TransCon PTH approved in Europe as Yorvipath, under review in U.S.
PTH replacement therapy to treat adults with chronic hypoparathyroidism
The European Commission has approved palopegteriparatide, also known as TransCon PTH, to treat adults with chronic hypoparathyroidism.
Ascendis Pharma, the therapy’s developer, will market it in the European Union under the brand name Yorvipath, and expects the therapy’s commercial launch to begin in Germany next month. An approval request is before the U.S. Food and Drug Administration (FDA).
“By focusing on patient need and using science to drive our decisions, we have brought YORVIPATH — our second approved TransCon product — from concept through EU marketing authorization in only eight years,” Jan Mikkelsen, president and CEO of Ascendis, said in a company press release.
Yorvipath treats hypoparathyroidism by acting as a PTH replacement therapy
“Knowing the urgent need that many patients and physicians have expressed for new treatment options, we will continue our work to make YORVIPATH widely available,” Mikkelsen added.
Ascendis also announced the submission of a revised application asking that TransCon PTH be approved in the U.S. This followed a meeting with FDA regulators to discuss issues with manufacturing control over dose variability in the company’s initial application.
“With results from two … placebo-controlled clinical trials of TransCon PTH and no new safety concerns identified in follow-up reaching up to four years, we believe TransCon PTH is well-positioned to meet the needs of the hypoparathyroidism community,” Mikkelsen said in a separate press release, adding that “no concerns were expressed by the FDA about the clinical data submitted as part of our original [application].”
Hypoparathyroidism is caused by abnormally low levels of parathyroid hormone (PTH), which normally helps regulate levels of calcium and phosphorus in the body. Standard treatment for hypoparathyroidism involves supplements of calcium and vitamin D, which helps the body absorb calcium.
Yorvipath is a daily subcutaneous (under-the-skin) injection that contains a precursor to PTH that can be converted into the hormone once in the body. It is designed to lower the need for conventional treatments by normalizing parathyroid hormone levels.
“Each patient living with chronic hypoparathyroidism faces serious health and quality of life concerns,” said Lorenz Hofbauer, MD, a professor at the Technical University of Dresden in Germany. “These patients need new treatment options that go beyond the limits and risks of conventional therapy, which today consists of oral calcium and active vitamin D.”
In a Phase 3 clinical trial called PaTHway (NCT04701203), nearly all patients using Yorvipath long term were free from a need of conventional therapy, defined as being off of vitamin D and taking less than 600 mg/day of calcium supplement. Normalized calcium levels and a stop to vitamin D supplements in treated patients also were reported in the Phase 2 PaTH Forward (NCT04009291) study.
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