FDA Approves First Treatment for Hypoparathyroidism in Adults
Ascendis Pharma said it is currently completing manufacturing of commercial product, which it anticipates will be available in the US in the first quarter of 2025.
The FDA approved palopegteriparatide (Yorvipath) for the treatment of hypoparathyroidism in adults, Ascendis Pharma announced in a release.1 The therapy is the first and only approved treatment in the United States for hypoparathyroidism, a rare endocrine disease that lowers the levels of calcium and increases levels of phosphorus in the blood. The disease occurs when 1 or more parathyroid gland is not active and affects 70000 to 90000 people in the country.
FDA Approves First Treatment for Hypoparathyroidism in Adults / Tada Images - stock.adobe.com
The approval was based on data from a clinical trial package, which included the phase 2 PaTH Forward and phase 3 PaTHway trials. In the phase 3 trial, palopegteriparatide—which was developed as TransCon PTH—demonstrated significant and sustained improvements in renal function in adults with chronic hypoparathyroidism. The treatment was also seen to be generally well tolerated.
READ MORE: FDA Rejects MDMA-Assisted Therapy to Treat PTSD in Setback for Psychedelic Medicine
“FDA approval of YORVIPATH is such an important milestone for our community,” Patty Keating, executive director of the HypoPARAthyroidism Association, said in a release.1 “We are thankful that the seriousness of our condition has been understood and our voices heard. We look forward to having this new treatment option to help us move beyond the limits and risks of conventional therapy.”
PaTHway (NCT04701203) was a multicenter, randomized, double-blind, placebo-controlled, parallel group phase 3 trial evaluating the safety, tolerability and efficacy of palopegteriparatide in adult patients with hypoparathyroidism. The study cohort included 82 patients who received either palopegteriparatide or placebo. The trial included a 26-week blinded period followed by a 156-week open-label extension period. Both groups were initially co-administered active vitamin D and oral calcium.
At week 52, 81% of patients treated with the therapy achieved independence from the conventional therapy of active vitamin D and oral calcium, and maintained mean albumin-adjusted serum calcium levels in the normal range. Scores from the Hypoparathyroidism Patient Experience Scale (HPES) and SF-36 Health Survey also showed that patients reported sustained improvements in disease-related physical and cognitive symptoms, as well as physical functioning and daily life.2
Additionally, treatment with palopegteriparatide resulted in a mean estimated glomerular filtration rate increase of 8.9 mL/min/1.73m2 from baseline at Week 52, which was sustained at Week 104.3 There were no new safety signals identified in the phase 3 study and most adverse events were mild to moderate in severity.
“The consequences of hypoparathyroidism on the health and quality of life of our patients can be extraordinarily debilitating,” Lynn Kohlmeier, MD, an investigator in the PaTHway trial, said in a release.1 “The ability to address the underlying cause of this disease is crucial and will be an important advancement for our patients with hypoparathyroidism.”
Ascendis had previously received a complete response letter for palopegteriparatide in May 2023 due to the FDA having concerns related to the manufacturing control strategy.4 The agency at that time did not express any concerns regarding the submitted clinical data for the therapy. Ascendis resubmitted the new drug application in December 2023 and was assigned a PDUFA date of May 2024.5 In May 2024, the FDA extended the PDUFA by 3 months to provide time for a full review of the submission.6
The company said it is currently completing manufacturing of commercial product, which it anticipates will be available in the US in the first quarter of 2025. Ascendis also plans to offer multiple patient services at product launch, including support for navigating the treatment journey and financial assistance programs for eligible patients.
“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,” Jan Mikkelsen, president and CEO of Ascendis, said in a release.1 “We are deeply grateful to patients, clinicians, and advocates for their many contributions to this important milestone.”
READ MORE: FDA Updates Resource Center
Pharmacy practice is always changing. Stay ahead of the curve: Sign up for our free Drug Topics newsletter and get the latest drug information, industry trends, and patient care tips, straight to your inbox.
