FDA Rejects Ascendis Pharma's TransCon PTH For Hypoparathyroidism

The FDA issued a complete response letter (CRL) for Ascendis Pharma A/S's ASND TransCon PTH (palopegteriparatide) New Drug Application (NDA) for hypoparathyroidism.

One month ago, the FDA notified deficiencies in its marketing application for TransCon PTH for hypoparathyroidism, though the deficiencies were not disclosed in the letter.

In the letter, the FDA cited concerns related to the manufacturing control strategy for variability of delivered dose in the TransCon PTH drug/device combination product. 

Investors are reacting to the fact that the FDA did not express concern about the clinical data submitted as part of the NDA package, and no new preclinical studies or Phase 3 clinical trials to evaluate safety or efficacy were requested in the letter. 

As recently announced, 145 of 154 clinical trial participants continue treatment with TransCon PTH for up to 3 years, and the U.S. Expanded Access Program continues to enroll new patients. 

TransCon PTH has been generally well tolerated in the clinical trials, with no discontinuations related to the study drug. 

The European Commission is expected to decide on the marketing application for TransCon PTH in Q4 of 2023. 

If approved, the company plans the first EU launch in Germany in early 2024. In addition, it expects to enroll the first patient in a German early access program, if approved, during Q2 of 2023.

Price Action: ASND shares are up 23.70% at $86.52 on the last check Monday.