Ascendis Pharma’s pursuit of the Food and Drug Administration (FDA) approval for its hormone replacement therapy, TransCon PTH (palopegteriparatide), has hit another hurdle, as the U.S. regulator announced a three-month extension for its review. The decision, now expected by August 14, 2024, comes after the submission of additional information by Ascendis, which the FDA deemed a “major amendment” to the new drug application (NDA).
This latest delay adds to the prolonged journey for Ascendis, which has been waiting for approval for its hormone replacement therapy for hypoparathyroidism, an endocrine disorder affecting approximately 70,000 people in the U.S. Hypoparathyroidism occurs when the parathyroid glands fail to produce adequate calcium, leading to muscle aches, spasms, and potentially severe complications like renal issues and cognitive impairment.
On Tuesday, the Copenhagen-based company disclosed the extension, which resulted in a 6% drop in its share price. Ascendis’ CEO, Jan Mikkelsen, expressed the company’s commitment to working closely with the FDA: “We have responded to all requests received to date from the FDA and will work with the agency as they continue their review of our NDA. Adults with hypoparathyroidism in the United States, who are receiving TransCon PTH in our clinical trials and our Expanded Access Program (EAP), will continue to receive their medication, and the EAP remains open for enrollment for eligible patients.”
Hormone Replacement Therapy TransCon PTH Faces FDA Delay, Gains Approval in Europe and UK
The extension comes just over a year after the FDA issued a complete response letter to Ascendis in April 2023. The letter cited concerns over a manufacturing issue and variability in the delivered doses of the hormone replacement therapy but did not request additional clinical data. This setback came despite promising results from a phase 3 trial in which TransCon PTH demonstrated its efficacy by raising calcium levels in 79% of patients and allowing 95% of patients to discontinue conventional therapies, such as oral calcium and active vitamin D.
Despite the delays in the U.S., TransCon PTH has found success in Europe. The European Commission granted marketing authorization to the drug, branded as Yorvipath, in November of last year. It was also approved in the U.K. in April and has since been launched in Germany and Austria. These approvals highlight the drug’s potential and the rigorous standards it has met in other major markets.
Hypoparathyroidism presents significant challenges for those affected, given the critical role of the parathyroid glands in regulating calcium and phosphorus balance in the body. Effective management of the condition is essential to prevent severe complications, making the availability of a reliable hormone replacement therapy like TransCon PTH crucial for patients.
Ascendis Pharma’s Proven Success with Skytroka Raises High Hopes for TransCon PTH Approval
Ascendis is not new to the FDA approval process, having successfully gained approval for Skytroka, a long-acting growth hormone for children, in 2021. Skytroka has since been a commercial success, with sales reaching 179 million euros ($194 million) last year. The company projects even higher sales for Skytroka this year, expecting figures between 320 million euros ($346 million) and 340 million euros ($368 million). This track record underscores Ascendis’ capability in bringing innovative therapies to market and managing regulatory processes.
The anticipation around TransCon PTH’s approval is high, given its potential to significantly improve the quality of life for those with hypoparathyroidism. The continued support from the FDA, as well as the ongoing EAP, ensures that patients currently benefiting from the therapy will maintain access during the review period.
Ascendis Pharma remains dedicated to addressing the needs of patients with hypoparathyroidism and is hopeful that the extended review period will ultimately lead to a positive decision from the FDA. The company’s efforts to provide comprehensive responses to the FDA’s queries reflect its commitment to regulatory excellence and patient care.
Resource: Ascendis Pharma, May 14, 2024
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