Expanded Access Program to TransCon PTH Opens to Clinicians
FDA decision on whether to approve TransCon PTH is expected by end of April
Online enrollment of an expanded access program for TransCon PTH, an experimental hormone replacement therapy for hypoparathyroidism, is now open to physicians who want to access the therapy for their patients, the treatment’s developer Ascendis Pharma has announced.
Ascendis announced last month that the U.S. Food and Drug Administration (FDA) had given the go-ahead for the expanded access program, which allows patients to access the medication before it is officially approved for sale in the U.S.
“We are pleased to have worked with the FDA to make TransCon PTH available for eligible patients in the U.S.,” Edward Trott, MD, vice president of global medical affairs at Ascendis, said in a company press release.
TransCon PTH also under review in Europe
The FDA is currently reviewing an application from Ascendis that seeks approval of TransCon PTH to treat adults with hypoparathyroidism, with a decision expected by the end of April. Ascendis is also seeking approval of the therapy in Europe.
“Recognizing the needs expressed by the hypopara community, we will continue to work with the FDA and others to make TransCon PTH, if approved, more broadly available as quickly as possible,” Trott said.
Hypoparathyroidism is characterized by low hormones of parathyroid hormone (PTH). TransCon PTH contains an inactive molecule that gets converted into PTH once inside the body. The experimental treatment is designed to be administered once daily via injection under the skin.
Ascendis’ applications for TransCon PTH in the U.S. and Europe are supported by findings from two clinical trials that compared the therapy against a placebo: the Phase 2 PaTH Forward study (NCT04009291) and the Phase 3 PaTHway trial (NCT04701203).
Findings from these studies indicated that treatment with TransCon PTH helped to normalize levels of calcium in the blood, as well as lessen the severity of hypoparathyroidism symptoms.
Patients in U.S. must meet eligibility criteria to access Transcon PTH
The program is open to adults, ages 18 and up, who have a diagnosis of hypoparathyroidism and have previously been treated with a PTH therapy. To be eligible, patients must have blood calcium levels of at least 7.8 mg/dL and vitamin D levels in the normal range at least two weeks before the first dose. They must also have a body mass index between 17 and 40 kg/square meter (effectively excluding patients who are very underweight or very overweight).
Expanded access is not open to patients in other clinical trials, or to individuals with some forms of heart disease or reduced kidney function. Patients are not eligible if they have a known risk for osteosarcoma, a form of bone cancer, or if they had cancer in the past two years (excluding thyroid carcinoma and non-melanoma skin cancer that have been successfully removed). The program is also not open to patients who are pregnant, lactating, or intending to become pregnant.