Advanced therapy medicinal product (ATMP)

An advanced therapy medicinal product is a medicinal product for human use based on genes, cells or tissues. Regulation (EC) No 1394/2007 distinguishes three categories: gene therapy medicinal products, somatic cell therapy medicinal products and tissue engineered products, to which combined products incorporating a medical device are added. These biological medicinal products are subject to a specific assessment and authorisation regime.

Authorisation of advanced therapy medicinal products

Authorisation must go through the centralised procedure. The Committee for Advanced Therapies of the European Medicines Agency prepares a draft opinion, which is submitted to the Committee for Medicinal Products for Human Use before the Commission’s decision. On request, it also issues recommendations on the classification of a product, which are useful for products at the borderline between several regimes. These medicinal products often benefit from conditional marketing authorisation, accelerated assessment or orphan medicinal product status, and regulatory data protection applies to them under the ordinary conditions.

The hospital exemption

The Regulation excludes from its scope advanced therapy medicinal products prepared on a non-routine basis according to specific quality standards and used within a hospital in the same Member State, under the responsibility of a medical practitioner, to comply with an individual medical prescription for a specific patient. These products require a national authorisation, issued in France by the ANSM. The exemption, essential for academic research, is purely regulatory and does not constitute an exception to patent law.

Intellectual property issues

Protection of advanced therapies relies heavily on process patents covering vectors, genetic constructs, methods of modifying cells or production methods. Proving their infringement often requires access to the competitor’s facilities, hence the importance of saisie-contrefaçon. The rules on biotechnological inventions exclude from patentability processes for modifying the germ line genetic identity of human beings, and the exclusion of methods of treatment leads to claims being drafted as products or uses. Finally, the concept of product within the meaning of the supplementary protection certificate may raise difficulties for cell-based products.

Key points

An advanced therapy medicinal product always falls under the centralised procedure, save for the hospital exemption granted at national level. The classification of the product should be checked early with the Committee for Advanced Therapies, and a portfolio then built combining product, process and use patents, regulatory protection and, where appropriate, orphan market exclusivity. In litigation, access to evidence of the processes is the first issue to anticipate.

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This glossary entry is general in scope and does not constitute legal advice.

Dhenne Avocats acts in pharmaceutical patent disputes and on the medicines regulatory questions that accompany them, from the marketing authorisation to the supplementary protection certificate.

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