In mid-June, DHC's Master Practice Expert in Regulatory, Don Fink, Ph.D., contributed a guest column to cellandgene.com titled "EMA Guideline On Clinical-Stage ATMPs Comes Into Effect: On The Verge Of Convergence?"
It begins thusly:
On July 1, 2025, the EMA’s Guideline on quality, non-clinical and clinical requirements for investigational advanced therapy medicinal products in clinical trials1 is slated to come into effect. First adopted by EMA’s Committee for Advanced Therapies (CAT) in December 2018 with initial public consultation instigated February 21, 2019, the current version of the guideline was adopted by EMA’s Committee for Medicinal Product for Human Use (CHMP) on January 20, 2025. Twice released for public consultation, the advanced therapy medicinal products (ATMP) guideline has been intentionally compiled as a primary-source multidisciplinary reference document. It provides guidance and recommendations pertaining to the structural organization and content expectations related to quality, non-clinical, and clinical data to be included in clinical trial applications involving investigational ATMPs. The clinical trial applications are for ATMPs under study in either early-phase exploratory or late-stage confirmatory clinical trials, with the latter intended to provide pivotal data for a marketing authorization application (MAA) that represents a perspective bias of the guidance. The drafting of this guideline embodies a credible, if not overly ambitious, effort to consolidate information drawn from over 40 separate guidelines and reflection papers, as well as question and answer documents that delve into quality, non-clinical, and clinical aspects of investigational ATMPs that collectively comprise gene therapy medicinal products, somatic cell therapy medicinal products, tissue engineered products, and combined ATMPs (i.e., combination products).
Over the course of the guideline’s six-year maturation and curation...click here to continue reading at cellandgene.com.
