BioCryst Presents New Real-World Evidence Showing Reduced Angioedema-Related Medical Visits After Switching to ORLADEYO® (berotralstat) at ACAAI 2026 Annual Scientific Meeting

Rhea-AI Summary

BioCryst Pharmaceuticals (BCRX) announced two real-world studies showing reduced healthcare use after patients with hereditary angioedema initiated or switched to ORLADEYO.

Among 78 patients switching from another long-term preventive therapy, angioedema-related visits fell 30% per patient-year; outpatient/emergency department visits also fell 30%, both statistically significant. Hospitalizations fell to 0.12 per patient-year, but that reduction was not statistically significant. In a separate study, 128 patients starting ORLADEYO had 53% fewer all-cause hospitalizations and 61% fewer angioedema-related emergency department visits. No statistically significant differences in the magnitude of reductions were observed between ORLADEYO and lanadelumab or subcutaneous plasma-derived C1-inhibitor. Both studies were retrospective and are being presented at the ACAAI 2026 Annual Scientific Meeting.

Positive

• Minor pointORLADEYO switchers had 30% fewer angioedema-related visits per patient-year, a statistically significant reduction (p=0.039).
• Minor pointAngioedema-related outpatient/emergency department visits fell 30% per patient-year after switching, statistically significant (p=0.040).
• Minor pointPatients initiating ORLADEYO had 53% fewer all-cause hospitalizations than before treatment.
• Minor pointPatients initiating ORLADEYO had 61% fewer angioedema-related emergency department visits than before treatment.

Negative

• Minor pointSwitchers’ angioedema-related hospitalizations fell to 0.12 per patient-year, but the reduction was not statistically significant.
• Minor pointORLADEYO showed no statistically significant difference in reduction magnitude versus lanadelumab or subcutaneous plasma-derived C1-inhibitor.
• Minor pointBoth studies used retrospective pre-post designs, comparing healthcare use before and after treatment.

Key Figures

Reduction in overall angioedema-related visits: 30% per-patient-year (p=0.039) Reduction in outpatient/emergency department visits: 30% per-patient-year (p=0.040) Post-index hospitalization rate: 0.12 per-patient-year +5 more

Reduction in overall angioedema-related visits

30% per-patient-year (p=0.039)

After switching to ORLADEYO; statistically significant

Reduction in outpatient/emergency department visits

30% per-patient-year (p=0.040)

After switching to ORLADEYO; statistically significant

Post-index hospitalization rate

0.12 per-patient-year

After switching to ORLADEYO; rate ratio 0.54 [95% CI: 0.21–1.39], non-significant

Switching-study sample size

78 patients

Patients with HAE who switched from another long-term prophylactic therapy

All-cause hospitalization reductions

-26% to -53%

Across the treatment-naïve cohorts; SC-pdC1-INH to ORLADEYO

All-cause emergency department visit reductions

-34% to -48%

Across the treatment-naïve cohorts; SC-pdC1-INH to lanadelumab

Angioedema-related hospitalization reductions

-46% to -59%

Across the treatment-naïve cohorts; SC-pdC1-INH to lanadelumab

Angioedema-related emergency department visit reductions

-43% to -61%

Across the treatment-naïve cohorts; SC-pdC1-INH to ORLADEYO

Historical Context

1 past event · Latest: Oct 29

1 event

1. Oct 29

   Data presentation preview

   24h Move

   +1.8%

   Previewed ACAAI posters on ORLADEYO healthcare use, including after switching therapies.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Key Terms

hereditary angioedema, plasma kallikrein, rate ratio, QTc interval prolongation, +1 more

5 terms

hereditary angioedema medical

"patients with hereditary angioedema (HAE) aged 2 and older"

A rare inherited disorder that causes sudden, painful swelling under the skin or in internal tissues, including the airway, because a natural blood‑control protein is missing or not working. Attacks can be unpredictable and sometimes life‑threatening, so people often need ongoing medication or emergency treatment. For investors, hereditary angioedema represents a niche but stable market for specialized therapies, diagnostics, and emergency care solutions.

plasma kallikrein medical

"decreasing the activity of plasma kallikrein"

Plasma kallikrein is an enzyme in the blood that acts like a pair of molecular scissors, cutting a larger protein to release bradykinin, a small molecule that widens blood vessels and promotes swelling and fluid movement. Its activity helps regulate inflammation, blood pressure and clotting pathways, so drugs that block or modify it can change disease symptoms and safety profiles—making it an important therapeutic target and risk factor for investors watching drug development or regulatory news.

rate ratio technical

"rate ratio [95% CI]: 0.54 [0.21–1.39]"

Rate ratio is a single number that compares how often an event happens in one group versus another by dividing one group’s event rate by the other’s, often taking the time people were observed into account. Think of it like comparing how many cars pass two toll booths per hour: a rate ratio of 2 means one booth sees twice the traffic. Investors use it to judge the size of treatment effects or safety risks and to compare outcomes across studies, which can affect regulatory outlooks and the value of related products.

QTc interval prolongation medical

"potential for QTc interval prolongation"

QTc interval prolongation is a lengthening of the heart’s electrical “reset” time measured on an electrocardiogram after adjusting for heart rate; think of it like a loading bar that takes longer than normal to return to zero. It matters to investors because pronounced prolongation can signal a risk of dangerous irregular heartbeats, trigger regulatory review, clinical-trial changes, safety warnings or market setbacks for drugs and medical devices, and therefore can affect a company’s valuation and timelines.

P-glycoprotein medical

"Berotralstat is a substrate of P-glycoprotein (P-gp)"

P-glycoprotein is a protein in cell membranes that works like a molecular bouncer, actively pumping certain drugs and chemicals out of cells and controlling how much of a medicine reaches organs such as the brain and gut. For investors, P-glycoprotein matters because it can reduce a drug's absorption, cause interactions with other medicines, or make cancers resistant to treatment, all of which affect a drug's effectiveness, dosing, safety profile and regulatory or commercial prospects.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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—Two real-world studies demonstrate reductions in healthcare resource utilization in HAE patients who initiated or switched to ORLADEYO^® from another prophylactic therapy—

RESEARCH TRIANGLE PARK, N.C., Nov. 13, 2026 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced new real-world evidence on the use of once-daily ORLADEYO^® (berotralstat), the first and only targeted oral prophylactic therapy for patients with hereditary angioedema (HAE) aged 2 and older, showing reductions in hospitalizations and emergency department visits following ORLADEYO initiation in patients new to long-term prophylactic therapy (LTP) and in patients switching to ORLADEYO from another LTP.

This practice-informing research will be featured across two poster presentations during the American College of Allergy, Asthma & Immunology (ACAAI) 2026 Annual Scientific Meeting taking place in Phoenix, Arizona, November 12-16. 

“These real-world studies add to our understanding of healthcare resource utilization among patients with HAE who either initiate ORLADEYO or switch from another long-term prophylactic therapy,” said Sandra Christiansen, MD, professor of medicine and director of translational research at the US HAEA Angioedema Center at the University of California, San Diego. “Patients initiating long-term prophylaxis were found to have comparable reductions in hospitalizations and emergency department visits for ORLADEYO, lanadelumab and subcutaneous plasma-derived C1-inhibitor. Patients who switched to ORLADEYO from an alternative first-line long-term prophylactic therapy experienced a decrease in their angioedema-related medical visits. Taken together, these findings support ORLADEYO’s clinical and economic benefits as a long-term prophylactic therapy for HAE.”  

A poster titled Reductions in Healthcare Utilization Among Individuals with HAE Who Switched to Berotralstat from Another Prophylaxis (Poster #R096) details findings from a retrospective pre-post study that featured analysis of administrative claims data of patients with HAE in the United States linked with proprietary data from the ORLADEYO patient base through BioCryst’s sole source specialty pharmacy. The analysis focused on individuals aged ≥12 years with HAE who switched from another LTP to ORLADEYO who had a baseline of at least six months of continuous insurance plan eligibility prior to starting ORLADEYO (n=78).

Patients with HAE who switched from another LTP to ORLADEYO experienced significant reductions in angioedema-related healthcare resource utilization. All statistical tests were two-sided, with an alpha level of 0.05. Results with p<0.05 were considered statistically significant:

• Overall angioedema-related visits decreased significantly by 30% per-patient-year (PPY) (p=0.039, sig)
• Rates of angioedema-related outpatient/emergency department visits decreased significantly by 30% PPY (p=0.040, sig)
• Rates of angioedema-related hospitalizations decreased to 0.12 PPY post-index (rate ratio [95% CI]: 0.54 [0.21–1.39], non-significant)
  

This poster will be on display in the expo hall of the Phoenix Convention Center and will be presented on Saturday, November 14, 11:30-11:45 a.m. MT.

A second poster titled Hospitalization and Emergency Department Reductions After Initiating Berotralstat, Lanadelumab, or Subcutaneous Plasma-Derived C1-Inhibitor for Hereditary Angioedema (Poster #R077) reported findings from a retrospective observational pre-post cohort study of all-cause and angioedema-related hospitalization and emergency department visits. The assessment focused on LTP treatment naïve patients aged ≥12 years who initiated treatment with ORLADEYO (n=128), lanadelumab (n=236) or subcutaneous plasma-derived C1-inhibitor (SC-pdC1-INH) (n=95) between June 2017 and October 2024 with ≥6 months of continuous insurance enrollment and evidence of HAE prior to LTP treatment initiation.

All cohorts experienced statistically significant reductions in emergency department visits and reductions were also observed in hospitalizations:

• Reductions in all-cause hospitalization ranged from -26% (SC-pdC1-INH cohort) to -53% (ORLADEYO cohort)
• Reductions in all-cause emergency department visits ranged from -34% (SC-pdC1-INH cohort) to -48% (lanadelumab cohort)
• Reductions in angioedema-related hospitalization ranged from -46% (SC-pdC1-INH cohort) to -59% (lanadelumab cohort)
• Reductions in angioedema-related emergency department visits ranged from -43% (SC-pdC1-INH cohort) to -61% (ORLADEYO cohort)
• No statistically significant differences in magnitude of reductions were observed between ORLADEYO and lanadelumab or SC-pdC1-INH
  

This poster will be on display in the expo hall of the Phoenix Convention Center and will be presented on Friday, November 13, 4:20-4:35 p.m. MT.

In addition to these real-world studies, BioCryst will also present new clinical data from its HAE portfolio designed to support diverse patient needs and preferences at the ACAAI 2026 Annual Scientific Meeting, including:

• Growth-Related Berotralstat Dose Adjustment in Children with Hereditary Angioedema: 48-Week Dosing Experience from APeX-P; Poster #R097; Monitor 15; Saturday, November 14, 11:45 a.m.-12:00 p.m. MT 
• Long-Term Safety, Efficacy, and Quality of Life with Navenibart in Hereditary Angioedema: ALPHA-SOLAR Interim Results; Poster #R110; Monitor 13; Sunday, November 15, 12:00-12:15 p.m. MT 

Visit www.ORLADEYO.com for more information. 

About ORLADEYO^® (berotralstat)
ORLADEYO^® (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 2 years and older. One dose of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein.

U.S. Indication and Important Safety Information

INDICATION
ORLADEYO^® (berotralstat) is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 2 years and older.

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 the treatment of acute HAE attacks. Additional doses or doses of ORLADEYO higher than the prescribed once-daily dose are not recommended due to the potential for QTc interval prolongation.

IMPORTANT SAFETY INFORMATION
An increase in QTc interval was observed in adults at dosages higher than 150 mg once daily and was concentration dependent.

The most common adverse reactions (≥10%) in patients receiving ORLADEYO were abdominal pain, vomiting, diarrhea, back pain and gastroesophageal reflux disease.

In adult and pediatric patients aged 12 years and older with moderate or severe hepatic impairment (Child-Pugh B or C), the recommended dosage of ORLADEYO capsules is 110 mg once daily with food. In pediatric patients aged 2 to <12 years with moderate or severe hepatic impairment (Child-Pugh B or C), avoid use of ORLADEYO.

Berotralstat is a substrate of P-glycoprotein (P-gp) and breast cancer resistance protein. P-gp inducers may decrease berotralstat plasma concentration, leading to reduced efficacy of ORLADEYO. Avoid concomitant use of P-gp inducers with ORLADEYO.

ORLADEYO at a dose of 150 mg is a moderate inhibitor of CYP2D6 and CYP3A4. Concomitant use of ORLADEYO with CYP2D6 or CYP3A4 substrates can increase exposure of the CYP2D6 or CYP3A4 substrates and may increase the risk of adverse reactions associated with the substrates. If ORLADEYO is concomitantly used with CYP2D6 or CYP3A4 substrates where minimal increases in the concentration of the substrates may lead to serious adverse reactions, closely monitor or modify the dosage of the CYP2D6 or CYP3A4 substrate.

The safety and effectiveness of ORLADEYO in pediatric patients <2 years of age have not been established.

There are insufficient data available to inform drug-related risks with ORLADEYO use in pregnancy. There are no data on the presence of berotralstat in human milk, its effects on the breastfed infant, or its effects on milk production.

To report SUSPECTED ADVERSE REACTIONS, contact BioCryst Pharmaceuticals, Inc. at 1-833-633-2279 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

About navenibart
Navenibart is an investigational YTE-modified monoclonal antibody inhibitor of plasma kallikrein, an established and safe mechanism, currently being evaluated in clinical trials for long-term prevention of HAE attacks with potential best-in-class dosing every 3 or 6 months. 

About BioCryst Pharmaceuticals 
BioCryst is a global biotechnology company focused on developing and commercializing medicines for hereditary angioedema (“HAE”) and other rare diseases, driven by its deep commitment to improving the lives of people living with these conditions. BioCryst has commercialized ORLADEYO^® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor and is advancing a pipeline of potential first-in-class or best-in-class therapeutics for rare diseases. For more information, please visit www.biocryst.com or follow us on LinkedIn.

Forward-Looking Statements
This press release contains forward-looking statements, including statements regarding the clinical and economic benefits of ORLADEYO and the development, potential dosing profile, competitive positioning, and expectations for navenibart. These statements involve known and unknown risks, uncertainties and other factors which may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: risks related to the development and interpretation of real-world data; BioCryst’s ability to successfully implement or maintain its commercialization plans for ORLADEYO; BioCryst’s ability to successfully progress its development plans for navenibart; the outcome of preclinical testing and early clinical trials may not be predictive of the success of later clinical trials, and interim results of a clinical trial do not necessarily predict final results; ongoing and future clinical development of product candidates, including navenibart, may take longer than expected and may not have positive results; BioCryst may not advance human clinical trials with product candidates, including navenibart, as expected; the FDA or other applicable regulatory agencies may require additional studies beyond the studies planned for products and product candidates, may not provide regulatory clearances which may result in delay of planned clinical trials, may not review regulatory filings on our expected timeline, may impose certain restrictions, warnings, or other requirements on products and product candidates, may impose a clinical hold with respect to product candidates, or may withhold, delay or withdraw market approval for products and product candidates, may ultimately determine that there are deficiencies in the development program or execution thereof, may require additional information or studies, may disagree with our safety and efficacy conclusions, or may impose certain restrictions, warnings, or other requirements; product candidates, if approved, may not achieve market acceptance; and BioCryst’s ability to successfully commercialize its products and product candidates. This list is not exclusive. To see a more comprehensive set of risks, please refer to the documents BioCryst files periodically with the Securities and Exchange Commission, specifically BioCryst’s most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K, which identify important factors that could cause actual results to differ materially from those contained in BioCryst’s forward-looking statements.

Contact:

Investors:
investorrelations@biocryst.com

Media:
media@biocryst.com

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

What did BioCryst’s study show after patients switched to ORLADEYO?

Among 78 patients switching from another long-term preventive therapy, angioedema-related medical visits declined 30% per patient-year. Outpatient/emergency department visits also declined 30%; both reductions were statistically significant. Angioedema-related hospitalizations decreased to 0.12 per patient-year, but that reduction was not statistically significant.

How did ORLADEYO compare with other preventive treatments in BioCryst’s real-world study?

There were no statistically significant differences in the magnitude of reductions between ORLADEYO and lanadelumab or subcutaneous plasma-derived C1-inhibitor. Patients initiating ORLADEYO had 53% fewer all-cause hospitalizations and 61% fewer angioedema-related emergency department visits compared with before treatment.

Which patients were included in BioCryst’s ORLADEYO healthcare-use studies?

Both studies included patients with hereditary angioedema aged 12 years and older with at least six months of continuous insurance eligibility or enrollment before treatment initiation. The switching study linked administrative claims with specialty-pharmacy data. The treatment-naïve study included patients initiating ORLADEYO, lanadelumab or subcutaneous plasma-derived C1-inhibitor between June 2017 and October 2024.