When FDA grants a Rare Pediatric Disease (RPD) designation, it...

When FDA grants a Rare Pediatric Disease (RPD) designation, it means the FDA recognizes that the drug is intended to treat a rare pediatric disease, one that affects fewer than 200,000 children in the United States. This designation doesn't imply that the FDA believes the drug is likely to be approved; rather, it acknowledges the potential importance of the drug for addressing a significant unmet medical need in a rare pediatric population. The RPD designation also provides the opportunity for incentives, such as a Priority Review Voucher (PRV), if the drug is ultimately approved.

The sunset clause of approval by 30th Sep 2026 is the legislation rather than the FDA thinks we have a chance of approval. Why aren't we focusing on end stage heart failure when FDA has given the nod for potential AA. FDA will not approve 19 patients phase 2 trial, it's likely we will require a phase 3 trial.

See below from ChatGPT:

When a drug is granted Rare Pediatric Disease (RPD) designation, the rough timeline before the sunset clause affects the eligibility for a Priority Review Voucher (PRV) is as follows:

1: RPD Designation Date: The drug must receive RPD designation before September 30, 2024.
2: Approval Deadline: The drug must be approved by the FDA before September 30, 2026, to be eligible for a PRV.

These deadlines are subject to legislative changes, which may extend or modify these dates.