Resolution criteria

The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of November 30, 2025 to complete its review of TransCon CNP (navepegritide) for the treatment of children with achondroplasia. This market resolves YES if the FDA approves the drug on or before November 30, 2025. Resolution will be determined by checking the FDA's official approval status at https://www.fda.gov/drugs/development-approval-process/drug-approvals-and-databases or Ascendis Pharma's official investor relations announcements. The market resolves NO if the FDA does not approve the drug by November 30, 2025, including if the decision is delayed beyond this date or if the application is rejected.

Background

The FDA accepted the NDA for Priority Review on June 2, 2025. The FDA informed Ascendis that they are not currently planning to hold an advisory committee meeting to discuss this application. The absence of an advisory committee meeting often suggests the application data is straightforward enough for reviewers to evaluate without additional expert input—a positive procedural signal.

Pivotal Week 52 results from the randomized double-blind, placebo-controlled ApproaCH Trial were published in JAMA Pediatrics on November 17, 2025, showing that treatment with TransCon CNP led to significantly higher annualized growth velocity at Week 52 compared to placebo, as well as improved lower-limb alignment and body proportionality and positive changes in health-related quality of life, with a safety and tolerability profile similar to placebo.

Considerations

Priority Review designation is granted for medicines that, if approved, would provide a significant improvement in the safety or effectiveness of the treatment, prevention, or diagnosis of a serious condition. While this accelerates the review timeline, PDUFA dates are goal dates and not guarantees—the FDA can extend review periods if additional information is needed. The tight timeline (8 days from today) means approval would need to occur imminently.