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Berotralstat

Phase 3

Hereditary Angioedema | Small molecule | Other |BioCryst Pharmaceuticals, Inc.|Last Updated: Feb 25, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
CONTROLLEDDMC
Total Trials2
Total Enrollment168
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05453968Berotralstat Treatment in Children With Hereditary AngioedemaPHASE3 ACTIVE NOT_RECRUITING 29 — — Oct 25, 2022Feb 1, 2027Feb 25, 202615 Austria, Canada +8
NCT04933721Open-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat StudiesPHASE3 ENROLLING BY_INVITATION 139 — — Jul 23, 2021Aug 1, 2031Dec 23, 202516 Canada, Czechia +9
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Study Endpoints
Primary Endpoints
Area Under the Plasma Concentration-Time Curve From Time 0 to the Last Measurable Concentration (AUC0-last) of Berotralstat
Week 2

AUC0-last is the area under the plasma concentration-time curve from time 0 to the time of the last measurable concentration.

Area Under the Plasma Concentration-Time Curve From Time 0 to 6 Hours Post-dose (AUC0-6) of Berotralstat
Predose and up to 6 hours post dose at Week 2

AUC0-6 is the area under the plasma concentration-time curve from time 0 to 6 hours.

Concentration at the End of the Dosing Interval (Ctrough) of Berotralstat
Predose at Week 2

Ctrough is the concentration at the end of a dosing interval of berotralstat.

Maximum Observed Plasma Concentration (Cmax) of Berotralstat
Predose and up to 6 hours post dose at Week 2

Cmax is the maximum observed plasma concentration of berotralstat.

Time of Last Measurable Plasma Concentration (Tlast) of Berotralstat
Predose and up to 6 hours post dose at Week 2

Tlast is the time of the last measurable concentration (Clast) of berotralstat collected over the sampling interval.

Time to Maximum Plasma Concentration (Tmax) of Berotralstat
Predose and up to 6 hours post dose at Week 2

Tmax is the time taken to reach the maximum observed plasma concentration of berotralstat.

Number and proportion of subjects with a treatment-related TEAE
240 weeks
Number and proportion of subjects who experience a serious adverse event (SAE)
240 weeks
Number and proportion of subjects who experience a treatment-related Grade 3 or 4 TEAE
240 weeks
Number and proportion of subjects who experience a treatment-related Grade 3 or 4 treatment-emergent chemistry abnormality
240 weeks
Number and proportion of subjects who discontinue due to a TEAE
240 weeks
Secondary Endpoints
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
From first dose of study treatment up to approximately 73 weeks
Number of Adjusted Hereditary Angioedema (HAE) Attacks
Week 1 through Week 12 and Week 1 through Week 48
Rate of Adjusted HAE Attacks
Week 1 through Week 12 and Week 1 through Week 48
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposePREVENTION
Treatment Arms
ArmTypeDescription
Cohort 1: ≥ 40 kg body weight (Berotralstat 150 mg)EXPERIMENTALParticipants received 150 milligram (mg) berotralstat capsule orally once daily for up to 144 weeks. Dose modifications were permitted due to weight changes, PK results, or safety.
Cohort 2: 32 to < 40 kg body weight (Berotralstat 108 mg)EXPERIMENTALParticipants received 108 mg berotralstat granules orally once daily for up to 144 weeks. Dose modifications were permitted due to weight changes, PK results, or safety.
Cohort 3: 24 to < 32 kg body weight (Berotralstat 96 mg)EXPERIMENTALParticipants received 96 mg berotralstat granules orally once daily for up to 144 weeks. Dose modifications were permitted due to weight changes, PK results, or safety.
Cohort 4: 12 to <24 kg body weight (Berotralstat 78 mg)EXPERIMENTALParticipants received 78 mg berotralstat granules orally once daily for up to 144 weeks. Dose modifications were permitted due to weight changes, PK results, or safety.
BCX7353 capsules or granules once dailyEXPERIMENTALBerotralstat (BCX7353) capsules or granules orally administered once daily.
Interventions
NameTypeDescription
BerotralstatDRUGAdministered orally once daily
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Eligibility Criteria
Age Range2 Years — 11 Years
SexALL
Healthy VolunteersNo
Study Sites15

Inclusion Criteria: * Male and non-pregnant, non-lactating females 2 to \< 12 years of age * Body weight ≥ 12 kg * Clinical diagnosis of HAE * In the opinion of the investigator, the participant would benefit from long term oral HAE prophylaxis * For subjects who are not currently receiving prophyl...

Countries:AustriaCanadaFranceGermanyIsraelItalyPolandRomaniaSpainUnited KingdomCzechiaNorth MacedoniaSlovakiaSouth AfricaSouth Korea
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Competitive Landscape -Hereditary Angioedema 10 trials
CompanyTickerTrialsLead PhaseDrugs
Intellia Therapeutics, Inc.NTLA3PHASE3NTLA-2002, Normal Saline Administration, Biological NTLA-2002
BioCryst Pharmaceuticals, Inc.BCRX2PHASE3Berotralstat, berotralstat
Ionis Pharmaceuticals, Inc.IONS1PHASE3Donidalorsen
KalVista Pharmaceuticals, Inc.KALV1PHASE3KVD900, Drug: KVD900
Pharvaris N.V.PHVS1PHASE2deucrictibant
BioMarin Pharmaceutical Inc.BMRN1PHASE1Dose 1 of BMN 331
Astria Therapeutics, Inc.ATXS1PHASE2STAR-0215
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT04933721primaryCompletionDate: changed
LOWMay 26, 2026NCT05453968primaryCompletionDate: changed
LOWMay 24, 2026NCT04933721studyFirstPostDate: changed
LOWMay 24, 2026NCT05453968studyFirstPostDate: changed