IXA FDA meeting update
Dear IXA members,
We would like to share with you the key points of a discussion that took place between your IXA council and two FDA representatives in Boston during the American Transplant Congress.
As you all know, xenotransplantation research has made significant progress during the last couple of years in a variety of preclinical models. Consequently, we believe that one or more xeno-based treatment methods may soon make their way to the clinic. The IXA council therefore opened a dialogue with the FDA, offering to make them aware of your scientific progress, and to seek their advice regarding regulatory issues that may arise in the US, and in other jurisdictions, as xenotransplantation moves towards clinical trials.
This was only an information-sharing meeting. Specifically, the FDA representatives warned us in advance that they would be unable to officially consider any changes to current policy, or commit to specific plans with respect to reconsidering official guidance regarding regulation of xenotransplantation. Members of the IXA council explained the bases for our enthusiasm about the near-term future of xenotransplantation. We then brought up 3 specific concerns regarding existing FDA guidance on xenotransplantation. These issues were: 1) the risk that PERV will emerge as a human pathogen appears to be low, and deserves to be recalibrated in light of improving prospects for therapeutic efficacy of one or more xeno-based treatments; 2) the requirement to assure 50 years of secure archiving of donor and recipient materials represents a formidable and arguably extraordinary burden for a clinical trial sponsor; and 3) some ambiguity exists regarding how genetically engineered source pigs would be classified with respect to the regulatory approvals path.
FDA shares IXA’s core goal: to facilitate the transition of xenotransplantation to the clinic, as safely as possible, as soon as preclinical evidence supports doing so. The FDA representatives gave their personal, individual opinions about the significant progress toward preclinical benchmarks, and we discussed the current regulatory recommendations. Unofficially, the FDA representatives were interested in exploring further discussions between the xenotransplantation community and the FDA. Specifically, the FDA representatives recommended that IXA provide a summary of the current ‘state of the art’ in those candidate xeno applications which appear closest to clinical application. That information will be reviewed within the FDA as the basis for making a decision regarding further official discussions with IXA and other interested parties, and regarding a possible recommendation to FDA and HHS leadership regarding revisiting xenotransplantation regulatory guidance. IXA was asked to be prepared to participate in a series of mutual information-sharing workshops with FDA subject matter experts for the purpose of better understanding each other’s viewpoints. IXA Council intends to send pertinent documents soon to the FDA.
Overall it was a very useful meeting. FDA leadership is now engaged, and aware that xenotransplantation research appears to be progressing rapidly towards clinical trials. Our meeting paved the way for further discussions and workshops. Importantly, we expect that future official interactions with FDA will include public consultative sessions open to all xenotransplantation researchers, potential sponsors, and other interested parties, in accordance with US regulatory policies and procedures.
On behalf of the IXA council we thank you all for your excellent work that triggered this discussion with the FDA.
Peter Cowan, IXA President
Muhammad Mohiuddin, IXA Councillor
WELCOME TO THE INTERNATIONAL XENOTRANSPLANTATION ASSOCIATION
Dear Colleagues and Friends,
It was a pleasure to see so many of you at the joint congress of IXA, IPITA and CTS, held in my home city of Melbourne in November 2015. The meeting was a great success both scientifically and socially. IXA and TTS provided a number of travel awards to allow young members to attend and present their work, and sponsored presentations by the 2015 IXA Honorary Member (Uri Galili) and the inaugural Reemtsma Lecturer (Mark Hardy). Several major advances in the field were highlighted, notably in heart, kidney and islet xenotransplantation; the rapid development of techniques for precise genetic engineering continues apace, with the promise of further prolongation of xenograft survival and the minimization of infectious risk. I was especially heartened by comments from members of the other Sections indicating that xenotransplantation is very much ‘back on the radar’ – hopefully a message that will reach our major funding agencies!
I am fortunate to be taking over the presidency of IXA at this exciting time. Under the able leadership of Takaaki Kobayashi, the Council has worked hard over the last 2 years to expand the Association and promote xenotransplantation. I would like to personally thank Takaaki, Immediate Past President Bernhard Hering, and outgoing Councilors Chung-Gyu Park, Gilles Blancho and Michael Breimer, for their invaluable contributions. Thanks also to those continuing on Council – Secretary/Treasurer Agnes Azimzadeh and Councilors Eckard Wolf, David Sachs and Shuji Miyagawa - and welcome to new Councilors Muhammad Mohiuddin, Wayne Hawthorne and Curie Ahn, and President-Elect Leo Buhler, who maintain the diversity of geographical location and areas of expertise in the Council. Our goals in 2016-7 include continuing to provide opportunities and benefits to members, promoting the active involvement of younger members, and ensuring that regulatory and ethical frameworks develop in line with advances in the field. Please feel free to contact your Council with any new ideas and suggestions.
We look forward to the next biannual scientific meeting, to be held in Baltimore in 2017, organised by Robin Pierson and Agnes Azimzadeh.
Let’s approach 2016 with a renewed sense of enthusiasm, excitement and optimism!