2012-10-05 Amicus/IPA Rotterdam Meeting Follow-Up

Jayne Gershkowitz
Category: Archive
October 5, 2012

From: Jayne Gershkowitz [mailto:jgershkowitz@amicusrx.com]
Sent: 05 October 2012 23:15
To: ‘Allan Pompe’; Tiffany House; ‘Thomas Schaller’; ‘Helmut.Erny’; Paula Waddell; Paula Waddell; ‘Ria Broekgaarden’; Zimmerman, Marsha A; maryze@xs4all.nl; ‘wilma treur’
Cc: Nita Patel
Subject: Amicus/IPA Rotterdam Meeting Follow-Up
Importance: High

Hello Everyone,

On behalf of my colleagues at Amicus, I want to thank each of you for a great meeting in Rotterdam last month. The discussions were very productive, the questions and comments all insightful and most helpful to us as we continue to work on the AT2220 pharmacological chaperone program for Pompe disease.

A message which we received loud and clear from all of you is that you want to see industry – those companies working in the Pompe “space” – collaborate as much as may be allowed by regulatory standards. Certainly, there are common goals we all share – industry and the patient advocacy community, alike – and that is to make life better for individuals affected by Pompe disease and their families. There is a range of issues that can play into any common goals, with developing treatments and access to approved treatments primary among them. Since our meeting I have been doing a great deal of thinking about the potential for collaboration and I am optimistic that it can happen, at least to a certain degree. In fact, in many ways, it already does occur. As a professional in the lysosomal storage disease field for many years, I already interact with my Patient Advocacy colleagues on a fairly regular basis. Biomarin and Amicus already have discussed, at a high level, how we can work together and with the IPA. We plan on meeting with our Genzyme counterpart soon to broaden this preliminary discussion.

In this spirit, and on behalf of Amicus Patient Advocacy, I submit this recommendation to the Board of Directors of IPA:

That the IPA strongly considers creating an Industry Partners Committee, comprised of one or two representatives from each of the companies currently working in the Pompe disease space with either an approved or investigational treatment in clinical development for Pompe. These representatives most likely would come from Patient Advocacy and possibly Medical Affairs or Commercial Operations, whichever is preferred by the IPA and the companies and which is in accordance with the individual company’s rules of Scientific Engagement and pharmaceutical industry guidelines. The IPA Board and its Industry Partners can meet twice yearly, at least once face-to-face. At these meetings, with agenda to be set by the IPA, information about current IPA business and future plans, Pompe patient community concerns and preferences, etc., can be discussed and shared in a true exchange of information and ideas with the companies. In this manner, all interested parties hear the same information at the same time and have the opportunity to hear each other’s opinions and share best practices, as appropriate. These public meetings would not include any proprietary agendas or preclude private meetings or TCs between the IPA and individual companies at other times during the year. Naturally, the Industry Partners committee could expand in the future to include any new or additional companies once they reach a certain level of drug development or involvement with the Pompe community. This model already exists with the Fabry International Network (FIN) and I think it is successful. While FIN is not as established a patient organization as is the IPA, I think the mutual information sharing that occurs between the FIN Board of Directors and its Industry Partners is productive and helps in building relationships on many levels.

Thanks for your consideration. I’m happy to discuss my ideas with you, so let me know if there are any questions for me. I look forward to receiving your notes from the Rotterdam meeting for our files.

Best regards,



Jayne C Gershkowitz
Senior Director
Patient Advocacy & Public Policy
Amicus Therapeutics
1 Cedar Brook Drive. Cranbury, NJ 08512
Direct: 609.662.2094
Mobile: 732.789.4320

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