Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Berotralstat · 2 trials · 3 indications
AUC0-last is the area under the plasma concentration-time curve from time 0 to the time of the last measurable concentration.
AUC0-6 is the area under the plasma concentration-time curve from time 0 to 6 hours.
Ctrough is the concentration at the end of a dosing interval of berotralstat.
Cmax is the maximum observed plasma concentration of berotralstat.
Tlast is the time of the last measurable concentration (Clast) of berotralstat collected over the sampling interval.
Tmax is the time taken to reach the maximum observed plasma concentration of berotralstat.
| Arm | Type | Description |
|---|---|---|
| Cohort 1: ≥ 40 kg body weight (Berotralstat 150 mg) | EXPERIMENTAL | Participants 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) | EXPERIMENTAL | Participants 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) | EXPERIMENTAL | Participants 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) | EXPERIMENTAL | Participants 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 daily | EXPERIMENTAL | Berotralstat (BCX7353) capsules or granules orally administered once daily. |
| Name | Type | Description |
|---|---|---|
| Berotralstat | DRUG | Administered orally once daily |
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...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Intellia Therapeutics, Inc. | NTLA | 3 | PHASE3 | NTLA-2002, Normal Saline Administration |
| BioCryst Pharmaceuticals, Inc. | BCRX | 2 | PHASE3 | Berotralstat |
| Ionis Pharmaceuticals, Inc. | IONS | 1 | PHASE3 | Donidalorsen |
| Pharvaris N.V. | PHVS | 1 | PHASE2 | deucrictibant |
| BioMarin Pharmaceutical Inc. | BMRN | 1 | PHASE1 | Dose 1 of BMN 331 |
| Astria Therapeutics, Inc. | ATXS | 1 | PHASE2 | STAR-0215 |
Berotralstat is an investigational small molecule being developed for the treatment of Hereditary Angioedema (HAE), a rare genetic condition characterized by recurrent episodes of severe swelling. It is currently in Phase 3 clinical development and has not yet been approved by regulatory authorities.
Berotralstat targets and inhibits the KLKB1 protein, which plays a role in the kallikrein-kinin system. By inhibiting KLKB1, Berotralstat aims to reduce the production of bradykinin, a key mediator of swelling attacks in Hereditary Angioedema. This mechanism is being evaluated in clinical trials.
Berotralstat is being developed by BioCryst Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol BCRX. The company is conducting Phase 3 clinical trials to evaluate the drug's safety and efficacy in patients with Hereditary Angioedema.
Berotralstat is currently in Phase 3 clinical development for Hereditary Angioedema. It is an investigational drug, meaning it has not yet received regulatory approval and is still being studied in clinical trials to assess its safety and effectiveness in patients.
Berotralstat is being studied in two Phase 3 clinical trials. NCT04933721 is an open-label access study for HAE patients previously enrolled in Berotralstat studies, enrolling 139 participants. NCT05453968 is a study of Berotralstat treatment in children with Hereditary Angioedema, enrolling 29 pediatric participants.
Yes, Berotralstat is being studied in pediatric patients with Hereditary Angioedema. The clinical trial NCT05453968, titled 'Berotralstat Treatment in Children With Hereditary Angioedema,' is actively enrolling children as young as 2 years old across multiple countries, including Austria, Canada, France, Germany, and the United Kingdom.