Ascendis Pharma’s palopegteriparatide was previously rejected by the regulator due to manufacturing problems, and the review period for its resubmitted application was extended by three months.
The FDA on Monday approved Ascendis Pharma’s palopegteriparatide, which was developed as TransCon PTH, for the treatment of adults with the rare endocrine disorder hypoparathyroidism. The drug will be marketed under the brand name Yorvipath.
The company is currently wrapping up the manufacturing of Yorvipath and expects the product to hit U.S. shelves in the first quarter of 2025. However, Yorvipath could be available before then if the FDA allows Ascendis to commercialize existing batches of the drug. In case of this approval, the company estimates that Yorvipath could reach the market in the fourth quarter of this year.
At launch, Ascendis will offer patients a “suite” of services through its access program, which seeks to help patients through the treatment process and provide financial assistance to eligible patients.
Patty Keating, executive director of the patient advocacy group HypoPARAthyroidism Association, in a statement said that Yorvipath’s approval is “such an important milestone” for patients suffering from the condition. “We look forward to having this new treatment option to help us move beyond the limits and risks of conventional therapy,” Keating said.
Hypoparathyroidism is a rare disease characterized by abnormally low levels of the parathyroid hormone, which are caused by dysfunctions in the parathyroid glands. Patients with this condition typically experience fatigue, weakness, muscle aches and spasms and dry skin. When left unchecked, hypoparathyroidism can also severely impair calcium levels in the blood, which can then lead to abnormal bone growth, developmental delays and calcium deposits in the brain.
Patients with hypoparathyroidism are usually prescribed oral calcium supplements or are given high-dose vitamin D treatments. However, neither of these can adequately control the disease.
Yorvipath is designed as a once-daily subcutaneous injection and is a long-acting prodrug of the parathyroid hormone. It addresses the underlying cause of hypoparathyroidism by restoring normal levels of the hormone for 24 hours each day, according to Ascendis’ website. As a result, Yorvipath can ease both the short- and long-term symptoms of the condition.
Ascendis first tried for an approval for Yorvipath in October 2022, which the FDA ended up rejecting in May 2023. In its Complete Response Letter at the time, the regulator cited manufacturing concerns but did not name safety or efficacy problems or ask for additional trials.
The company resubmitted its application a few months later, which the FDA accepted in December 2023 and gave an initial target action date of May 14, 2024. However, the regulator pushed back its decision date by three months after Ascendis filed a major amendment to the application.
