FDA rejects Ascendis’ parathyroid candidate, specifying manufacturing concerns

The FDA issued a complete response letter to Ascendis Pharma for palopegteriparatide, a drug-device combination that is being tested as a treatment for hypoparathyroidism, citing dosing problems with the technology.

Jan Mikkelsen

The Danish biotech said Monday morning that the regulator’s letter cited concerns about the “variability of delivered dose.” The company said in early April that the FDA had “identified deficiencies” in the company’s application for palopegteriparatide, but the agency did not disclose specifics at the time.

Ascendis CEO Jan Mikkelsen told investors during a conference call that the FDA did not mention any other concerns about the therapy. Ascendis plans to request a Type A meeting within the next three months.

“We believe we are well prepared to address the FDA’s concerns related to control strategy,” Mikkelsen said.

The biotech is looking to get the prodrug approved for patients with hypoparathyroidism, which occurs when the body does not produce enough parathyroid hormone — which regulates levels of calcium.

Joseph Schwartz

Manufacturing and control issues with hypoparathyroidism drugs are not new. Takeda recently said it plans to discontinue Natpara, its hypoparathyroidism drug, next year due to manufacturing and supply issues. The drug, which is an artificial form of the parathyroid hormone, was recalled back in 2019 over the potential for rubber particulates to detach into the Natpara cartridge.

SVB Securities analyst Joseph Schwartz told investors the issue “looks fairly benign to us, relative to other issues that we have seen in the past, which might be tougher to resolve.”

Shares of $ASND were up about 18% in trading on Monday morning.

The company also said Monday that it’s expecting a decision from the European Commission on palopegteriparatide by the end of the year. If the treatment is approved, the biotech plans to first launch it in Germany early next year.

manufacturing
biotech
pharma
application
therapy
fda
shares
trading