FDA Delays Review of Ascendis' Hormone Therapy by Three Months

Ascendis Pharma has encountered another obstacle in its pursuit of approval for its hormone replacement therapy, TransCon PTH (palopegteriparatide), intended for the treatment of hypoparathyroidism. The U.S. Food and Drug Administration (FDA) has prolonged its review of the medication by an additional three months, with a new decision date set for August 14.

The extension is due to the FDA requiring more time to evaluate new data submitted by Ascendis, which significantly amended the original application for TransCon PTH. According to CEO Jan Mikkelsen, the company has already addressed all the requests from the FDA so far and will continue to collaborate with the agency during its ongoing review process.

This delay comes after the FDA issued a complete response letter to Ascendis last year. The letter highlighted issues related to the manufacturing control strategy, specifically the variability in the delivered dose of the TransCon PTH drug/device combination. However, the FDA did not express any concerns regarding the efficacy or safety of the investigational drug in its letter.

The application for TransCon PTH by Ascendis includes clinical data from two significant trials: the Phase III PaTHway trial and the Phase II PaTH Forward trial. Results from these studies showed promising outcomes, with 79% of patients in the Phase III trial and 86% of patients in the Phase II trial achieving normalization of serum calcium levels and independence from conventional therapy after six months of treatment.

Despite these positive results, the need for additional information has resulted in the extended review period, causing another delay in the potential approval of TransCon PTH. Ascendis remains committed to working closely with the FDA to address any remaining concerns and to support the review process in the coming months.

Overall, this setback underscores the challenges that pharmaceutical companies often face in the regulatory approval process, particularly when it comes to complex drug/device combinations. Ascendis Pharma is dedicated to providing effective treatments for patients with hypoparathyroidism and is optimistic about the eventual approval of TransCon PTH.

As the August 14 decision date approaches, both Ascendis and the medical community will be closely monitoring the FDA's review process, hoping for a favorable outcome that could bring new therapeutic options to patients suffering from hypoparathyroidism.