Regulatory and Standard Setting Organizations

FDA's Francis R.W. Godwin (CDER) and Kathleen Jones, PhD (CBER) detail a record pace of drug warning letters and the CGMP deficiencies most commonly cited in biologics facility inspections.

Sanofi's Dr Jack Prior detailed a data maturity framework for biologics manufacturing, while FDA and Gilead panelists debated risk-based AI validation.

FDA's consumer safety officer, Jason Chancey, detailed real inspection findings, such as taped gaskets and delayed retesting, behind sterile facility deficiencies.

Experts Harpreet Singh and Steven Quay discuss FDA expedited regulatory pathways for biologics, including Fast Track, Breakthrough Therapy, RMAT, Real-Time Oncology Review, and the National Priority Voucher Pilot Program, and explain how these mechanisms can accelerate development without lowering evidentiary standards.

Inebilizumab (Uplizna) cut MG-ADL scores by 1.9 points versus placebo, supporting its approval in Japan for myasthenia gravis.

Phio's PH-762 posted a 70% response rate in skin cancer, supporting its FDA briefing package for a planned phase 2b trial.

Lundbeck's asedebart, which blocks ACTH signaling at its source, has won FDA orphan status for endogenous Cushingsyndrome.

Satralizumab cut MOGAD relapse risk 68% in a phase 3 trial, positioning it as the first potential treatment for this rare disease.