitor, is the second drug approved for the indication and the first since Ofev in 2020. In phase 3 FIBRONEER-ILD, forced vital capacity fell 86 mL on 18 mg and 69 mL on 9 mg at 52 weeks, versus 152 mL on placebo. The trial allowed continued Ofev use; diarrhoea affected about half of those patients versus 37% on placebo plus Ofev. Adverse events were similar across groups.
FDA approves nerandomilast for progressive pulmonary fibrosis - PF Foundation
FDA approves Jascayd for treatment of progressiv pulmonary fibrosis - BI
AstraZeneca licensed a pan-KRAS inhibitor from China’s Jacobio for up to $2bn.
AZ paid $100m upfront for JAB-23E73, with up to $1.92bn in milestones and tiered royalties, taking global rights ex-China, where the two will develop and sell it together. The drug is in phase 1 in China and the US. KRAS mutations are present in about 23% of cancer patients, and the two approved inhibitors, Amgen’s Lumakras (sotorasib) and BMS’s Krazati (adagrasib), reach only the G12C variant, leaving the G12D and G12V mutations that dominate pancreatic and colorectal disease untreated.
Samsung Biologics agreed to buy Human Genome Sciences from GSK for $280m.
Coming soon after Congress passed The Biosecure Act, the deal hands the Korean CMO its first US plants: two cGMP facilities at Rockville, Maryland with a combined 60,000 litres of drug substance capacity and >500 staff (vs 785k litres at home). Samsung will keep making GSK’s existing products there, with closing expected by the end of Q1 2026. GSK bought Human Genome Sciences in 2012 for about $3bn.
Samsung Biologics expands US manufacturing capabilities with a strategic acquisition from GSK
Samsung Biologics to buy GSK’s US plant for $280 million - The Korea Herald
The FDA approved Genentech’s Lunsumio for relapsed follicular lymphoma.
Lunsumio VELO, administered in a minute rather than a 2-4 hr infusion, received accelerated approval for adults with relapsed or refractory follicular lymphoma after two or more lines. In phase 1/2 GO29781, the response rate was 75%, including 59% complete responses, with median response duration of 22.4 months. Cytokine release syndrome affected 30%, grade 3 in 2.1%. Genentech is positioning its fixed eight-cycle course against subcutaneous Epkinly, which moved into second line a month earlier.
FDA approves Genentech’s Lunsumio VELO for relapsed follicular lymphoma
AbbVie announces FDA approval of Epkinly for relapsed follicular lymphoma
Monday 22 December
The FDA approved a pill version of Wegovy, the first oral GLP-1 for weight loss.
The once-daily 25 mg semaglutide tablet is cleared both to reduce and maintain weight loss and to cut major cardiovascular events in overweight or obese adults with established cardiovascular disease. In the 64-week phase 3b OASIS 4 trial, 307 patients lost 16.6% of body weight, which Novo Nordisk says is similar to injectable Wegovy 2.4 mg, and one in three lost >20%. Novo will priced the 1.5 mg starting dose at $149 a month and said it would launch in the US in early January.
Shionogi agreed to pay $2.5bn for Tanabe Pharma’s ALS franchise.
Shionogi is buying a new company holding global rights to Radicava (edaravone), in both its intravenous and oral suspension forms, for a lump sum plus a possible royalty on future sales. It expects the franchise to add about $700m in annual global sales and to be accretive in its 2026 financial year, with closing on or after 1 April 2026. Shionogi said the purchase gives it a US rare-disease commercial platform ahead of its own planned launches in Fragile X, Jordan’s syndrome and Pompe disease.
A Baltimore jury ordered Johnson & Johnson to pay $1.56bn over talc-based baby powder.
Cherie Craft, 54, developed peritoneal mesothelioma after using talc powder for >40 years. A Baltimore jury awarded $59.8m in compensatory damages and $1.5bn in punitive damages against J&J and Pecos River Talc, finding J&J, two units and Kenvue liable for failure to warn. It is the largest talc award to a single plaintiff, amid >70,000 pending claims. J&J called the verdict unconstitutional and said it would appeal.
J&J talc jury awards $1.56 billion to asbestos cancer victim - Claims Journal
AZ’s ceralasertib missed overall survival in the phase 3 LATIFY lung-cancer trial.
The 594-patient study tested the oral ATR inhibitor with Imfinzi against docetaxel in locally advanced or metastatic non-small cell lung cancer without actionable genomic alterations, in patients who had progressed on immunotherapy and platinum chemotherapy. Safety was consistent with the known profiles of both medicines, with no new concerns. The data will be presented at a forthcoming medical meeting.
Update on the LATIFY phase III trial of ceralasertib - AstraZeneca
Neurocrine’s Ingrezza failed its phase 3 trial in dyskinetic cerebral palsy.
KINECT-DCP randomised 86 people aged 6 to 70 with choreiform movements to Ingrezza (valbenazine) or placebo for 14 weeks and missed both its primary endpoint, chorea score on the Unified Huntington Disease Rating Scale, and its key secondary endpoints. Nothing is approved for dyskinetic cerebral palsy, which accounts for about 15% of cerebral palsy cases; Ingrezza is already cleared in tardive dyskinesia and Huntington’s chorea, and sold $687m in Q3 2025, up 12% YoY.
Pfizer told haemophilia groups that a patient in a Hympavzi study had died.
A haemophilia A patient with inhibitors treated with weekly Hympavzi for three years, suffered a cerebellar infarction followed by cerebral haemorrhage after a minor urethroscopic procedure managed with recombinant factor VIIa. Pfizer is assessing causality with an independent monitoring committee, and advised close thrombosis monitoring around surgery when clotting factors or bypassing agents are used. Hympavzi was approved in the US in October 2024 only for patients without inhibitors.
WFH and NBDF statement on a severe adverse event with marstacimab - World Federation of Hemophilia
Statement on the marstacimab clinical trial adverse event - Coalition for Hemophilia B
Abbott won FDA approval for its Volt pulsed field ablation system.
The clearance covers symptomatic, recurrent, drug-refractory paroxysmal or persistent atrial fibrillation lasting under a year, and makes Abbott another major cardiac-device maker to enter the US pulsed-field ablation market. In the 392-patient VOLT-AF study, serious device- or procedure-related events within seven days were 1.9%; six-month freedom from recurrence was 93.1% in paroxysmal and 81.9% in persistent AF.
Premarket Approval (PMA) - FDA
Japan approved Guardant test as companion diagnostic for Lilly’s imlunestrant.
Guardant Health said the health ministry cleared Guardant360 CDx to detect ESR1 mutations in hormone receptor-positive, HER2-negative metastatic or recurrent breast cancer after endocrine therapy. Announced alongside approval of Lilly’s oral degrader, it is Japan’s first blood-based companion diagnostic for ESR1, found in 20% to 40% of patients previously treated with an aromatase inhibitor. In phase 3 EMBER-3, median progression-free survival was 5.49 months versus 3.84 on standard endocrine therapy.
Japanese approval of imlunestrant (EL25-74) - Eli Lilly Japan
Tuesday 23 December
The FDA approved Edwards Lifesciences’ Sapien M3, the first transseptal mitral valve replacement.
The valve is delivered through the femoral vein and septum in two steps, dock then valve, for patients unsuitable for surgery or edge-to-edge repair, including those with annular calcification. In the 299-patient ENCIRCLE trial, 95.7% had trace or mild regurgitation at one year, while 25.2% died or were hospitalised for heart failure, within the performance goal. Abbott’s Tendyne instead enters through the chest wall.
FDA approves Edwards Lifesciences’ Sapien M3 mitral valve replacement system
FDA approves Sapien M3 for transseptal mitral valve replacement - TCTMD
Weekend catch-up (27 December-28 December)
Diasorin won FDA clearance and a CLIA waiver for a four-in-one respiratory test.
The LIAISON NES panel separates influenza A, influenza B, RSV and SARS-CoV-2 from a dry nasal swab in 15 minutes across a three-step workflow, and the waiver lets it run in doctors’ surgeries and urgent care rather than certified laboratories. It is Diasorin’s first molecular point-of-care product, developed with an unnamed large US pharmacy chain and funded in part by BARDA.