Update on FDA regulatory reviews of TransConPTH in hypoparathyroidism
Ascendis Pharma A/S announced that the FDA has notified the Company that, as part of their ongoing review, the FDA has identified deficiencies in the Company’s New Drug Application (NDA) for TransCon PTH (palopegteriparatide) in hypoparathyroidism that at this time precludes them from holding further discussions about labeling and post-marketing requirements/commitments
The deficiencies were not disclosed in the letter. The FDA also stated that this does not reflect their final regulatory decision on the Company’s application.
“This notification from FDA relates to their review of our NDA, and since the NDA deficiencies were not disclosed in the letter, we are eager to work with FDA to learn, understand, and address them. This development a month from the agency’s PDUFA action date may lead to a delay in the FDA’s final regulatory decision on the TransCon PTH NDA,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “The safety of patients remains our highest priority and, since our NDA submission, no new safety signals have been observed to date in our ongoing TransCon PTH Phase II and Phase III clinical trials or in our Expanded Access Program (EAP) program and these programs continue unchanged.”
To date, 145 out of 154 clinical trial participants continue to be treated with TransCon PTH in Phase II and Phase III clinical trial open label extensions, including 57 patients in the Phase II PaTH Forward Trial (greater than 3 years), 76 in the Phase III PaTHway Trial (greater than 2 years), and 12 in the Phase III PaTHway Japan ( greater than 1 year). In these studies, TransCon PTH has been generally well tolerated, with no discontinuations related to study drug.
Learning Zones
The Learning Zones are an educational resource for healthcare professionals that provide medical information on the epidemiology, pathophysiology and burden of disease, as well as diagnostic techniques and treatment regimens.