ASND: Transcon PTH CRL re Manuf Control Issues

COPENHAGEN, Denmark, May 01, 2023 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that the U.S. Food & Drug Administration (FDA) has issued a complete response letter (CRL) for the TransCon PTH (palopegteriparatide) New Drug Application (NDA) for the treatment of adults with hypoparathyroidism. 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. 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. Ascendis will request a Type A meeting with the FDA as soon as possible to agree on the best path forward.

“We are committed to working collaboratively with the FDA and, because the agency did not suggest that additional Phase 3 studies may be needed to demonstrate the product’s safety and efficacy, we believe we are well prepared to address their concerns,” said Jan Mikkelsen, Ascendis Pharma’s President and CEO. “People with hypoparathyroidism need new treatment options and we are working with urgency to resolve the FDA’s concerns, with the goal of bringing TransCon PTH to patients in the U.S.”

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. In the clinical trials, TransCon PTH has been generally well tolerated, with no discontinuations related to study drug. We anticipate a European Commission decision on our MAA during the fourth quarter of 2023. If approved, we are planning our first EU launch in Germany in early 2024. In addition, we expect to enroll the first patient in a German early access program, if approved, during the second quarter of 2023.

Conference Call and Webcast Information
Ascendis Pharma will host a conference call and webcast today at 8:30 am Eastern Time (ET).

Those who would like to participate may access the live webcast here , or register in advance for the teleconference here . The link to the live webcast will also be available on the Investors & News section of the Ascendis Pharma website at investors.ascendispharma.com . A replay of the webcast will be available on this section of our website shortly after conclusion of the event for 30 days.

About TransCon PTH
TransCon PTH (palopegteriparatide) is an investigational prodrug in development for adult patients with hypoparathyroidism. The safety and efficacy of TransCon PTH have not been established, and TransCon PTH is not currently approved by the FDA.

About Ascendis Pharma A/S
Ascendis Pharma is applying its innovative TransCon technology platform to build a leading, fully integrated, global biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of patients, science and passion, the company uses its TransCon technologies to create new and potentially best-in-class therapies. Ascendis is headquartered in Copenhagen, Denmark, and has additional facilities in Germany (Heidelberg, Berlin and Munich) and the United States (Palo Alto and Redwood City, California, and Princeton, New Jersey). Visit ascendispharma.com to learn more.