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.
In the short term, symptoms include weakness, muscle cramps, abnormal sensations such as tingling, burning and numbness (paresthesias), memory loss, impaired judgment and headache. Patients often experience decreased quality of life. Over the long-term, this complex disorder can increase risk of major complications, such as extraskeletal calcium depositions, occurring within the brain, lens of the eye, and kidneys, which can lead to impaired renal function.
Hypoparathyroidism affects approximately 80,000 patients in the U.S., the majority of whom develop the condition following damage or accidental removal of the parathyroid glands during thyroid surgery.
Until recently, hypoparathyroidism remained among the few hormonal insufficiency states not treated by replacement of the missing hormone. Conventional therapy for hypoparathyroidism with active vitamin D analogs and calcium supplementation do not fully control the disease and may lead to renal disease caused by progressive calcification of the kidney and kidney stones resulting from increased calciuria. As a result, patients with hypoparathyroidism have an estimated 4-fold greater risk of renal disease compared to healthy controls.
TransCon PTH is designed to restore PTH to physiologic levels, normalizing blood and urinary calcium levels, serum phosphate levels and bone turnover.
TransCon PTH has completed a phase 1 clinical trial in healthy adults which evaluated safety and pharmacodynamics and supported its target profile as a true replacement therapy for hypoparathyroidism. A phase 2 trial of TransCon PTH in adult subjects with hypoparathyroidism is ongoing.
Hypoparathyroidism is defined as a decrease or lack of parathyroid hormone, leading to low levels of calcium in the blood and an increase in serum phosphorus. Calcium and/or vitamin D supplementation can help relieve symptoms, but these treatments can have undesirable side effects including an increased risk of kidney stones and chronic kidney disease.