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Advanced Therapy Medicinal Products for the Eye: Definitions and Regulatory Framework
Marina López-Paniagua1,2,3, Ana de la Mata1,2,3, Sara Galindo1,2,3
1Ocular Surface Group, Instituto de Oftalmobiología Aplicada (IOBA), University of Valladolid, Campus Miguel Delibes, Paseo de Belén 17, 47011 Valladolid, Spain.
Pharmaceutics
|April 3, 2021
Summary
Advanced therapy medicinal products (ATMPs) offer novel treatments for eye diseases. This review details the EU regulatory framework and classification criteria for these innovative therapies, using examples of approved products.
Area of Science:
- Ophthalmology
- Biotechnology
- Regulatory Science
Background:
- Advanced therapy medicinal products (ATMPs) represent a class of complex biological treatments including cell, tissue, and gene therapies.
- The application of ATMPs for ocular diseases is a rapidly expanding area, offering new therapeutic avenues for previously untreatable conditions.
- Familiarity with the regulatory landscape is crucial for professionals developing these innovative treatments.
Purpose of the Study:
- To provide an overview of the regulatory framework governing ATMPs in the European Union and other key international jurisdictions.
- To clarify the classification criteria for different types of ATMPs.
- To illustrate regulatory definitions with examples of ATMPs currently used in treating eye diseases.
Main Methods:
- Review of the legal framework for ATMP development.
- Analysis of classification criteria for ATMPs.
- Case study presentation of approved ATMPs for ophthalmic applications.
Main Results:
- The review outlines the specific legal requirements for ATMP development and marketing authorization within the EU.
- Key criteria for classifying somatic cell therapy, tissue engineered, gene therapy, and combined ATMPs are detailed.
- Examples of successfully developed ATMPs for eye diseases are provided to exemplify regulatory definitions.
Conclusions:
- Understanding the regulatory principles is essential for advancing ATMP development in ophthalmology.
- The established legal framework provides a pathway for innovative therapies to reach patients with eye diseases.
- The presented examples highlight the successful translation of ATMPs from research to clinical application in eye care.
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