BioCryst is a rare disease biotech almost entirely focused on ORLADEYO (berotralstat), an oral once-daily capsule for preventing hereditary angioedema (HAE) attacks. HAE is a rare genetic disorder causing potentially fatal swelling episodes, and ORLADEYO — the first oral targeted prophylactic for HAE — works by inhibiting plasma kallikrein, the protein that drives the swelling response. BioCryst sells ORLADEYO primarily in the U.S. through a specialty pharmacy model, marketing directly to HAE specialists via a focused sales force. The U.S. now represents over 90% of ORLADEYO revenue following the 2025 sale of BioCryst's European operations to Neopharmed Gentili. Revenue scales with net patient adds, the proportion of commercially reimbursed patients, and gross-to-net dynamics; BioCryst has reached roughly 3,000 of approximately 11,000 diagnosed U.S. HAE patients, and targets ORLADEYO peak revenue of ~$1B by 2029. BioCryst also earns modest revenue from RAPIVAB, an IV influenza antiviral, though this is non-core. The company's key pipeline asset is navenibart, an injectable monoclonal antibody for HAE prophylaxis acquired through the January 2026 merger with Astria Therapeutics, currently in Phase 3 with a potential approval in late 2028. Navenibart is designed to target the injectable prophylaxis segment — dosing just two to four times per year — complementing ORLADEYO's oral positioning. BioCryst's broader strategy is to layer additional rare disease products onto its existing commercial infrastructure, targeting assets near proof-of-concept that can be commercialized with minimal incremental cost.