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On 18 November 2022, the European Medicines Agency (EMA) published a draft reflection paper (Paper) for public consultation on the criteria for the evaluation of new active substance (NAS) status for biological substances. The Paper provides guidance for applicants on the elements required for a successful NAS claim, including practical examples and a Q&A. This is an area of significant practical importance for pharmaceutical companies and the Paper is a key element to consider during the development of regulatory strategies for biological, biotechnology-derived and advanced therapy medicinal products (ATMPs).

Chemical active substances are excluded from the scope of the Paper, given they are already addressed in two previous reflection papers. The Paper builds on these reflection papers and provides guidance to applicants based on the EMA’s experience with biological products to date, and the current scientific thinking.

The public consultation on the Paper is open until 31 May 2023.…

Continue Reading Evaluation of New Active Substance Status for Biological Products in the EU

On 14 July 2022, the European Commission published a proposal for a Regulation on the safety and quality of substances of human origin (SoHO) intended for human application. When adopted, the proposed Regulation will repeal and replace the currently applicable Directive 2002/98/EC on blood (the Blood Directive) and Directive 2004/23/EC on tissues and cells (the Tissue and Cells Directive), with the aim of reforming and modernising the existing EU legislation. The proposal sets out requirements and standards for the safety and quality of blood, tissues, and cells (BTC), as well as other SoHOs, through a single instrument that will apply in all EU Member States in a (hopefully) harmonised manner.

This will be a major development for life sciences companies operating in the EU, including companies developing advanced therapy medicinal products (ATMPs, such as cell and gene therapies) manufactured from or using SoHOs. The Regulation will apply from donation to human application, unless the SoHOs are used in the manufacture of medicinal products or medical devices, in which case the Regulation will apply to donation, collection and testing of the substances only. A public consultation is open until 8 September 2022, and the proposal will also be discussed by the Council and the European Parliament. Once the final text is agreed and adopted, it will come into force, with the proposal setting out a 2-year or 3-year transition period depending on the provision.…

Continue Reading EU Commission adopts Proposal for a Regulation on substances of human origin

On 16 October 2019, the European Commission published the Guidelines for Good Clinical Practice (GCP) specific to advanced therapy medicinal products (ATMPS) covering gene therapies, cell therapies and tissue engineered products. These Guidelines are available at the website of the European Commission.

Background

The European Commission was legally required to adopt the GCP Guidelines specific to ATMPs (the Guidelines) by Regulation (EC) No 1394/2007 (ATMP Regulation) with the technical input from the European Medicines Agency (EMA). The Guidelines were adopted after a period of public consultation with the targeted stakeholder consultation in the second half of 2018.

The Guidelines reflect the experience gained by the European Commission and the EMA in the field of ATMPs and in the assessment and authorisation of this type of medicinal products. Due to their complex nature, ATMPs present specific practical operational and regulatory challenges related to GCP compliance.…

Continue Reading Latest Updates on Good Clinical Practice for Advanced Therapies in the EU