The Alliance for Regenerative Medicine Submits Comments to the European Medicines Agency’s Draft Guideline on Gene Therapy Medicinal Products

– September 1, 2015

BRUSSELS, Belgium and WASHINGTON, D.C., September 1, 2015 – The Alliance for Regenerative Medicine (ARM), the international organization representing the gene therapy, cell therapy and tissue engineering sectors, announced today it has submitted its comments in response to the European Medicines Agency’s (EMA) draft guidance entitled “Guideline on the Quality, Non-Clinical and Clinical Aspects of Gene Therapy,” first published for comment on May 20, 2015.

“ARM and its member companies welcome the publication of this guideline and the direction it seeks to provide those developing gene therapy medicinal products,” said Annie Hubert, director of European public policy for ARM. “Such direction is essential in moving these very innovative products forward to market while ensuring their quality, efficacy and safety.”

In its filed comments, ARM welcomes further guidance regarding quality, non-clinical and clinical requirements at various stages of development using a risk-based approach to the differential requirements for gene therapy medicinal products (GTMP) throughout the development cycle.

Given the very specific nature, and very small patient populations of many GTMP, the risk-based approach as outlined in annex I, part IV of Directive 2001/83/EC is particularly relevant and has been referred to in several of ARM’s proposed changes.

The organization also suggests that, in order to avoid unnecessary delays in commercializing GTMP, the EMA should ensure convergence of requirements for GTMPs with other international regions and encourage further collaboration and harmonization across ICH regions, aligned with the EMA’s own strategy to promote global medicines development.

“We have received significant input from ARM member companies, the majority of which are small- and medium-sized enterprises entirely dedicated to the development of GTMP, testifying to the interest and importance of this guideline to this sector,” concludes Jacqueline Barry, head of regulatory affairs, Cell Therapy Catapult and chair of ARM’s European Regulatory Affairs Subcommittee.

Once finalized, the guidance will replace the previous note for guidance on quality, non-clinical and clinical aspects of gene transfer medicinal products.

The draft guidance is available online here. ARM’s comments are available here.

About The Alliance for Regenerative Medicine

The Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine worldwide. ARM also works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its member companies and research organizations. Prior to the formation of ARM in 2009, there was no advocacy organization operating in Washington, D.C. to specifically represent the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today, ARM has more than 225 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visit