Article · Wikipedia archive · Last revised Jun 27, 2026

Berotralstat

Berotralstat, sold under the brand name Orladeyo, is a medication used to prevent attacks of hereditary angioedema. Berotralstat is a plasma kallikrein inhibitor. It is taken by mouth.

Last revised
Jun 27, 2026
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≈ 3 min
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Citations
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Source
Wikipedia ↗
Berotralstat
Clinical data
Trade namesOrladeyo
Other namesBCX7353, BCX-7353
AHFS/Drugs.comMonograph
License data
Routes of
administration		By mouth
Drug classPlasma kallikrein inhibitor
ATC code
Legal status
Legal status
Identifiers
  • 2-[3-(aminomethyl)phenyl]-N-[5-[(R)-(3-cyanophenyl)-(cyclopropylmethylamino)methyl]-2-fluorophenyl]-5-(trifluoromethyl)pyrazole-3-carboxamide
CAS Number
PubChem CID
DrugBank
ChemSpider
UNII
KEGG
CompTox Dashboard (EPA)
Chemical and physical data
FormulaC30H26F4N6O
Molar mass562.573 g·mol−1
3D model (JSmol)
  • NCC1=CC(=CC=C1)N1N=C(C=C1C(=O)NC1=CC(=CC=C1F)[C@H](NCC1CC1)C1=CC=CC(=C1)C#N)C(F)(F)F
  • InChI=1S/C30H26F4N6O/c31-24-10-9-22(28(37-17-18-7-8-18)21-5-1-3-19(11-21)15-35)13-25(24)38-29(41)26-14-27(30(32,33)34)39-40(26)23-6-2-4-20(12-23)16-36/h1-6,9-14,18,28,37H,7-8,16-17,36H2,(H,38,41)/t28-/m1/s1
  • Key:UXNXMBYCBRBRFD-MUUNZHRXSA-N

Berotralstat, sold under the brand name Orladeyo, is a medication used to prevent attacks of hereditary angioedema.35784 Berotralstat is a plasma kallikrein inhibitor.3 It is taken by mouth.3

The most common side effects include abdominal pain, vomiting, diarrhea, back pain, and heartburn.4

Berotralstat was approved for medical use in the United States in December 2020,349 and in the European Union in April 2021.5

Medical uses

Berotralstat is indicated for prophylaxis to prevent attacks of hereditary angioedema.310

History

Berotralstat was approved based on evidence from one clinical trial (Trial 1 / NCT03485911) of 120 participants with hereditary angioedema.4 The trial was conducted at 40 sites in the United States, the European Union, and Canada.4 Trial investigators evaluated participants twelve years of age and older11 with hereditary angioedema for eight weeks to determine the number of attacks for each participant.4 The trial enrolled only participants who had at least two attacks during the eight-week period.4 Participants were assigned to receive one of two doses of berotralstat or placebo once every day for 24 weeks.4 Neither the participants nor the investigators knew which treatment was being given until after the trial was completed.4 All participants could use other medications for treatment of attacks.4

Society and culture

Berotralstat was approved for medical use in the United States in December 2020,4 and in the European Union in April 2021.5

Names

Berotralstat is the international nonproprietary name.12

References

References

  1. "Summary Basis of Decision - Orladeyo". Health Canada. 23 October 2014. Retrieved 28 November 2022.
  2. Product monograph hres.ca
  3. "Orladeyo- berotralstat hydrochloride capsule". DailyMed. Retrieved 25 December 2020.
  4. "Drug Trials Snapshot: Orladeyo". U.S. Food and Drug Administration. 3 December 2020. Archived from the original on 18 December 2020. Retrieved 25 December 2020. This article incorporates text from this source, which is in the public domain.
  5. "Orladeyo EPAR". European Medicines Agency (EMA). 24 February 2021. Retrieved 12 July 2021.
  6. "Orladeyo Product information". Union Register of medicinal products. Retrieved 3 March 2023.
  7. Hwang JR, Hwang G, Johri A, Craig T (December 2019). "Oral plasma kallikrein inhibitor BCX7353 for treatment of hereditary angioedema". Immunotherapy. 11 (17): 1439–1444. doi:10.2217/imt-2019-0128. PMID 31635497.
  8. Zuraw B, Lumry WR, Johnston DT, Aygören-Pürsün E, Banerji A, Bernstein JA, et al. (October 2020). "Oral once-daily berotralstat for the prevention of hereditary angioedema attacks: A randomized, double-blind, placebo-controlled phase 3 trial". The Journal of Allergy and Clinical Immunology. 148 (1): 164–172.e9. doi:10.1016/j.jaci.2020.10.015. PMID 33098856.
  9. "Orladeyo: FDA-Approved Drugs". U.S. Food and Drug Administration (FDA). Retrieved 25 December 2020.
  10. "BioCryst Announces FDA Approval of ORLADEYO® (berotralstat) Oral Pellets, First and Only Oral Prophylactic Treatment for Patients with HAE Aged 2 to <12 Years". BioCryst Pharmaceuticals. 12 December 2025. Retrieved 13 December 2025 – via GlobeNewswire.
  11. "Berotralstat (Oral Route) Side Effects - Mayo Clinic". www.mayoclinic.org. Retrieved 3 March 2021.
  12. "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 82". WHO Drug Information. 33 (3). 2019. hdl:10665/330879.
External links
  • Clinical trial number NCT03485911 for "Efficacy and Safety Study of BCX7353 as an Oral Treatment for the Prevention of Attacks in HAE (APeX-2)" at ClinicalTrials.gov