While the studies showed statistically significant decreases in the primary endpoints, the FDA reviewers questioned the magnitude of the clinical benefit. Other issues raised by FDA reviewers included the “urologically heterogeneous” population of patients in the two studies, which included, for example, patients with small-capacity bladders and those taking medications for overactive bladder or benign prostatic hypertrophy.
Currently, there is no FDA-approved treatment for nocturia or nocturnal polyuria. Desmopressin, in a tablet formulation, is approved in the United States for treating central diabetes insipidus and nocturnal enuresis in children and is occasionally used off-label for patients with nocturia. It is also approved for preventing bleeding in patients with hemophilia A or type 1 von Willebrand’s disease with a subcutaneous injection or nasal spray formulation.
The 25-mcg and the 50-mcg doses of the desmopressin orally disintegrating tablet are equivalent to the 42-mcg and 83-mcg doses of the desmopressin tablet, respectively, according to the FDA.
The orally disintegrating tablet formulation of desmopressin was approved in Canada in 2014 as a treatment for nocturia due to nocturnal polyuria at the same doses were proposed for approval in the United States. Higher doses have been approved in more than 65 countries).
The FDA usually follows the recommendations of its advisory panels.
In a statement issued after the meeting, Dr. Paul Korner, senior vice president for U.S. development at Ferring, said, “We look forward to working with the FDA to ensure it has the data needed to complete its evaluation of the safety and efficacy of this medication.”
The FDA is expected to make a decision during the first quarter of 2015, according to the company.
FDA panelists were cleared of conflicts.