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Ascendis Pharma Announces Orphan Drug Designation Granted for TransCon PTH as Treatment for Hypoparathyroidism
Hypoparathyroidism is a rare endocrine disorder characterized by insufficient levels of PTH resulting in low calcium and elevated phosphate levels in the blood.
“We are pleased about the ODD for TransCon PTH, which reflects the need for a true PTH replacement therapy that more fully addresses all aspects of hypoparathyroidism,” said
The FDA grants orphan status to drugs that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the
TransCon PTH is designed to restore PTH to physiologic levels, normalizing blood and urinary calcium levels, serum phosphate levels and bone turnover.
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.
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.
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 four-fold greater risk of renal disease compared to healthy controls.
Ascendis Pharma is applying its innovative prodrug technology to build a leading, fully integrated rare disease company focused on making a meaningful difference in patients’ lives. The company utilizes its TransCon technology with clinically validated parent drugs to create new therapies with potential for best-in-class efficacy, safety and/or convenience.
Additionally, Ascendis Pharma has multi-product collaborations with Sanofi in diabetes and Genentech in the field of ophthalmology.
For more information, please visit www.ascendispharma.com.
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 our 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) our ability to apply our prodrug technology to build a leading, fully integrated rare disease company, (ii) our expectations regarding when we will initiate a phase 3 program for TransCon PTH, (iii) our expectations regarding our ability to create therapies with potential for best-in-class efficacy, safety and/or convenience and (iv) our product pipeline. We may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements that we make, including the following: unforeseen safety or efficacy results in our TransCon Growth Hormone, TransCon PTH and TransCon CNP or other development programs; unforeseen expenses related to the development of TransCon Growth Hormone, TransCon PTH and TransCon CNP or other development programs, general and administrative expenses, other research and development expenses and our business generally; delays in the development of TransCon Growth Hormone, TransCon PTH and TransCon CNP or other development programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; dependence on third party manufacturers to supply study drug for planned clinical studies; and our ability to obtain additional funding, if needed, to support our business activities. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to our business in general, see our current and future reports filed with, or submitted to, the U.S. Securities and Exchange Commission (
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