Filing of the pediatric (ages 2–11) supplemental NDA for ORLADEYO in HAE, supported by positive Phase 3 APeX-P data. ORLADEYO is the only oral HAE prophylactic.
What’s at stake
ORLADEYO (berotralstat) is a once-daily oral therapy developed by BioCryst for the prevention of hereditary angioedema (HAE) attacks. HAE is a rare genetic condition in which C1 inhibitor deficiency leads to uncontrolled plasma kallikrein activation and excess bradykinin production, triggering sudden severe swelling in the skin, gut, and airway — laryngeal attacks can be immediately fatal. Berotralstat directly inhibits plasma kallikrein to prevent attacks before they start; it is the only once-daily oral HAE prophylactic approved in the United States, and BioCryst is pursuing label extension to children aged 2 to 11.
No primer in glossary yet.
This event was extracted from a primary disclosure. The full chain of citations is in the disclosure trail below.
- NDAregulatoryNew Drug Application
FDA submission for approval of a small-molecule drug. Triggers the PDUFA clock.
Competitive landscape
| Drug | Company | Modality | Mechanism | Phase | Next catalyst |
|---|---|---|---|---|---|
| NTLA-2002 | NTLA | gene therapy | in vivo CRISPR knockout of KLKB1 | ENROLLMENT · Sep 26 | |
| DUPIXENTdupilumab | REGN | mAb | IL-4Rα blocker | READOUT · Jan 27 | |
| batoclimab | IMVT | mAb | anti-FcRn mAb | CONFERENCE · Apr 26 |
Disclosure trail
- Apr 19, 2026·11d agopinned · highest confidenceMED confPRMONTHtop claimJUN2026
“Following positive Phase 3 APeX-P pediatric data, BioCryst plans to submit the supplemental NDA for ORLADEYO in patients aged 2 to 11 in June 2026.”
conf 84%via llm