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Orladeyo (BEROTRALSTAT)
Orladeyo works by blocking the plasma kallikrein enzyme, which is involved in the production of bradykinin, a key mediator of HAE symptoms.
At a glance
| Generic name | BEROTRALSTAT |
|---|---|
| Sponsor | Biocryst |
| Drug class | Plasma Kallikrein Inhibitor |
| Target | Plasma kallikrein |
| Modality | Small molecule |
| Therapeutic area | Neuroscience |
| Phase | FDA-approved |
| First approval | 2020 |
Mechanism of action
Berotralstat is plasma kallikrein inhibitor that binds to plasma kallikrein and inhibits its proteolytic activity. Plasma kallikrein is protease that cleaves high-molecular-weight-kininogen (HMWK) to generate cleaved HMWK (cHMWK) and bradykinin, potent vasodilator that increases vascular permeability resulting in swelling and pain associated with HAE. In patients with HAE due to C1-inhibitor (C1-INH) deficiency or dysfunction, normal regulation of plasma kallikrein activity is not present, which leads to uncontrolled increases in plasma kallikrein activity and results in angioedema attacks. Berotralstat decreases plasma kallikrein activity to control excess bradykinin generation in patients with HAE.
Approved indications
- Hereditary angioneurotic edema
Common side effects
- Abdominal Pain
- Vomiting
- Diarrhea
- Back Pain
- Gastroesophageal Reflux Disease
- Headache
- Fatigue
- Flatulence
- Gastrointestinal reactions
Key clinical trials
- Berotralstat Treatment in Children With Hereditary Angioedema (PHASE3)
- Open-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat Studies (PHASE3)
- Study to Evaluate the Efficacy and Safety of BCX7353 as an Oral Treatment for the Prevention of HAE Attacks in Japan (PHASE3)
- Efficacy and Safety Study of BCX7353 as an Oral Treatment for the Prevention of Attacks in HAE (PHASE3)
- A Long Term Safety Study of BCX7353 in Hereditary Angioedema (PHASE2,PHASE3)
- Study of BCX7353 as a Treatment for Attacks of Hereditary Angioedema (PHASE2)
- Efficacy and Safety of BCX7353 to Prevent Angioedema Attacks in Subjects With Hereditary Angioedema (PHASE2)
- Oral Berotralstat Expanded Access Program
Patents
| Patent | Expiry | Type |
|---|---|---|
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Orladeyo CI brief — competitive landscape report
- Orladeyo updates RSS · CI watch RSS
- Biocryst portfolio CI