Palopegteriparatide earns FDA approval for treatment of hypoparathyroidism
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Key takeaways:
- The FDA approved a once-daily parathyroid hormone prodrug for adults with hypoparathyroidism.
- Approval comes more than 15 months after FDA declined to approve the drug due to manufacturing concerns.
A parathyroid hormone prodrug developed to treat hypoparathyroidism received FDA approval, according to an industry press release.
Palopegteriparatide (Yorvipath, Ascendis Pharma) is a once-daily medication designed to provide continuous exposure to released PTH over the 24-hour dosing period. Originally branded as TransCon PTH, approval was based on FDA’s review of the drug’s clinical package submitted with the new drug application and findings from the phase 2 PaTH Forward and phase 3 PaTHway trials.
FDA approval comes more than a year after the agency initially declined to approve palopegteriparatide. As Healio previously reported, the FDA issued a complete response letter in May 2023 stating it had concerns related to the manufacturing control strategy for variability of the delivered dose in the drug/device combination product. There were no concerns related to data from the trials in the letter. Ascendis Pharma resubmitted the new drug application for palopegteriparatide in November 2023. The FDA accepted the resubmitted application for review in December 2023, according to an industry press release, with an initial Prescription Drug User Fee Act goal date of May 14, 2024. The PDUFA date was extended by 3 months on May 14 as the FDA determined the information in the re-submitted application was a major amendment and additional time was needed for a full review.
Ascendis said it plans to offer several patient services for palopegteriparatide through its U.S. Ascendis Signature Access Program. Services will include patient support for navigating the treatment and financial assistance programs.
“The consequences of hypoparathyroidism on the health and quality of life of our patients can be extraordinarily debilitating,” Lynn Kohlmeier, MD, an endocrinologist at Spokane Osteoporosis and Endocrinology, chair of HypoPARAthyroidism Association medical advisory board, and an investigator in the PaTHway trial, said in the release. “The ability to address the underlying cause of this disease is crucial and will be an important advancement for our patients with hypoparathyroidism.”
In the release, Ascendis said it plans to have an initial supply of palopegteriparatide available in the first quarter of 2025. The company also said it plans to ask the FDA for approval to commercialize its existing manufactured product, which could be introduced in the U.S. as early as the fourth quarter of 2024 if approved.
References:
FDA accepts for review resubmitted NDA for TransCon PTH (palopegteriparatide) in adult patients with hypoparathyroidism. Available at https://investors.ascendispharma.com/news-releases/news-release-details/fda-accepts-review-resubmitted-nda-transcon-pth. Published Dec. 11, 2023. Accessed Aug. 12, 2024.
Ascendis Pharma announces extension of U.S. Food and Drug Administration review period for TransCon PTH for adults with hypoparathyroidism. Available at https://investors.ascendispharma.com/news-releases/news-release-details/ascendis-pharma-announces-extension-us-food-and-drug-1. Published May 14, 2024. Accessed Aug. 12, 2024.
Perspective
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The new treatment for palopegteriparatide developed by Ascendis was approved for treatment of the rare disease chronic hypoparathyroidism by the FDA on Aug. 14. This prodrug slowly releases parathyroid hormone 1-34, giving stable levels of parathyroid hormone 1-34 to keep circulating calcium in the desired range over 24 hours. This prevents hypocalcemia leading to tingling paresthesias, muscle cramps, tetany, bronchospasm, laryngospasm, cardiac rhythm disturbances, seizures or loss of consciousness.
This is the only treatment for chronic hypoparathyroidism currently approved in the U.S. The timing of this approval was critical, as the only other medication previously approved for this indication has been off the market since 2019. Teriparatide, approved for treatment of significant osteoporosis, was never approved for treatment of chronic hypoparathyroidism, although it is sometimes used off-label for this purpose. Palopegteriparatide was well-tolerated in the phase 2 and 3 clinical trials in patients with chronic hypoparathyroidism, quality of life improved, and serum and urine calcium levels improved into the normal range as desired.
Palopegteriparatide is available currently in an expanded access program through the manufacturer and will be commercially available no later than the first quarter of 2025. This is a major advance in therapeutics for this endocrine disorder.
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