FDA cites ‘deficiencies’ in Ascendis’ application for thyroid drug, jeopardizing potential approval

The FDA is asking for more information about Ascendis Pharma’s drug for hypoparathyroidism, a rare condition in which the body doesn’t produce enough of a key hormone for regulating calcium.

In a letter, the FDA said it “identified deficiencies” in Ascendis’ application for palopegteriparatide, though the FDA did not disclose what those deficiencies were, according to Ascendis. The FDA also noted that the letter was not their final decision on the application, the Danish biotech said.

Palopegteriparatide is a long-acting prodrug of the parathyroid hormone, which regulates calcium and phosphorus levels in the body. With too little of the hormone, people with hypoparathyroidism can experience tingling or burning sensations, muscle pain, and fatigue.

Jan Mikkelsen

The FDA was supposed to decide on whether or not to approve Ascendis’ drug by the end of April. In a press statement, Ascendis president and CEO Jan Mikkelsen noted the FDA letter may result in a delay in the FDA’s deadline. Previous similar FDA letters have also led to rejection.

In the case of Ardelyx, the FDA rejected the biotech’s chronic kidney disease drug after sending a letter asking for more information. However, after a post-rejection advisory committee meeting, in which the outside panel of experts voted in favor of the drug, the FDA in December granted an appeal to the biotech’s drug. Previously, Provention Bio (now under Sanofi) received a letter and later a CRL for its diabetes drug, but won approval after resubmitting its application. In the case of women’s health biotech ObsEva though, such a letter led to disaster — the biotech ended work on the drug and laid off 70% of its staff.

Ascendis’ stock $ASND fell around 30% on the news to about $75.

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