Overview of Advanced Therapy Medicinal Products (ATMPs) in the European Union

Background

Advanced Therapy Medicinal Products (ATMPs) encompass cell therapies, gene therapeutics, and tissue-engineered products. Following the implementation of Regulation 1394/2007, only a few products have obtained centralized European marketing authorization.

Objectives

The study aims to provide an overview of ATMPs available within the European Union, either through centralized marketing authorization or national Hospital exemption. It also forecasts innovative ATMPs in the process of EMA approval and those in phase III and IV clinical trials.

Methods

A systematic literature search was conducted, including 'grey literature' and database reviews, following pre-defined search terms.

Results

Currently, 8 ATMPs are available via centralized marketing authorization, with 6 new product launches expected before 2020. Additionally, 32 ATMPs are available in individual EU member states via Hospital exemption, and 31 potential ATMP candidates are identified in industry-driven phase III research projects.

Conclusion

Advanced therapeutic medicinal therapies are still evolving, with innovative therapies targeting conditions such as retinal dystrophy, β-thalassemia, scleroderma, sickle-cell anaemia, adrenoleukodystrophy, and leukaemia expected to be available by 2020.

Introduction

ATMPs represent a shift towards personalized medicinal strategies, comprising somatic cell therapies, gene therapeutics, and tissue-engineered products. The Regulation 1394/2007 established a European Union-wide regulatory framework for ATMPs, requiring central marketing authorization issued by the EMA.

ATMPs with Central European Marketing Authorization

Since 2009, 12 products have obtained central European marketing authorization, with 8 currently available. These include gene therapies and cell-based therapies targeting various conditions.

ATMPs Withdrawn from the Market

Several ATMPs have been withdrawn due to commercial reasons, including Provenge®, MACI, Glybera®, and ChondroCelect®.

ATMPs in the Process of EMA Approval

Six new ATMPs are expected to be launched by 2020, offering new treatment modalities for various conditions. These include gene therapies and cell therapeutic products currently in the final stages of clinical trials.

ATMPs Available via Hospital Exemption

A significant number of ATMPs are available in individual EU member states through the Hospital exemption, allowing for the use of custom-made products for individual patients.

ATMPs in Phase III or IV Clinical Trial

Apart from those following the centralized marketing authorization pathway, numerous ATMPs are in phase III and IV clinical trials, targeting a wide range of conditions.

Conclusion

The landscape of ATMPs in the European Union is rapidly evolving, with a growing number of therapies expected to reach the market in the coming years. Despite challenges, the potential for personalized medicinal strategies offers hope for patients with previously untreatable conditions.