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NCT04933721Phase 3ENROLLING BY INVITATION

Open-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat Studies

Study Summary

This is a phase 3b open-label study providing access to berotralstat for HAE patients who were previously enrolled in berotralstat studies.

Conditions
Hereditary AngioedemaHAE
Interventions / Drugs
berotralstat
View on ClinicalTrials.gov ↗
Sponsor
BCRX
BioCryst Pharmaceuticals
Rare Disease
D37/100
Study Details
Enrollment139 participants
Primary Completion—
Lead SponsorBioCryst Pharmaceuticals
Data Retrieved2026-04-11 04:07:07.352051+00:00
What This Trial Means for Biotech Investors
Trial Phase Context

Phase 3 trials are pivotal studies with large patient populations (hundreds to thousands) designed to prove efficacy and safety for FDA submission. A successful Phase 3 readout is typically the last major hurdle before a New Drug Application (NDA) or BLA filing.

Status & Expected Data

Current status: ENROLLING BY INVITATION.

Enrolled: 139 participants
Sponsor Catalyst Profile

BioCryst Pharmaceuticals (BCRX) is the sponsoring company for this trial. BiotechSign currently grades this company D (37/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

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