Sarepta Therapeutics Inc.’s adventure with the Duchenne muscular dystrophy (DMD) AAV-based gene therapy Elevidys (delandistrogene moxeparvovec) continued as the firm said it would restart shipments of the compound for ambulatory patients “imminently,” with the U.S. FDA’s blessing.
Total venture capital funding in U.K. biotech was £1.23 billion (US$1.64 billion) in the first half of 2025, almost matching the 2023 total of $1.66 billion and looking to be in position to equal the 2024 12-month total of $2.8 billion.
With reduced sales and operating expectations for the rest of the year, Novo Nordisk A/S is on the receiving end of a huge stock drop. For a big pharma, where the stock drops are usually more modest, the shares (NASDAQ:NVO) fell off a cliff on July 29, closing 21.8% downward to $53.93 each, the lowest price per share in the past 12 months. Lower U.S. sales of semaglutide blockbusters Wegovy and Ozempic for treating obesity and diabetes are at the heart of the plunge. In May, the Danish company had expected 13% to 21% sales growth but now is looking at only 8% to 14%.
Radiopharma Clarity Pharmaceuticals Ltd. raised AU$203 million (US$132.22 million) in an institutional placement on Australia’s Securities Exchange to advance its late-stage pipeline of copper-based radiopharmaceuticals.
PTC Therapeutics Inc. will be launching its oral phenylketonuria therapy, Sephience (sepiapterin) in both the U.S. and Europe this summer, following the U.S. FDA approval just ahead of its July 29 PDUFA date. Sephience previously gained marketing authorization by the European Commission, roughly three months after a thumbs up from the EMA’s Committee for Medicinal Products for Human Use.
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Astrazeneca, Biogen, Biontech, Eli Lilly, Gilead, GSK, Ipsen, Krystal, Moderna, Novo Nordisk, Omeros, Pfizer, Roche, Sanofi, Servier, Sobi.
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