Ascendis Pharma will likely wait three more months to get an answer from the FDA on the review of its hypoparathyroidism drug.
On Tuesday, Ascendis announced that US regulators had delayed their decision on the drug, TransCon PTH (also known as palopegteriparatide), because of a “major amendment” to the company’s application.
It’s yet another hurdle for a drug that’s already been rejected once. Last May, the FDA rejected Ascendis’ application, citing “concerns related to the manufacturing control strategy for variability of delivered dose.” A month prior, the FDA had asked for more information on the drug after finding “deficiencies” in the new drug application.
“We have responded to all requests received to date from FDA and will work with the agency as they continue their review of our NDA,” Ascendis CEO Jan Mikkelsen said in a press release.
Patients getting the drug through the clinical trials and through an expanded access program can continue to be treated, the company said.
After last year’s rejection, Ascendis resubmitted the application last November. A month later, the FDA accepted it and set a decision deadline of May 14. The drug is under development in Japan with the company Teijin and in China by way of Visen Pharmaceuticals.
TransCon PTH has already been approved in Europe and Great Britain as Yorvipath, and about 100 patients were on the commercial treatment in Germany and Austria as of March 31, Ascendis said earlier this month.
If approved in the US, it would give patients a new option. Takeda has previously said it would stop manufacturing its hypoparathyroidism drug Natpara by the end of 2024, and let supplies run out. It will stop manufacturing Natpara globally at the end of December and it will “intend to supply available doses until inventory is depleted or expired,” a spokesperson for the Japan-based drugmaker confirmed to Endpoints News via email on Tuesday.
Meanwhile, AstraZeneca made a $1.05 billion deal in March to buy Amolyt Pharma, which is developing its own treatment. That drug is expected to have a Phase 3 topline readout before year’s end. AstraZeneca called it “one of the largest known rare diseases” that impacts about 115,000 people in the US and 107,000 people in the EU.
Low levels of the parathyroid hormone, key to balancing calcium and phosphate in the body, can lead to the endocrine disease, which can cause muscle pain, renal complications and other issues. Ascendis’ treatment is a prodrug of the parathyroid hormone.