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FDA Advisors Recommend Against Approval of Tolvaptan for Treatment of Autosomal Dominant Polycystic Kidney Disease

Approval review date set for September 1 (August 6)

The FDA’s Cardiovascular and Renal Drugs Advisory Committee has voted against approval of tolvaptan (Otsuka Pharmaceutical Co.) for the treatment of autosomal dominant polycystic kidney disease (ADPKD). The committee voted 9 to 6 not to approve tolvaptan for ADPKD. The FDA is not bound by the committee’s guidance, but takes its advice into consideration.

ADPKD is characterized predominantly by the formation of cysts in both kidneys that cause progressive kidney enlargement. The disease is associated with pain, hypertension, decreased kidney function, and, ultimately, kidney failure. The average age at which ADPKD patients enter end-stage renal disease (ESRD) is 56 years. Currently, there are no treatment options specifically indicated for ADPKD, and physicians have had limited resources to manage the disease, relying on symptom management, dialysis, and transplantation as treatments of last resort when ADPKD progresses.

The FDA accepted the new drug application (NDA) for tolvaptan earlier this year, granting the drug a priority review status and assigning a Prescription Drug User Fee Act (PDUFA) goal date of September 1, 2013.

Tolvaptan is currently under review as a treatment to slow the progression of kidney disease for patients at risk of rapidly progressing ADPKD. The drug was studied in patients with enlarged kidneys who were in chronic kidney disease (CKD) stages 1 to 3 at the initiation of treatment. The results were published in the New England Journal of Medicine in December 2012.

Source: Otsuka Pharmaceutical Co.; August 6, 2013.

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