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EMA Releases Final Guideline for ATMP Clinical Trials
The European Medicines Agency (EMA) has published its finalized “Guideline on quality, non-clinical and clinical requirements for investigational advanced therapy medicinal products (ATMPs) in clinical trials”, effective July 2025. The guideline covers investigational gene therapies, cell-based therapies, and tissue-engineered products.
It outlines comprehensive requirements across manufacturing, quality documentation, non-clinical studies (including pharmacology, pharmacokinetics, and toxicity), and clinical development, emphasizing robust study design, endpoint selection, and long-term patient follow-up.
Special attention is given to first-in-human and confirmatory trials, as well as emerging technologies like genome editing, which require additional safety assessments.
To ensure product quality and participant safety, developers must document manufacturing processes, API characterization, and validated quality control methods.
Approved by CAT and CHMP, the guideline provides a critical regulatory framework to help ATMP developers meet EU standards while advancing innovative therapies safely and effectively.
04-04-2025