Wednesday, June 28, 2017

FDA to consider both pediatric and adult issues

According to a recent Ultragenyx press release, the company has had a meeting with the FDA to discuss the planned submission for approval of KRN23/burosumab, with encouraging results. 

The press release states: "At the meeting, the FDA agreed that the BLA [Biologics License Agreement] can be submitted based on available clinical data and confirmed that both pediatric and adult indications would be included in the review. Based on the agreement, the submission of the burosumab BLA is planned for the second half of 2017."

Of particular importance to the XLH community is the FDA's decision to consider both pediatric and adult indications for the use of KRN23/burosumab, since previously it appeared that the FDA might only consider pediatric use of the new therapy during its review under the Breakthrough Therapy designation. Without FDA approval for adults, insurance companies might only cover the treatment for children, not for adults. Anecdotal evidence suggests that the treatment can be extremely beneficial for adults, not just for children.

The Network is investigating a variety of ways to provide the FDA with information from the patient perspective, particularly with respect to adult issues that need treatment, which have not been addressed as widely in the medical literature as the pediatric issues. We'll let you know  how you can help too as soon as we have more information.

You can read the entire press release here:

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