Fayuvi scored 23.5 points higher than untreated peers on cognitive testing, backing the first-ever approval for a Sanfilippo syndrome A gene therapy.
|Articles|May 21, 2021
Regulatory Requirements for Biologic Drug Development
Author(s)BioPharm International Editors
Developing and manufacturing a therapy in a regulated environment demands careful adherence to quality practices.
Advertisement
In this Expert Interview with BioPharm International, Steven Lynn, executive vice-president, pharmaceuticals, Regulatory Compliance Associates, shares insight on regulatory guidances, potential stumbling blocks on the development pathway, strategies to navigate the regulatory approval process, accelerated review applications, and maintaining quality standards.
Advertisement
Related to this article
Panelists from FDA, Genentech, and Minaris discussed raw material risk, supplier oversight, and at-risk release strategies for advanced therapies at the PDA/FDA Joint Regulatory Conference.
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.
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.
Advertisement
Advertisement