▲ApprovalUS9 Aug 20241 min read
Palopegteriparatide approved in the US after a year’s delay over its pen
Ascendis wins FDA approval for Yorvipath in adult hypoparathyroidism, after an earlier rejection that centred on dose consistency of the injection pen.
Filed → Approved The molecule is closer to patients than before.
The readThe science was never the issue; the device was. For daily self-injected peptides, the pen is part of the drug, and regulators review it that way.
The approval
The FDA approved palopegteriparatide (Yorvipath) for adults with hypoparathyroidism. In this condition the parathyroid glands, often damaged during neck surgery, make too little parathyroid hormone (PTH), and blood calcium drops. Standard care is calcium tablets and active vitamin D several times a day, which do not replace the hormone itself.
The evidence
In the PaTHway phase 3 trial, about four in five patients on the drug met the composite primary endpoint, which required normal blood calcium without active vitamin D and with limited calcium tablets, against about one in twenty on placebo. Symptom scores improved too.
The delay
An earlier application received a complete response letter in 2023. The FDA’s concern was the control strategy for the injection pen and how consistent each dose would be, not the trial data. Ascendis resolved that and refiled.
How it works
Palopegteriparatide is PTH(1-34) linked to a carrier through a TransCon linker that breaks down slowly in the body. The result is a steady hormone level from one daily injection, closer to how the gland works than short-acting PTH.
- Stage moves from filed to approved (US). The EU approved it in 2023.
Launch uptake, supply, and whether patients come off calcium and active vitamin D in routine care as they did in the trial.
Primary sourceFDA approval and Ascendis Pharma press release, August 2024; PaTHway phase 3
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