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Important Safety InformationPrescribing Information
About HAE

About HAE

  
  
  

HAE is chronic, unpredictable, and life limiting1

Characterized by recurrent angioedema attacks, hereditary angioedema (HAE) is associated with considerable morbidity. While HAE attacks can be debilitating, laryngeal attacks are life-threatening and often occur without warning.1

Diagnosing suspected HAE

The journey to diagnosis is long for patients in the United States. A 2016 study showed that nearly half of all patients with HAE reported prior misdiagnoses.2

1

If a patient presents with angioedema, manage airways and anaphylaxis risk per local protocols3

For angioedema that does not respond to epinephrine, corticosteroids, or antihistamines, proceed with suspicion of bradykinin-mediated angioedema3

Female patient displaying HAE symptoms
2

Discuss personal history of recurrent angioedema and family
history of HAE4

3

Assess for key characteristics of bradykinin-mediated angioedema3

Clock
Timing of attack onset
  • Gradual worsening over several hours3
  • Lasts 3 to 5 days3
Symptoms
Symptom presentation
  • Pain rather than itching3,4
  • Abdominal and cutaneous swelling and pain3,4
  • Erythema marginatum
    (a nonpruritic rash pathognomonic of HAE)3,4
Treatment response
Treatment response
  • No response to epinephrine, corticosteroids, or antihistamines3
4

To confirm clinical suspicion, continue with diagnostic workup for bradykinin-mediated angioedema to determine if patient has HAE type 1, HAE type 2, or HAE due to normal C1 esterase inhibitor (HAE-nl-C1INH)4,5

5

Upon confirmation of HAE (ICD-10 code D84.1), testing of first-degree relatives is strongly encouraged4-6

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Download HAE diagnostic resource

For more information, download this diagnostic resource

For more information on diagnosing HAE, visit this site

Research shows a majority of patients with HAE prefer oral prophylaxis8

HAE consensus guidelines state that the overall goals of HAE treatment are to reduce morbidity and mortality, and to restore a normal quality of life to patients5

In the life of a patient with HAE, burden can take many forms

HAE disease burden

HAE consensus guidelines state that the overall goals of HAE treatment are to reduce morbidity and mortality, and to restore a normal quality of life to patients4

Each patient has unique needs and goals—prophylactic therapy should be determined based on individual considerations.

Disease-related factors (physical, emotional, quality of life)1
+
Treatment-related factors (route of administration, side effects, availability/supply)1,4
+
Patient preference (lifestyle, dosing schedule, flexibility)1,7

Shared decision making empowers patients to play a more active role in their own care choices and be more adherent with their treatment, leading to better overall outcomes10

Research shows a majority of patients with HAE prefer oral prophylaxis9

According to an October 2023 survey of 150 adults with HAE9,a:

  • Efficacy is the most important treatment attribute when choosing a prophylactic therapy
  • Where efficacy is comparable, most people prefer an oral once-daily prophylactic option even with less frequent subcutaneous dosing available

In a 2020b study of 75 adults living with HAE, approximately 90% say they have learned to tolerate difficult aspects of treatment and approximately 75% say they try not to think about the demanding nature of their treatment despite a positive view regarding the impact of their prophylaxis.10

a98% of respondents were receiving treatment for HAE at the time of the survey and experienced >1 attack per month.9

b100% of respondents in the study were on injectable, intravenous, or androgen prophylaxis.10

Top reasons for oral treatment preference7

Avoid unpleasantness of using needles

Ease of use and convenience

Easy to carry and travel with

Enhances and normalizes quality of life

Faster administration

References: 1. Banerji A. The burden of illness in patients with hereditary angioedema. Ann Allergy Asthma Immunol. 2013;111(5):329-336. doi:10.1016/j.anai.2013.08.019 2. Zanichelli A, Longhurst HJ, Maurer M, et al; for IOS Study Group. Misdiagnosis trends in patients with hereditary angioedema from the real-world clinical setting. Ann Allergy Asthma Immunol. 2016;117(4):394-398. doi:10.1016/ j.anai.2016.08.01 3. Bernstein JA, Cremonesi P, Hoffmann TK, Hollingsworth J. Angioedema in the emergency department: a practical guide to differential diagnosis and management. Int J Emerg Med. 2017;10(1):15. doi:10.1186/s12245-017-0141-z 4. Busse PJ, Christiansen SC, Riedl MA, et al. US HAEA Medical Advisory Board 2020 Guidelines for the Management of Hereditary Angioedema. J Allergy Clin Immunol Pract. 2021;9(1):132-150.e3. doi:10.1016/j.jaip.2020.08.046 5. Maurer M, Magerl M, Betschel S, et al. The international WAO/EAACI guideline for the management of hereditary angioedema: the 2021 revision and update. Allergy. 2022;77(7):1961-1990. doi:10.1111/all.15214
6. 2024 ICD-10-CM Diagnosis Code D84.1. ICD10Data.com website. Updated October 1, 2023. Accessed October 25, 2023. https://www.icd10data.com/ICD10CM/Codes/D50-D89/D80-D89/D84-/D84.1 7. Banerji A, Davis KH, Brown MT, et al. Patient-reported burden of hereditary angioedema: findings from a patient survey in the United States. Ann Allergy Asthma Immunol. 2020;124(6):600-607. doi:10.1016/j.anai.2020.02.018 8. Aygören-Pürsün E, Bygum A, Beusterien K, et al. Socioeconomic burden of hereditary angioedema: results from the hereditary angioedema burden of illness study in Europe. Orphanet J Rare Dis. 2014;9:99. doi:1186/1750-1172-9-99 9. Data on file, BioCryst Pharmaceuticals Inc. 10. Radojicic C, Riedl MA, Craig TJ, et al. Patient perspectives on the treatment burden of injectable medication for hereditary angioedema. Allergy Asthma Proc. 2021;42(3):S4-S10. doi:10.2500/aap.2021.42.210025

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 12 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 dosages of ORLADEYO higher than 150 mg once daily are not recommended due to the potential for QT prolongation.

IMPORTANT SAFETY INFORMATION

An increase in QT prolongation was observed at dosages higher than the recommended 150 mg once-daily dosage and was concentration dependent.

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

A reduced dosage of 110 mg taken orally once daily with food is recommended in patients with moderate or severe hepatic impairment (Child-Pugh B or C).

Berotralstat is a substrate of P-glycoprotein (P-gp) and breast cancer resistance protein. P-gp inducers (eg, rifampin, St. John‘s wort) may decrease berotralstat plasma concentration, leading to reduced efficacy of ORLADEYO. The use of P-gp inducers is not recommended with ORLADEYO.

ORLADEYO at a dose of 150 mg is a moderate inhibitor of CYP2D6 and CYP3A4. For concomitant medications with a narrow therapeutic index that are predominantly metabolized by CYP2D6 or CYP3A4, appropriate monitoring and dose titration is recommended. ORLADEYO at a dose of 300 mg is a P-gp inhibitor. Appropriate monitoring and dose titration is recommended for P-gp substrates (eg, digoxin) when coadministering with ORLADEYO.

The safety and effectiveness of ORLADEYO in pediatric patients <12 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.

Please see the full Prescribing Information.