FDA advisory committee completes review of tolvaptan

The U.S. Food and Drug Administration’s (FDA) Cardiovascular and Renal Drugs Advisory Committee met on Aug. 5, 2013, to hear presentations from researchers, clinicians and the public. Several people associated with the PKD Foundation spoke about the burden of PKD, and the impact on entire families. The committee repeatedly discussed the burden of the disease, impact of kidney size and the problem of pain. They were also positively impacted by the 157 written testimonies that were submitted. These topics were brought to the forefront by these presenters, so we know their voices were heard. Thanks to everyone for helping when we put out requests for action.

After hearing these presentations, and reviewing and discussing data, the Advisory Committee voted 9 to 6 against approval of tolvaptan. The FDA is not bound by the committee’s guidance but takes its advice into consideration as the agency makes its decision. It is important to understand that this is not a final decision and not the end of the process. The FDA’s responsibility is to ensure the safety of drugs and they are conducting the appropriate review of the submission by Otsuka Pharmaceutical Co., Ltd.

“While we are disappointed in the Committee’s recommendation, we remain committed to providing patients and physicians with a novel treatment for ADPKD, a rare genetic disease. We are looking forward to continuing discussions with the FDA to address the panel’s concerns,” said Robert McQuade, Ph.D., Otsuka Executive Vice President and Chief Strategic Officer

We cannot speculate what the final decision will be from the FDA. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) goal date of Sept. 1, 2013. This means that the FDA should publicly announce their decision by that date. We will keep you informed as we learn any developments.

The meeting was recorded by the FDA. We will post the link as soon as it is available (possibly two to three weeks).

Read the press release from Otsuka