FDA LogoFDA Responds to Tolvaptan New Drug Application

Otsuka Pharmaceutical Development & Commercialization, Inc. announced that they received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding the new drug application for tolvaptan to treat adult patients with ADPKD. The FDA issues CRLs to convey that their initial review of an application is complete and they cannot approve it in its present form. According to Otsuka, the FDA has requested they provide additional information. Next steps will be determined by Otsuka.

Like you, we were hoping for approval and are disappointed to hear this news. We know the PKD community is anxious for a treatment to become available and we share your frustration. We aren't giving up on our mission, and we don't want you to either. This is not the end of finding treatments, but the beginning.

Otsuka released this statement:

"Otsuka is evaluating the content of the FDA's response and will work closely with the Agency to determine if there are viable paths forward to address its outstanding questions," said Robert McQuade, Ph.D., Executive Vice President and Chief Strategic Officer, Otsuka Pharmaceutical Development & Commercialization, Inc. "Otsuka remains committed to patients with ADPKD and their healthcare providers." Read Otsuka's press release

We hope that Otsuka continues to pursue the approval of tolvaptan, and that other researchers and pharmaceutical companies seek development of drug treatments for PKD. In fact, there are several other clinical trials currently underway.

A special webinar will be held Sept. 12, 7-8:30 p.m. CST. Dr. Arlene Chapman, who played a critical part in the tolvaptan clinical trial, and testified at the FDA's Cardiovascular and Renal Drugs Advisory Committee meeting, will present and answer questions. One of the Foundation's medical advisors will talk about other clinical trials that are currently underway. A separate email will be sent with more details and registration information so please stay tuned or visit pkdcure.org for updates. We'll also discuss the Foundation's advocacy initiative to help patients share their voice about the need for treatments.


Some positive breakthroughs have occurred because of the tolvaptan clinical trials. A path has been opened up that has never been charted before in finding PKD treatments. The PKD Foundation is dedicated to continuing its critical work in the Accelerating Treatments to Patients program (ATP).

  • There are currently two drug candidates being screened for potential in being tested through the Foundation's drug repurposing program.
  • To move basic science forward, the Foundation is once again accepting research grant applications. In early 2014, funding of grants focused on the development of PKD therapies and the basic science of PKD will be announced.

It has also become clear that the voices of PKD patients are being heard. More than ever, it is time for individuals to share their voices to have an impact on decision-makers. Dialysis, transplantation and homeopathic living should not be the only options for people with PKD. We are developing tools to help PKD patients, their friends and family advocate about the importance of finding treatments.

We want to thank all who helped to get tolvaptan to where it is now. It is because of people like you who participated in clinical trials and research studies, submitted letters, and testified in person, that we came as far as we did. It is important at this critical time that we not be discouraged. While tolvaptan is an important piece in the fight against PKD, it is not the only one, and it is not over. We must continue to move forward in identifying new potential treatments and therapies. Thank you for your unwavering support. We will continue to keep you updated on any developments we learn about tolvaptan.

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©2014, PKD Foundation ·The PKD Foundation is a 501 (c)(3), 509 (a)(1) public charity.

©2014, PKD Foundation ·The PKD Foundation is a 501 (c)(3), 509 (a)(1) public charity.

The PKD Foundation is a 501 (c)(3), 509 (a)(1) public charity.
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