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FDA approves first, only treatment for hypoparathyroidism
Yorvipath, manufactured by Acendis Pharma, was approved as the first and only treatment for hypoparathyroidism on August 12, 2024.
Acendis Pharma secured the first and only approval for a drug to treat hyperparathyroidism on August 12, 2024. Yorvipath, also known as palopegteriparatide or TransCon PTH, is a prodrug of the parathyroid hormone that regulates and balances calcium and phosphorus in the body.
Hypoparathyroidism is a rare condition that affects up to 90,000 people in the United States. According to Johns Hopkins Medicine, the disease is caused when one or more of an individual’s four parathyroid glands are underactive.
An underactive parathyroid gland or hypoparathyroidism can cause lowered calcium and phosphorus levels, compromising muscles, teeth, and nerves. Although symptoms may vary, common symptoms include painful spasms in the face, hands, arms, and feet, pins and needles in the hands and feet or around the mouth, and seizures.
“The consequences of hypoparathyroidism on the health and quality of life of our patients can be extraordinarily debilitating,” said Lynn Kohlmeier, MD, an endocrinologist at Spokane Osteoporosis & Endocrinology, Chair of the Medical Advisory Board of the HypoPARAthyroidism Association, and an investigator in the PaTHway Trial, in the press release. “The ability to address the underlying cause of this disease is crucial and will be an important advancement for our patients with hypoparathyroidism.”
Traditionally, hypoparathyroidism is treated with intravenous infusions of calcium to manage symptoms. Some patients are advised to take calcium or active vitamin D pills and make dietary changes.
Yorvipath offers a different solution by administering injections of a prodrug of the parathyroid hormone to replace the hormone deficiency. The FDA approved the drug based on a 26-week, randomized, double-blind, placebo-controlled trial that studied 82 adults with hypoparathyroidism.
Among the patients randomized to the Yorvipath group, 69% maintained normal calcium levels without requiring active vitamin D or high doses of calcium. Comparatively, only 5% of patients in the placebo group could maintain normal calcium levels.
“FDA approval of our second TransCon product, YORVIPATH, reflects our values and dedication to following the science to help patients, as well as our unwavering commitment these past years to addressing the significant unmet medical needs of the hypoparathyroidism community in the United States,” added Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “We are deeply grateful to patients, clinicians, and advocates for their many contributions to this important milestone.”
