Tag: #fda

FDA proposes excluding semaglutide, tirzepatide and liraglutide from 503B bulks list

FDA proposes removing semaglutide, tirzepatide and liraglutide from the 503B bulks list, potentially limiting outsourcing facilities' ability to compound GLP‑1 drugs.

#fda, #glp1, #compounding, #semaglutide, #tirzepatide

FDA approves Otarmeni, first U.S.-cleared gene therapy for genetic hearing loss

FDA approves Otarmeni, the first U.S. gene therapy for OTOF-linked deafness; Regeneron's dual-AAV treatment wins accelerated approval, follow-up data required.

#fda, #regeneron, #genetherapy, #hearingloss, #otology

Trump signs executive order to speed psychedelic treatments for serious mental illness

Trump signed an executive order directing agencies to speed development, review and patient access for psychedelic treatments for serious mental illness.

#psychedelics, #executiveorder, #mentalhealth, #fda

FDA Issues Draft Guidance on Using Next-Generation Sequencing to Assess Genome-Editing Safety

FDA's draft guidance explains how developers should use next-generation sequencing to detect off-target edits and chromosomal damage before human trials.

#geneediting, #fda, #ngs, #genetherapy

FDA Approves Lifyorli Combo for Platinum-Resistant Ovarian Cancer, Showing Survival Gain

FDA clears Corcept’s Lifyorli with nab-paclitaxel for platinum-resistant ovarian-type cancers, the first cortisol-receptor blocker to extend survival.

#ovariancancer, #fda, #chemotherapy, #drugapproval, #oncology

FDA proposes new pathway to approve one-patient gene-editing drugs for ultra-rare diseases

New FDA draft guidance would let individualized CRISPR and RNA therapies win approval with tiny studies, using strong biology and natural-history data.

#fda, #geneediting, #raredisease, #crispr, #drugapproval