September PDUFA Dates

There are two drugs with September PDUFA dates: tenapanor, one of nine investigational Nephrology drugs and semaglutide, one of twenty-six investigational Endocrinology drugs.

Ardelyx’ tenapanor’s PDUFA Date is 9/12/2019, and is targeting hyperphosphatemia in dialysis patients, IBS with constipation. This NHE3 Inhibitor blocks the NHE3 transporter in the GI tract to reduce the absorption of dietary sodium, which reduces phosphate absorption and increases the amount of fluid in the gut. Tenapanor has the potential to reduce pill burden in reducing serum phosphorus, but may have a higher incidence of diarrhea. Research published in the March 7, 2019, Journal of the American Society of Nephrology concluded “Tenapanor significantly reduced elevated serum phosphate in patients with hyperphosphatemia receiving maintenance hemodialysis. Adverse effects were limited to those induced by its known mechanism of action, which increases stool sodium and water content.”

Novo Nordisk’s oral semaglutide has a September 20, 2019, PDUFA date. The drug is indicated for Type 2 Diabetes as a GLP-1 receptor agonist. The FDA approved the weekly subcutaneous injection on 12/5/17 for the treatment of Type 2 Diabetes. If approved, it would be the first oral GLP-1 receptor agonist and a potential game-changer for patients who wish to avoid an injection. Novo has announced the results from ten Phase III trials demonstrating a reduction in HbA1c and weight. Results of seven of the trials have been published. In June we provided a brief summary of the 10 trials. In the Pharmaceutical Pipeline Tracker, you will find additional information about trials and links to the seven published studies. The drug has been compared to placebo, empagliflozin, sitagliptin, liraglutide and dulaglutide with favorable results in the Phase III trials. A Phase II trial demonstrated similar efficacy with a daily oral tablet as achieved with a weekly injection of semaglutide. The adverse effect profile is similar to injectable GLP-1 receptor antagonists with nausea and vomiting being the most frequent ADR. There does not appear to be another oral drug in this class in the late stages of development. Lilly’s tirzepatide, an injectable GIP and GLP-1 receptor agonist is the most advanced drug behind oral semaglutide.