FDA
6th August 2013

The Results are In! FDA Panel Vetoes New Tolvaptan Indication

The Cardiovacular and Renal Drugs Advisory Committee voted 9 – 6 on Monday against endorsing an expanded indication for tolvaptan (Samsca), a product of Otsuka Pharmaceutical Co., Ltd., to treat autosomal dominant polycystic kidney disease (ADPKD). 

The Results are In! FDA Panel Vetoes New Tolvaptan Indication

The Cardiovacular and Renal Drugs Advisory Committee voted 9 – 6 on Monday against endorsing an expanded indication for tolvaptan (Samsca), a product of Otsuka Pharmaceutical Co., Ltd., to treat autosomal dominant polycystic kidney disease (ADPKD).

ADPKD is a rare disease that causes a proliferation of kidney cysts, resulted in grossly enlarged kidneys.

Tolvaptan was approved in 2009 to treat low serum sodium levels, but manufacturer Otsuka America Pharmaceutical is seeking to expand its indication to treat patients with ADPKD. There are no approved products for slowing the progression of kidney disease in patients with ADPKD, and the FDA granted orphan drug status for tolvaptan in April 2012 for that indication. Tolvaptan is a selective vasopressin V2-receptor antagonist that targets cyst growth and formation.

Panel members voiced concern that FDA reviewers had prior to the meeting regarding the inability to judge efficacy from the single trial supporting the treatment due to missing data. Furthermore, it was tough to balance the drug’s various safety issues including liver toxicity with the unmet need that ADPKD patients face.

“I felt that the benefit was real but very small. The risk was real but large,” Stuart Rich, MD, cardiologist at the University of Chicago Pritzker School of Medicine, said. “In balance, I felt the risk was too high given the marginal benefit.”

Many committee members agreed the drug was effective in reducing kidney size and pain, but differed in their opinion as to what extent.

“For me, this small change in GFR is not convincing and I have a major issue with the efficacy of the drug and slowing progression of renal disease,” said Vasilios Papademetriou, MD, staff cardiologist at the Veterans Affairs Medical Center in Washington, who voted against approval.

The FDA decision on the drug is due Sept. 1. The agency isn’t obligated to follow the advice of its advisory committees but usually does.

 

ISS has over a decade of experience in developing regulatory strategies including support for FDA Advisory Committee meetings. We are involved in more FDA AdComms per year than even the largest pharmaceutical companies. For more information on how ISS can help you prepare for your next meeting, contact info@innovativescience.net

 

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