Phase 3
Vertex Pharmaceuticals has a rolling biologics license application with the FDA for povetaciceptin IgA nephropathy. With new data from RAINIER, the biotech expects to complete its submission by the end of March.
Single-trial approvals are raising the bar on trial design and execution. The new paradigm is pushing sponsors to plan earlier, step up their data and risk‑based quality management and use modeling and AI to generate one compelling, regulator‑ready evidence package.
Roche’s shares tumbled nearly 5% on news that a key pillar in the five-pronged clinical plan for the breast cancer asset giredestrant was unsuccessful.
Azetukalner, a Kv7 potassium channel opener, reduced the frequency of focal onset seizures by a placebo-adjusted rate of 42.7%. Xenon Pharmaceuticals believes this is the highest such efficacy “observed in a pivotal epilepsy study,” CEO Ian Mortimer said Monday.
Ipsen will withdraw Tazverik’s follicular lymphoma and epithelioid sarcoma indications as emerging data point to an elevated safety risk in patients undergoing treatment.
The FDA last October paused Intellia Therapeutics’ late-stage CRISPR studies after detecting life-threatening enzyme elevations in one patient, who died a few days later.
Days after FDA Commissioner Marty Makary appeared to malign uniQure’s AMT-130 in an interview with CNBC, the agency confirmed to the biotech that a sham surgery–controlled study is needed before submitting the gene therapy for approval.
Aardvark Therapeutics is down 54% since Friday after the biotech said it detected “reversible cardiac observations” in a healthy volunteer study of its drug to treat extreme hunger in patients with the rare genetic disease.
A combination of Merck’s Keytruda and Pfizer’s Padcev could offer a chemotherapy-free treatment alternative for patients with muscle-invasive bladder cancer, even those eligible for cisplatin treatment.
Novo Nordisk reported a loss in a head-to-head trial of CagriSema against Lilly’s Zepbound earlier this week. This time around, Lilly’s orforglipron bested Novo’s oral semaglutide in blood sugar control and weight reduction—albeit with a few extra discontinuations as compared to its rival.
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