FDA
The senator, who has long advocated for expanding access to experimental therapies, reportedly called the FDA’s request for a sham surgery–controlled Phase 3 trial for uniQure’s Huntington’s disease gene therapy “bureaucratic idiocy.”
Industry and FDA representatives have reached a general agreement on planned pre-submission facility meetings but have expressed different views about the specifics.
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.
The FDA’s cancer advisors will discuss AstraZeneca’s application for the oral SERD camizestrant in breast cancer and the AKT inhibitor Truqap in prostate cancer.
Some biotechs that had seen regulatory setbacks under Center for Biologics Evaluation and Research director Vinay Prasad experienced stock bumps Monday morning. Under Prasad’s leadership, the rare disease space has suffered a series of controversial rejections.
Incyte is looking to expand the use of its PD-1 blocker Zynyz to treat non-small cell lung cancer. The FDA’s rejection did not flag problems with the drug’s efficacy or safety.
Center for Biologics Evaluation and Research Director Vinay Prasad will exit the FDA for the second time after a controversial run that saw the oncologist butt heads with colleagues and ruffle feathers in biopharma after presiding over the rejection of several rare disease therapies.
The FDA granted Johnson & Johnson a Commissioner’s National Priority Voucher in December 2025 after the combination of Darzalex and Tecvayli improved progression-free survival by 83%. The new approval is for second-line treatment of the blood cancer.
UniQure does not have to drill placebo burr holes in the skulls of patients with Huntington’s disease, an unnamed FDA senior official said on Thursday. Instead, the company would anesthetize them and put “one to three nicks” in their scalp.
Adam Urato, who is currently a vaccine advisor to the CDC, is closely associated with acting CDER director Tracy Beth Høeg and is a fellow skeptic of the use of selective serotonin reuptake inhibitors during pregnancy.
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