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▎WuXi AppTec content team editor
Ascendis Pharma today announced that the U.
S.
FDA has accepted a New Drug Application (NDA) for the treatment of hypoparathyroidism in adults with the investigational drug TransCon PTH (palopegteriparatide) and granted priority review status
.
FDA expects to complete the review
on April 30, 2023.
In addition, Ascendis expects to submit a marketing authorization application (MAA) to the European Medicines Agency (EMA)
in the fourth quarter of this year.
Hypoparathyroidism is a rare endocrine disorder characterized by insufficient levels of parathyroid hormone (PTH), resulting in low
levels of calcium and phosphate in the blood.
Most patients develop the disease
after injury during thyroid surgery or accidental removal of parathyroid glands.
Conventional treatment with calcium supplementation and active vitamin D is not effective in addressing short-term symptoms, long-term complications, or quality of life effects
of hypoparathyroidism.
There is currently no replacement therapy
to restore physiological hormone levels.
TransCon PTH is a once-daily long-acting parathyroid hormone prodrug
.
It aims to restore PTH to physiological levels 24 hours a day to address the short-term symptoms and long-term complications
of the disease.
TransCon PTH has been granted orphan drug designation
for the treatment of hypoparathyroidism in the United States and European Union.
In a randomized, double-blind, placebo-controlled Phase 3 clinical trial published in March, a total of 82 adult patients with chronic hypoparathyroidism were enrolled
.
The results of the trial showed that 78.
7% of patients treated with TransCon PTH achieved independence from conventional treatment and maintained serum calcium levels within the normal range (8.
3–10.
6 mg/dL), compared with only 4.
8% (p<0.
0001)
in the placebo group.
In addition, patients with reduced patient experience with hypoparathyroidism experienced a statistically significant reduction
in physical and cognitive symptoms, as measured by the Patient Experience Scale (HPES).
In terms of safety, TransCon PTH is generally well tolerated and there are no investigational drug-related
discontinuations.
3 patients discontinued the drug during treatment: 2 in the placebo group and 1 in
the TransCon PTH group.
Post-treatment adverse events (TEAEs) were reported in 82% of patients in the TransCon PTH group and 100% of patients in the control group, most of which were grade
1 and 2.
"We are pleased that TransCon PTH has received priority review status and look forward to working with the FDA during the review process," said Dr.
Birgitte Volck, head of clinical development and medical affairs and senior vice president of Endocrine Rare Diseases at Ascendis, "We believe the results of our clinical trial point to TransCon PTH as a potential hormone therapy
for the treatment of adult patients with hypoparathyroidism.
" We understand the urgent needs of these patients and will continue to work hard to bring this medicine to patients
as quickly as possible.
”
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