Ascendis Seeks EMA Approval of TransCon PTH for Adults in EU
FDA to decide by April 30 on whether to approve treatment in US
Ascendis Pharma is seeking approval of its treatment candidate TransCon PTH for adults with hypoparathyroidism in the European Union, the company has announced.
The request, submitted as a marketing authorization application (MAA) to the European Medicines Agency (EMA), follows a similar application submitted in September to the U.S. Food and Drug Administration (FDA). The FDA recently accepted and granted the submission priority review, a designation that can shorten the regulatory review from the standard 10 months to six months. A decision is expected by April 30, 2023.
What is TransCon PTH?
Hypoparathyroidism is caused by abnormally low levels of the parathyroid hormone (PTH), which regulates levels of calcium and phosphorus in the body.
TransCon PTH is a prodrug — an inactive compound that is converted by the body into an active therapy — that contains a long-lasting precursor of PTH. Delivered once daily via under-the-skin injection, the therapy was designed to normalize PTH levels for 24 hours.
“Our MAA submission is an important step in advancing our goal to bring TransCon PTH, if approved, to the substantial patient population in Europe who seek a treatment to address the short-term symptoms and long-term complications of their disease and support a normal daily life,” said Birgitte Volck, MD, PhD, senior vice president and head of clinical development and medical affairs, endocrinology rare diseases, at Ascendis.
Both EMA and FDA applications are based on results from the Phase 2 PaTH Forward trial (NCT04009291) and the Phase 3 clinical trial PaTHway (NCT04701203).
With a growing body of data showing durable, meaningful clinical and quality of life responses in the majority of treated patients, we believe TransCon PTH could, if approved, become the first treatment to address the underlying cause of hypoparathyroidism
What were the findings of the trials?
In the Phase 2 trial, patients were given once-daily TransCon PTH at one of three doses (15, 18, or 21 micrograms), or a placebo, for four weeks. Results showed that TransCon PTH outperformed a placebo at eliminating the need for conventional therapies as well as normalizing the levels of calcium in the blood and urine.
A total of 57 of the 59 people in the trial are now taking part in an open-label extension portion and have received the treatment over two years. After more than 1.5 years of treatment, patients continued to show normal calcium levels without a need for vitamin D supplements or high doses of calcium.
In the Phase 3 trial, 82 adults received either TransCon PTH or a placebo daily for six months.
Top-line data showed that significantly more patients given TransCon PTH had normalized their calcium levels without the need for standard treatments (78.7% vs. 4.8%). The therapy also outperformed the placebo at easing patient-reported symptoms and improving or stabilizing their physical function.
From the 79 participants who completed the trial’s six-month period and joined its open-label extension portion, 77 patients remain enrolled as of Sept. 30. In the extension study, the participants receive the experimental therapy for up to three years.
TransCon PTH has generally been well-tolerated. No participants discontinued the studies due to adverse events attributable to treatment.
“With a growing body of data showing durable, meaningful clinical and quality of life responses in the majority of treated patients, we believe TransCon PTH could, if approved, become the first treatment to address the underlying cause of hypoparathyroidism,” said Volck.
TransCon PTH has been granted orphan drug status — designed to boost the development of therapies for rare conditions — in the U.S. and in Europe.
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