Wednesday, December 6, 2017

Advocacy at the FDA

Seven members of the XLH community went to the U.S. Food and Drug Administration on Monday to argue in favor of approving KRN23/burosumab for the treatment of XLH in both children and adults. The focus of our presentation was that XLH is a whole-body, whole-life disorder, not limited to bone growth issues and not limited to childhood.

Executive Director Carol LaFleur led the presentation with an overview of our position and some stories by patients who could not be present in person. Dr. Suzanne Jan deBeur talked about her concerns as a clinician and shared some of her patients' experiences during the clinical trials. Network Vice-President Joyce Inman talked about the research that we'll be embarking on in 2018 with our natural history study and about her experience as the mother of a spontaneous XLHer. Network Treasurer Geoff Edelson, Director Gin Jones and Network member Marina Velazquez shared their experiences as adults with XLH.

There were about two dozen representatives of the FDA present to to hear our stories. While of course they could not tell us anything about the ultimate decision that they'll be making, they were attentive and acknowledged our concerns that too little is understood about XLH, particularly in adults, and that while symptoms vary, at least some adults need ongoing treatment.

The FDA's decision is due April 17, 2018. We won't be sitting back and idly waiting for the decision though. We have a lot of work to do in the meantime to make sure that everyone is ready for what we hope will be the approval of burosumab for treatment of XLH in both children and adults.

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