The European Medicines Agency (EMA) and European Commission on Friday published a joint action plan to further the development of new cell and gene therapies, as well as other advanced therapy medicinal products (ATMPs).

The plan, which features the release of several new and revised guidelines comes as on the international level, a regular forum for dialogue has been established to share experiences on advanced therapies between EMA, the US Food and Drug Administration, Health Canada and Japan’s Pharmaceuticals and Medical Devices Agency. 

Of the 18 marketing authorization applications submitted to EMA since the ATMP regulation came into force in 2009, EMA said nine products have been approved. 

But of those nine approved, four have been withdrawn from the market or suspended. For instance, UniQure’s Glybera, the first gene therapy authorized in Europe in 2012, was later withdrawn from the market. Similarly, Dendreon’s Provenge and TiGenix’s tissue-engineered product ChondroCelect, approved in 2009, were also withdrawn. Vericel Denmark’s Maci in 2013 was suspended at the recommendation of CHMP.

Other ATMPs remain on the market and include German company Co.Don AG’s Spherox, approved in 2017, MolMed Spa’s Zalmoxis, approved in 2016, as well as Chiesi Farmaceutici’s Holoclar, GlaxoSmithKline’s Strimvelis and Amgen’s Imlygic.

Background

The term ATMPs, according to EMA, is used to designate gene therapies, somatic cell therapies and tissue engineered products governed by Regulation 1394/2007 on advanced therapy medicinal products (also known as the ATMP Regulation).

The evaluation of ATMPs is led by EMA's Committee for Advanced Therapies, which offers a draft opinion before EMA’s Committee for Medicinal Products for Human Use (CHMP) adopts a final opinion and an authorization can be granted by the Commission.

The ATMP Regulation also allows member states to permit the use of advanced therapies that have not been authorized by the Commission under certain conditions, known as the "hospital exemption."

What’s Coming

As part of the action plan released Friday, before the end of the year, EMA says it will revise its procedures regarding the assessment of ATMPs: "To reduce administrative burden, avoid overlaps between the tasks of the various committees involved, and address the specific needs of ATMP developers (e.g. longer clock stops)."

The EC will also develop a guideline on GMPs for ATMPs: "To reduce administrative burden and adapt the manufacturing requirements to the specific characteristics of ATMPs. Subsequently to the adoption of the Guideline, EMA will organise specific training to inspectors with a view to achieve more harmonisation."

By the end of 2018, EMA is planning to release a draft guideline for consultation on investigational ATMPs to "create common standards for the assessment of these novel products" and to "avoid discrepancies across the EU regarding the requirements for ATMPs in the clinical trial phase."

In addition, in Q2 of 2018, there will be a revision of EMA’s Guideline on Safety and Efficacy and Risk Management Plans for ATMPs, "To reduce administrative burden in the post-marketing phase."

And scientific considerations on gene editing technologies will be put together by Q2 of 2018, "To reflect on emerging techniques on gene editing."

European Commission-DG Health and Food Safety and European Medicines Agency Action Plan on ATMPs