Biomarin Pharmaceutical Inc. is paying $14.50 per share to acquire rare disease drug developer Amicus Therapeutics Inc. in an all-cash transaction valued at about $4.8 billion. The deal will bolster Biomarin’s commercial portfolio, adding Galafold (migalastat), the first oral treatment for Fabry disease, and Pombiliti (cipaglucosidase alfa)/Opfolda (miglustat), a two-component therapy for Pompe disease, while adding U.S. rights to late-stage candidate DMX-200, in phase III testing for focal segmental glomerulosclerosis, a rare and fatal kidney disease. The deal, approved by the boards of both firms, is set to close in the second quarter of 2026.

Patient deaths prompt halt in Merck-Daiichi phase III in lung cancer

Daiichi Sankyo Inc. said it voluntarily placed a partial pause in recruitment and enrollment in the phase III IDeate-Lung02 study of antibody-drug conjugate ifinatamab deruxtecan because of a higher than anticipated incidence of grade 5 interstitial lung disease events. The study was temporarily halted, according to the European clinical trial registry, in Germany, the Netherlands, Hungary, Italy, Spain, Belgium, Poland, Romania, Portugal and France. The U.S. FDA also placed a partial clinical hold on the trial of those with relapsed small-cell lung cancer, the company said. During the partial clinical hold, the FDA, Daiichi Sankyo and the independent data monitoring committee will review accumulated safety data and determine any necessary further actions, Daiichi added.

B7-H3 money tree? Target catching on in ADCs

Focused on antibody-drug conjugates (ADCs), Neok Bio Inc. made a moderate splash by emerging from stealth mode in November with a $75 million series A financing, with funds going in part to advance antibody-drug conjugate (ADC) candidate NEOK-001 targeting ROR1 and B7-homolog 3, or B7-H3, also known as CD276, a novel member of the B7 immune protein family that makes an intriguing cancer target. The news came on the heels of preclinical findings aired by Macrogenics Inc., while several other players, including GSK plc and Biontech SE’s advance B7-H3-targeting ADCs

Rare diseases drive November’s US drug approvals

The U.S. FDA approved 18 drugs in November 2025, including four new molecular entities. Notably, two of the NMEs targeted rare diseases: UCB SA’s Kygevvi for thymidine kinase 2 deficiency and Arrowhead Pharmaceuticals Inc.’s Redemplo (plozasiran) for familial chylomicronemia syndrome.

Neutrophils put the brakes on fat loss

Fat tissue balances energy by storing lipids during times of abundance and mobilizing them when needed, yet sustained metabolic stress demands mechanisms that limit excessive lipid loss. Researchers at the University of California San Diego report that stress-induced fat breakdown triggers a neutrophil response in visceral adipose tissue that feeds back to restrain lipolysis and preserve lipid reserves. Neutrophils play a central role in immune defense by responding rapidly to acute inflammatory cues and coordinating with other immune cells to promote the resolution of inflammation. Beyond acute responses, neutrophils also contribute to the pathology of chronic inflammatory diseases, including dermatological conditions, atherosclerosis, diabetes and autoimmune disorders.

Also in the news

Alnylam, Atavistik, Athira, Beone, Biodexa, Biontech, CAMP4, Celltrion, Cormedix, Curevac, Daiichi, Decoy, DNA Nanobots, Forx, GSK, Incyte, Innovent, Insmed, J&J, Lixte, Memo, Muvon, Novo, Orum, Ovid, Peptidream, Prism, Profound, Puretech, Pyxis, Sagimet, Salarius, Sangamo, Sermonix, Syremis, Takeda, Talus, Werewolf