FDA issues draft guidance for TKV

We continue to make good progress toward FDA support for TKV as a bio-marker for trial enrichment. I recently let you know that the FDA issued a formal letter of support to C-Path’s PKDOC (PKD Outcomes Consortium) for TKV as a potential bio-marker in trials of new therapies for ADPKD. This morning, C-Path announced that the FDA has issued draft guidance for this use.

The draft guidance provides qualification recommendations for the use of TKV in ADPKD clinical trials. Once guidelines are finalized and part of the official government register, biotechnology and pharmaceutical companies will be able to design trials with confidence that the FDA will accept TKV as an enrichment of outcomes. These guidelines will also help them find appropriate candidates, potentially improving the accuracy and efficiency of those trails.

In the words of David Baron, Ph.D., Chief Scientific Officer of the PKD Foundation, “This regulatory support is key to encouraging those developing potential treatments for ADPKD patients to carry on their work in what will hopefully be a more streamlined and productive path to potential approval of new therapies.”

According to Ronald Perrone, M.D., Medical Director, Kidney Transplantation, Professor of Medicine at Tufts University School of Medicine, Co-Director of PKDOC and PKD Foundation supporter, “The impact of this biomarker qualification will be to enhance the recruitment and performance of clinical trials in ADPKD, and hopefully bring new therapies to patients in a shorter period of time.”

The FDA has requested comments to this draft by Oct. 15, 2015. The Foundation’s strategy is to work with PKD experts to write an official comment as a sign-on letter to submit to the FDA.

The Draft Guidance for Industry document is on the FDA website and the C-Path PKDOC website, where you can also read the full press release.