Ascendis Pharma Submits TransCon™ PTH New Drug Application to the U.S. FDA for Adult Patients with Hypoparathyroidism
• TransCon PTH could, if approved, become the first hormone replacement therapy to address the underlying cause of hypoparathyroidism, an area of major unmet medical need for the estimated 70,000-90,000 adults living with chronic disease in
• European MAA on track for Q4 2022 submission
“We believe the best way to treat hypoparathyroidism is to replace the missing endogenous hormone at stable, physiological levels 24 hours a day. Accordingly, our data support that TransCon PTH, if approved, could become the first-in-class therapy to address the underlying cause of disease and address the urgent needs of patients in the
The NDA is based on data from the global Phase 3 PaTHway Trial and the Phase 2 PaTH Forward Trial, as well as data from the Company’s ongoing open-label extension studies for both trials. Notably, 57 out of 59 patients continue in the open-label portion of the Phase 2 trial beyond two years of treatment, and 78 out of 79 patients continue in the open-label portion of the Phase 3 trial of TransCon PTH. In these studies, TransCon PTH has been generally well tolerated, with no discontinuations related to study drug.
In the fourth quarter of this year,
About TransCon™ Technology
TransCon refers to “transient conjugation.” Ascendis Pharma’s proprietary TransCon platform is an innovative technology designed to create new therapies that optimize therapeutic effect, including efficacy, safety and dosing frequency. TransCon molecules have three components: an unmodified parent drug, an inert carrier that protects it, and a linker that temporarily binds the two. When bound, the carrier inactivates and shields the parent drug from clearance. When injected into the body, physiologic conditions (e.g., pH and temperature) initiate the release of the active unmodified parent drug, in a predictable manner and at predictable levels. TransCon technology is designed to be applied broadly to a protein, peptide or small molecule in multiple therapeutic areas, and to be used systemically or locally.
About Hypoparathyroidism 1, 2, 3, 4, 5, 6
Hypoparathyroidism is a rare endocrine disorder characterized by insufficient levels of parathyroid hormone (PTH), resulting in low calcium and elevated phosphate levels in the blood. Hypoparathyroidism affects approximately 200,000 patients in the United States, Europe, and Japan, most of whom develop the condition following damage to or accidental removal of the parathyroid glands during thyroid surgery. Conventional treatment with calcium supplements and active vitamin D (also called calcitriol) does not effectively address the short-term symptoms, long-term complications, or quality-of-life impacts of hypoparathyroidism.
Short-term symptoms include weakness, severe muscle cramps (tetany), abnormal sensations such as tingling, burning and numbness (paresthesia), memory loss, impaired judgment, and headache. Patients often experience decreased quality of life, and, over the long term, this complex disorder can increase risk of major complications, such as calcium deposits in the brain, blood vessels, eye, and other soft tissues – including the kidneys, which can lead to impaired renal function.
Hypoparathyroidism remains among the few hormonal insufficiency states without a replacement therapy that restores the missing hormone at physiologic levels. Current standard of care with high doses of calcium and active vitamin D does not fully control the disease or address its underlying cause and may contribute to risk of renal disease. Patients with hypoparathyroidism have an estimated 4- to 8-fold greater risk of renal disease compared to healthy populations. Hypoparathyroidism is also associated with a 2-fold increased risk of depression or bipolar disorder compared to healthy populations.
This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding Ascendis’ future operations, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) the timing of topline results from PaTHway Japan, (ii) TransCon PTH’s ability to restore parathyroid hormone to physiological levels over 24 hours in adult patients with hypoparathyroidism, (iii) the potential for TransCon PTH to become the first hormone replacement therapy to address the underlying cause of hypoparathyroidism, (iv) Ascendis’ plan to submit a Marketing Authorisation Application for TransCon PTH in hypoparathyroidism to the
+1 (650) 374-6343
+1 (650) 709-8875
+1 (415) 513-1284
1 Mannstadt M, et al. Nature Reviews 2017, 3: 17055
3 Hadker N, et al. Endo Pract. 2014, 20(7);671-679
4 Powers J, et al. J Bone
5 Mitchell DM, et al. J Clin Endocrinol Metab 2012, 97(12): 4507-4514
6 Underbjerg L, et al. J Bone
Source: Ascendis Pharma