- Trial Phase
- Approved
- Time to Market
- Already approved and commercialized (Dec 2020 adult capsules; Dec 2025 pediatric oral pellets)
- Why 4/10
- ORLADEYO is already approved and marketed (first approved Dec 2020, pediatric pellets Dec 2025). No imminent PDUFA or pivotal readout for this drug; the next hard catalysts are quarterly commercial updates (Q2 earnings early Aug 2026) and pipeline readouts for navenibart (BCX10013), not ORLADEYO itself. As an approved asset with no upcoming binary decision, impactScore is capped at 4-5.
ORLADEYO is the sole commercial driver of BCRX stock. Watch quarterly ORLADEYO sales growth trajectory (2026 guidance $625M-$645M), pediatric pellet launch uptake, and competitive dynamics vs. Takhzyro and new injectable entrants. The company achieved its first annual profit in 2025, a significant de-risking milestone. The next major pipeline catalyst for the stock overall is the Phase 3 ALPHA-ORBIT navenibart readout, which could expand the HAE franchise.
Price Chart
Executive Summary
ORLADEYO (berotralstat) is BioCryst Pharmaceuticals' flagship product and the only FDA-approved once-daily oral prophylactic for hereditary angioedema (HAE). First approved in December 2020 for patients 12+, it generated $601.8M in net revenue in 2025 (+38% YoY) and drove the company to its first full-year profit. A pediatric oral pellet formulation was approved in December 2025, expanding the addressable market. The drug faces competition from Takeda's injectable Takhzyro (more efficacious but less convenient) and newer injectable entrants, but ORLADEYO's oral convenience and growing payer adoption support continued share gains. The company guides for $625M-$645M in 2026 ORLADEYO revenue.
How It Works
ORLADEYO is a once-daily pill that prevents swelling attacks in people with hereditary angioedema (HAE), a rare genetic disorder. It works by blocking plasma kallikrein, an enzyme that is overactive in HAE patients. By inhibiting this enzyme, the drug reduces production of bradykinin — a protein that causes blood vessels to leak fluid, leading to painful swelling in the face, throat, limbs, and gut. It is the only FDA-approved oral once-daily preventive treatment for HAE.
Trial Status
ORLADEYO (berotralstat) was first FDA-approved in December 2020 for prophylaxis of hereditary angioedema (HAE) attacks in patients 12 years and older (capsule form). In December 2025, the FDA approved a new oral pellet formulation for pediatric patients aged 2 to <12 years old, based on data showing sustained attack reduction. BioCryst presented 48-week pediatric follow-up data at EAACI 2026 confirming sustained efficacy.
The Two-Sided Case
Upcoming Catalysts
- Q2 2026 earnings report (early August 2026) — key for ORLADEYO sales trajectory and pediatric pellet launch update
- Phase 3 ALPHA-ORBIT trial of navenibart (factor D inhibitor) — enrollment completed June 2026; topline readout could be a major pipeline catalyst for BCRX overall
- Potential European expansion/partnering updates for ORLADEYO after the sale of European rights to Neopharmed Gentili
Source Filing
The SecBot filing or press-release report that triggered this drug report:
BIOCRYST PHARMACEUTICALS INC 8-K BCRX ·Originating report