TransCon PTH is designed as a long-acting prodrug of parathyroid hormone. It is intended to provide replacement therapy for hypoparathyroidism. Credit: Kateryna Kon / Shutterstock.

Ascendis Pharma has reported one-year data showing sustained improvement from its ongoing Phase III PaTHway Trial of TransCon PTH (palopegteriparatide), once-daily hormone replacement therapy, in adults with hypoparathyroidism.

This comes after the recent rejection of TransCon PTH by the US Food and Drug Administration (FDA), with the FDA citing concerns regarding the manufacturing control strategy for the variability of delivered doses in the TransCon PTH drug/device combination product.

Go deeper with GlobalData

Data Insights

The gold standard of business intelligence.

Find out more

Related Company Profiles

View all

The data from the randomised, placebo-controlled Phase III trial (NCT04701203) was presented at the annual meeting of the Endocrine Society (ENDO 2023) in Chicago, US.

Ascendis first reported the data from the Phase III trial in March 2022.

TransCon PTH is designed as a long-acting prodrug of the parathyroid hormone and is intended to provide replacement therapy for hypoparathyroidism.

Hypoparathyroidism is a rare disease where the parathyroid gland fails to produce adequate amounts of parathyroid hormone, or the parathyroid hormone produced is not biologically active. The disease is characterised by abnormally low calcium (hypocalcemia) and high phosphorus (hyperphosphatemia) levels in the blood.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

Therefore, conventional treatments include calcium supplements and activated vitamin D.

The FDA has approved recombinant human parathyroid hormone for patients with chronic hypoparathyroidism who are uncontrolled with conventional therapy, in accordance with National Organization for Rare Disorders (NORD).

TransCon PTH’s one-year data

The one-year (52-week) data is from the 156-week open-label extension (OLE) with TransCon PTH, an arm of the Phase III PaTHway Trial of TransCon PTH.

The 52-week results consist of a 26-week randomised period, with participants either on TransCon PTH or placebo, followed by 26 weeks of OLE with TransCon PTH.

Of the 82 study participants dosed at the start of the study, 79 completed the randomised period and entered the OLE, with 78 of these completing 52 weeks on TransCon PTH.

Of the 78 patients in the OLE, 74 achieved independence from conventional therapy, with no participant requiring active vitamin D supplements.

At week 52, 81% of the participants had normal serum calcium. Mean albumin-adjusted serum calcium levels of 8.9mg/dL were maintained through week 52 (normal range: 8.3mg/dL–10.6mg/dL).

TransCon PTH was well-tolerated in the extension period, with no new safety signals or trial discontinuations due to therapy.

Ascendis began enrolment in a German early access programme for TransCon PTH in June 2023. The company also expects to launch TransCon PTH in Germany following a possible positive decision on the marketing authorisation by the European Commission for TransCon PTH in the fourth quarter of 2023.