🇬🇧 AstraZeneca pulls prostate cancer filing: UK pharma AstraZeneca withdrew its application to expand Truqap (capivasertib) into PTEN-deficient metastatic hormone-sensitive prostate cancer. Truqap blocks AKT, a protein involved in cancer-cell growth, and is already used in certain breast cancers. The proposed prostate cancer use was particularly notable because the FDA approved it in June after a Phase 3 trial showed that adding Truqap to hormone therapy reduced the risk of disease progression or death by 19%. EMA has confirmed the withdrawal, but a detailed explanation has not yet been published.
🇫🇷 Orphelia gets a second shot with Kizfizo: Paris-based Orphelia Pharma won CHMP backing for Kizfizo, a liquid formulation of the chemotherapy temozolomide, for adults with newly diagnosed glioblastoma and adults and children with recurrent or progressing malignant glioma. Gliomas are tumors that arise from supporting cells in the brain, with glioblastoma among the most aggressive forms. The liquid formulation could make treatment easier for children and patients unable to swallow capsules. The recommendation follows a previous regulatory setback: CHMP rejected Kizfizo in 2025 for a different use, pediatric neuroblastoma. Orphelia was acquired by rare-disease specialist Orphalan later that year.
🇩🇰 Ascendis moves weekly achondroplasia drug toward EU market: Copenhagen-based Ascendis Pharma received a positive opinion for Yuviwel (navepegritide) for children aged 2 and older with achondroplasia, the genetic bone-growth disorder that is the most common cause of disproportionate short stature. The drug releases C-type natriuretic peptide (CNP), which counteracts the overactive growth signal responsible for the disease and promotes bone growth. In a 52-week study, children receiving Yuviwel grew 1.49 centimeters more per year than those on placebo. Crucially, Ascendis’ treatment is injected once weekly, compared with daily dosing for BioMarin’s established competitor Voxzogo.
🇸🇪 Sobi looks to take RNA drug into a much larger market: Stockholm-based Sobi secured CHMP backing to expand Tryngolza (olezarsen) into severe hypertriglyceridemia, a condition in which very high levels of triglyceride fats in the blood can trigger potentially life-threatening pancreatitis. The RNA-targeting drug reduces production of APOC3, a protein that helps regulate triglyceride metabolism. Already approved in Europe for the ultra-rare familial chylomicronemia syndrome, Tryngolza could now address a far larger population: Sobi estimates around 700,000 people across Europe’s five largest markets meet the proposed triglyceride threshold. In Phase 3 studies, the treatment cut triglycerides by up to 72%.
🇺🇸 Pfizer pulls European Ibrance expansion after EMA questions data: New York-based Pfizer withdrew an application to use its blockbuster CDK4/6 inhibitor Ibrance as maintenance therapy for HR-positive, HER2-positive advanced breast cancer. The combination is designed for tumors driven both by hormone signaling and the HER2 protein, following initial chemotherapy and HER2-targeted treatment. The FDA approved the use in June after the Phase 3 PATINA study showed a 24% reduction in the risk of progression. European regulators were less convinced: EMA said an independent review of scans did not clearly confirm the progression-free survival benefit reported by investigators. Pfizer plans further analyses.
🇺🇸 Revolution Medicines gets European backing for pancreatic cancer drug: California-based Revolution Medicines received a positive CHMP opinion for Rasonque (daraxonrasib) in previously treated metastatic pancreatic cancer — one of the deadliest solid tumors and an area where treatment advances have been scarce. The oral drug inhibits RAS, a family of cancer-driving proteins mutated in the vast majority of pancreatic tumors. In a 500-patient Phase 3 study, median overall survival reached 12.4 months versus 6.7 months with chemotherapy. EMA considered the medicine sufficiently promising to review it through its Cancer Medicines Pathfinder, allowing parts of the application to be assessed before the full dossier was submitted.
🇺🇸 Omeros fails to overturn European rejection: Seattle-based Omeros lost its attempt to reverse CHMP’s rejection of Yartemlea (narsoplimab) for transplant-associated thrombotic microangiopathy. The rare but potentially fatal complication can occur after a stem-cell transplant when damage to small blood vessels leads to clotting, organ injury and destruction of red blood cells. Narsoplimab is an antibody designed to block MASP-2, part of the complement immune system thought to contribute to the disease. The FDA approved the drug in late 2025, but CHMP has remained unconvinced that Omeros’ clinical evidence reliably demonstrates efficacy, confirming its negative opinion after a formal re-examination.
🇺🇸 Lilly brings its obesity pill closer to Europe: Eli Lilly secured CHMP backing for Foundayo (orforglipron) for both weight management and type 2 diabetes. Like injectable drugs such as Wegovy and Mounjaro, orforglipron activates the GLP-1 receptor to improve blood-sugar control, suppress appetite and support weight loss. The difference is chemistry: orforglipron is a small-molecule tablet rather than a peptide, allowing once-daily oral dosing without the food and water restrictions associated with some other oral GLP-1 drugs. The recommendation could add another major competitor to Europe’s fast-growing obesity market, where Lilly and Denmark’s Novo Nordisk are already battling for patients.
What’s next: A positive CHMP opinion is not yet a marketing authorization. The recommendations now go to the European Commission, which generally follows CHMP’s scientific assessment when deciding whether a medicine can be marketed across the EU.
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