Companies: BioCryst Pharmaceuticals, Inc.
Drugs: ORLADEYO, berotralstat
BioCryst’s New ORLADEYO Data Adds Real-World HAE Evidence
100% citation coverage2 regulatory sources
Intelligence Snapshot
Executive Summary
BioCryst announced new clinical data and real-world evidence for ORLADEYO (berotralstat) in hereditary angioedema.
Key Insights
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ORLADEYO is a plasma kallikrein inhibitor indicated for prophylaxis to prevent HAE…
ORLADEYO is a plasma kallikrein inhibitor indicated for prophylaxis to prevent HAE attacks in adults and pediatric patients 2 years of age and older.
-
A Phase 3 pediatric trial (NCT05453968) is active but not recruiting, and a Phase 3…
A Phase 3 pediatric trial (NCT05453968) is active but not recruiting, and a Phase 3 open-label access study (NCT04933721) is enrolling by invitation.
Market Impact
| Regulatory | medium |
|---|---|
| Commercial | medium |
| Competitive | high |
| Investment | medium |
BioCryst Pharmaceuticals is presenting new clinical data and real-world evidence for ORLADEYO (berotralstat) in hereditary angioedema. The update matters because it expands the evidence base around an FDA-approved oral prophylactic option for adults and pediatric patients 2 years and older.
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Quick Answer
Key Questions
- What is ORLADEYO (berotralstat) used to treat?
- What changed in BioCryst's ORLADEYO announcement?
- What clinical trials are currently active for berotralstat?
- Is ORLADEYO FDA approved?
- Are there any safety concerns with ORLADEYO dosing?
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BioCryst's New ORLADEYO Data Adds Real-World HAE Evidence
Key Takeaways
- BioCryst announced new clinical data and real-world evidence for ORLADEYO (berotralstat) in hereditary angioedema.
- ORLADEYO is a plasma kallikrein inhibitor indicated for prophylaxis to prevent HAE attacks in adults and pediatric patients 2 years of age and older.
- A Phase 3 pediatric trial (NCT05453968) is active but not recruiting, and a Phase 3 open-label access study (NCT04933721) is enrolling by invitation.
IntelligenceRegulatory Impact
FDA and EMA decisions frame this story. Regulatory relevance is medium for HAE, with ORLADEYO and berotralstat most exposed. Track designations, submission types, and label or guidance shifts that could move timelines.
BioCryst Announces New Clinical Data and Real-World Evidence for ORLADEYO
BioCryst Pharmaceuticals announced new clinical data and real-world evidence for ORLADEYO (berotralstat) in hereditary angioedema. The company is presenting findings across multiple data sources to document outcomes in patients using the therapy.
IntelligenceCompetitive Intelligence
Competitive pressure is high. BioCryst Pharmaceuticals, Inc. reshape positioning, formulary leverage, and partnership options. Benchmark pipeline differentiation and regional market access assumptions against this development.
Regulatory Context and Product Scope
ORLADEYO is indicated for prophylaxis to prevent attacks of hereditary angioedema in adults and pediatric patients 2 years of age and older. The drug is a plasma kallikrein inhibitor.
Important limitations apply to its use. ORLADEYO should not be used for treatment of acute HAE attacks. Additionally, doses of ORLADEYO higher than the prescribed once-daily dose are not recommended due to the potential for QTc interval prolongation. These constraints define the therapeutic window and differentiate ORLADEYO as a preventive rather than acute-rescue therapy.
IntelligenceMarket Signals
Commercial pull is medium and investment relevance medium for HAE. Expect implications for pricing, access, and launch sequencing.
Clinical Trial Pipeline
NCT05453968 is a Phase 3 study of berotralstat treatment in children with hereditary angioedema, currently active but not recruiting. NCT04933721 is a Phase 3 open-label access study providing berotralstat to patients previously enrolled in berotralstat studies and is currently enrolling by invitation.
BioCryst has also completed earlier-stage trials in the berotralstat program. NCT03485911 was a Phase 3 efficacy and safety study of BCX7353 (berotralstat's prior designation) as an oral treatment for the prevention of HAE attacks. NCT03472040 was a long-term safety study in hereditary angioedema. Both trials have completed enrollment and data collection.
IntelligenceStrategic Takeaways
BioCryst announced new clinical data and real-world evidence for ORLADEYO (berotralstat) in hereditary angioedema. ORLADEYO is a plasma kallikrein inhibitor indicated for prophylaxis to prevent HAE attacks in adults and pediatric patients 2 years of age and older. A Phase 3 pediatric trial (NCT05453968) is active but not recruiting, and a Phase 3 open-label access study (NCT04933721) is enrolling by invitation.
What Analysts and BD Teams Should Track
For investors and business development professionals monitoring ORLADEYO, several milestones warrant attention. The pediatric Phase 3 trial NCT05453968 remains active, and its eventual completion could provide additional safety and efficacy data in children ages 2 and older. The open-label access study NCT04933721 continues enrolling qualified patients by invitation. BioCryst may also release additional analyses or presentations of the newly announced clinical and real-world data at medical conferences or through peer-reviewed publications.
The evidence package announced by BioCryst adds to the clinical foundation for the approved indication. Analysts should monitor how the company positions these findings relative to other HAE prophylaxis options and whether the data inform prescriber or payer discussions around ORLADEYO's role in the HAE treatment landscape.
IntelligenceEvidence Quality
Grounded in 2 regulatory sources.
Drug Snapshot
| Drug | berotralstat |
|---|---|
| Generic name | BEROTRALSTAT HYDROCHLORIDE |
| Manufacturer | BioCryst Pharmaceuticals, Inc. |
| Route | ORAL |
| Indication | 1 INDICATIONS AND USAGE ORLADEYO ® is indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 2 years of age and older. ORLADEYO is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 2 years and older. ( 1 ) Limitations of Use : ORLADEYO should not be used for treatment of acute HAE attacks. ( 1 ) Limitations of Use : The safety and effectiveness of ORLADEYO for the treatment of acute HAE attacks have not been established. ORLADEYO should not be used for |
Regulatory Summary
- Approved indication: 1 INDICATIONS AND USAGE ORLADEYO ® is indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 2 years of age and older. ORLADEYO is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 2 years and older. ( 1 ) Limitations of Use : ORLADEYO should not be used for treatment of acute HAE attacks. ( 1 ) Limitations of Use : The safety and effectiveness of ORLADEYO for the treatment of acute HAE attacks have not been established. ORLADEYO should not be used for
- BioCryst Pharmaceuticals, Inc. develops berotralstat
Trial Snapshot
| Trial | Title | Status | Phase | Sponsor |
|---|---|---|---|---|
| NCT05453968 | Berotralstat Treatment in Children With Hereditary Angioedema | ACTIVE_NOT_RECRUITING | PHASE3 | BioCryst Pharmaceuticals |
| NCT04933721 | Open-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat Studies | ENROLLING_BY_INVITATION | PHASE3 | BioCryst Pharmaceuticals |
| NCT05453968 | Berotralstat Treatment in Children With Hereditary Angioedema | ACTIVE_NOT_RECRUITING | PHASE3 | BioCryst Pharmaceuticals |
| NCT04933721 | Open-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat Studies | ENROLLING_BY_INVITATION | PHASE3 | BioCryst Pharmaceuticals |
| NCT03873116 | Study to Evaluate the Efficacy and Safety of BCX7353 as an Oral Treatment for the Prevention of HAE Attacks in Japan | COMPLETED | PHASE3 | BioCryst Pharmaceuticals |
Competitor Matrix
| Company / Program | Indication | Active trials |
|---|---|---|
| CSL Behring | HAE | 3 |
| KalVista Pharmaceuticals, Ltd. | HAE | 2 |
| Astria Therapeutics, Inc. | HAE | 2 |
| Octapharma | HAE | 1 |
Timeline
- Active_Not_Recruiting trial NCT05453968 (PHASE3)
- Enrolling_By_Invitation trial NCT04933721 (PHASE3)
- Active_Not_Recruiting trial NCT05453968 (PHASE3)
- Enrolling_By_Invitation trial NCT04933721 (PHASE3)
Frequently Asked Questions
What is ORLADEYO (berotralstat) used to treat?
ORLADEYO is indicated for prophylaxis to prevent attacks of hereditary angioedema in adults and pediatric patients 2 years of age and older. It is a plasma kallikrein inhibitor. ORLADEYO should not be used for treatment of acute HAE attacks.
What changed in BioCryst's ORLADEYO announcement?
BioCryst announced new clinical data and real-world evidence for ORLADEYO in hereditary angioedema. The company is presenting findings from multiple data sources to document outcomes in patients using the therapy for prophylaxis.
What clinical trials are currently active for berotralstat?
NCT05453968 is a Phase 3 pediatric study of berotralstat in children with hereditary angioedema, currently active but not recruiting. NCT04933721 is a Phase 3 open-label access study for patients previously enrolled in berotralstat trials and is currently enrolling by invitation.
Is ORLADEYO FDA approved?
Yes. ORLADEYO (berotralstat) is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema in adults and pediatric patients 2 years of age and older.
Are there any safety concerns with ORLADEYO dosing?
Doses of ORLADEYO higher than the prescribed once-daily dose are not recommended due to the potential for QTc interval prolongation.
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- Sources analyzed
- 2
- Evidence strength
- 96/100
- Last verified
- Jun 13, 2026
- AI-assisted review
- Yes
- Editorial review
- Dr. Sarah Chen
Critical source quality · grounded in cited primary and secondary sources.
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