Introduction

Advanced therapy medicinal products (ATMPs) are at the forefront of modern medicine, offering innovative solutions for treating diseases and injuries. These therapies, which include cell and gene therapies, provide unprecedented potential for long-term disease management and even cures, particularly in areas with high unmet medical needs. But what exactly are ATMPs, and how are they classified and regulated? This article explores the key aspects of ATMPs, their classifications, and the regulatory landscape that governs their use.

What are ATMPs

ATMPs are medicines designed for human use that leverage gene therapy, cell therapy, or tissue engineering for the treatment of both disease and injury. They are categorised into three main types:

• Gene therapy medicines: These contain genes that introduce therapeutic, prophylactic, or diagnostic effects by inserting recombinant (a stretch of DNA created in the laboratory) into the body to treat diseases.
• Somatic-cell therapy medicines: These involve cells that have been manipulated to change their biological characteristics, which may not be used for the same essential functions as before. They are used to treat, diagnose, or prevent diseases.
• Tissue-engineered medicines: These contain modified cells or tissues aimed at repairing, regenerating, or replacing human tissue.

As of August 2022, only 16 ATMPs have received approval from the European Medicines Agency (EMA), with 11 holding valid marketing authorisations. However, the number is expected to grow as more ATMPs undergo regulatory approval.

How are ATMPs Regulated?

Like conventional medicines, ATMPs are subject to stringent regulatory control. In Europe, the EMA regulates ATMPs under Directive 2001/83/EC, which provides the legal framework for their development. In the UK, the Medicines and Healthcare products Regulatory Agency (MHRA) oversees ATMP clinical trials, manufacturing, and imports.

ATMP Classification and Submission Process

To classify an ATMP, developers can apply for an official classification through the EMA. While this process is optional, it can provide access to specific regulatory support and incentives.

Required documents for ATMP classification

Applicants must submit two key documents:

1. Administrative form: this includes details such as the product name, active substance description, indication, company information, and submission date.
2. ATMP classification briefing document: this provides scientific and development details, including:
   • Active substance: composition, additional substances, and any related medical devices.
   • Finished product: mode of administration, pharmaceutical form, and final product description.
   • Mechanism of action: how the product works, its properties, and intended therapeutic applications.
   • Development status: an overview of manufacturing, clinical, and non-clinical development progress.

Submission and Review Process

Applications must be submitted via the EMA website in accordance with specified deadlines. Requests should be received at least 15 days before the start of the evaluation process. Once submitted, the Committee for Advanced Therapies (CAT) reviews the application and drafts a scientific recommendation, finalising the decision within 60 days.

The Future of ATMPs

ATMPs represent a promising frontier in medicine, with potential applications in a wide range of diseases. While significant progress has been made, further research and development are essential to fully understand their long-term health impact. As regulatory frameworks evolve, businesses and researchers must stay informed to navigate the complexities of ATMP development and approval effectively.

Summary

ATMPs are revolutionising healthcare by providing innovative treatment options for previously untreatable conditions. With rigorous classification and regulatory processes in place, these therapies must meet high safety and efficacy standards before reaching patients. As research continues and new therapies emerge, ATMPs will play an increasingly vital role in the future of medicine.

List of References

• https://www.ema.europa.eu/en/human-regulatory/overview/advanced-therapy-medicinal-productsoverview
• https://www.ema.europa.eu/en/documents/regulatory-proceduralguideline/directive-2001/83/ec-european-parliament-council-6-november-2001-community-coderelating-medicinal-products-humanuse_en.pdf
• https://www.gov.uk/government/organisations/medicines-andhealthcare-products-regulatory-agency
• https://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:L:2007:324:0121:0137:en:PDF
• https://www.gov.uk/guidance/advanced-therapy-medicinal-productsregulation-and-licensing
• https://www.ema.europa.eu/en/documents/scientific-guideline/reflection-paper-classificationadvanced-therapy-medicinalproducts_en-0.pdf
• https://www.ema.europa.eu/en/human-regulatory/marketingauthorisation/advanced-therapies/