Drugs / avoralstat
Regulatory milestones approvals, filings & regulatory actions · 1 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Clinical hold | US (FDA) | — | angioedema, hereditary, 4 | 2013-07-31 | “the clinical hold placed on BCX4161 in November 2012” biocryst.com ↗ |
Trials 5
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 13 trials | ||||||
| Phase 1 | NCT07228559 | Oct 2025 → Apr 2026 | diabetic macular edema | BioCryst Pharmaceuticals | Terminated | No outcome recorded Stop: Business |
| Phase 1 | NCT02218294 | Aug → Sep 2014 | hereditary angioedema | BioCryst Pharmaceuticals | Completed | No outcome recorded |
| Phase 1 | NCT02125162 | Apr → Jul 2014 | hereditary angioedema | BioCryst Pharmaceuticals | Completed | No outcome recorded |
| Phase 2/31 trial · 1 missed | ||||||
| Phase 2/3 | NCT02303626 | Dec 2014 → Jan 2016 | hereditary angioedema | BioCryst Pharmaceuticals | Completed | Missed primary |
| Phase 31 trial | ||||||
| Phase 3 | NCT02670720 | Dec 2015 → Feb 2016 | hereditary angioedema | BioCryst Pharmaceuticals | Terminated | No outcome recorded |
| Comparator or background therapy1 trial | ||||||
| Phase 2 | NCT01984788 Comparator | Nov 2013 → May 2014 | hereditary angioedema | BioCryst Pharmaceuticals | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 9
| Date | Issuer | Release |
|---|---|---|
| 2025-05-05 | BioCryst Pharmaceuticals | Regulatory BioCryst Reports First Quarter 2025 Financial Results and Provides Business Update biocryst.com ↗
The first clinical trial with suprachoroidal delivery of avoralstat, the company’s investigational plasma kallikrein inhibitor for the treatment of diabetic macular edema (DME), has been granted authorization to proceed in Australia. |
| 2017-10-09 | BioCryst Pharmaceuticals | Regulatory FDA Grants Fast Track Designation for BCX4161 for the Treatment of Hereditary Angioedema biocryst.com ↗
BioCryst Pharmaceuticals, Inc. , (Nasdaq:BCRX) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for BCX4161 , a novel, orally administered, selective inhibitor of plasma kallikrein in advanced clinical development for the treatment of hereditary angioedema. |
| 2017-10-09 | BioCryst Pharmaceuticals | Results BioCryst Successfully Completes Its Phase 1 Clinical Trial of BCX4161 for the Treatment of Hereditary Angioedema biocryst.com ↗
BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today announced that the randomized, placebo-controlled, Phase 1 clinical trial of orally-administered BCX4161 in healthy volunteers successfully met all of its objectives. |
| 2017-10-09 | BioCryst Pharmaceuticals | Regulatory BioCryst Receives Positive Opinion on European Orphan Drug Designation for BCX4161 for the Treatment of Hereditary Angioedema biocryst.com ↗
BioCryst Pharmaceuticals, Inc. , (Nasdaq:BCRX) today announced that the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) issued a positive opinion on the application for orphan drug designation for BCX4161 for the treatment of patients with hereditary angioedema (HAE). |
| 2017-10-09 | BioCryst Pharmaceuticals | Regulatory BioCryst Receives Orphan Drug Designation for BCX4161 for the Treatment of Hereditary Angioedema biocryst.com ↗
BioCryst Pharmaceuticals, Inc. , (Nasdaq:BCRX) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to BCX4161 for the prevention of acute attacks of angioedema in patients with hereditary angioedema (HAE). |
| 2017-10-09 | BioCryst Pharmaceuticals | Results BioCryst Presents BCX4161 Phase 1 Trial and Kallikrein Inhibition Assay Results biocryst.com ↗
BCX4161 was generally safe and well tolerated in the Phase 1 clinical trial, and there were no serious or dose limiting adverse events reported. |
| 2017-10-09 | BioCryst Pharmaceuticals | Results BioCryst Announces Results From OPuS-2 biocryst.com ↗
Treatment with 500 mg and 300 mg of avoralstat three times daily failed to demonstrate a statistically significantly lower mean attack rate versus placebo. |
| 2017-10-09 | BioCryst Pharmaceuticals | Results BioCryst Announces Positive Results From OPuS-1, a Phase 2 Trial of BCX4161 for the Prophylactic Treatment of Hereditary Angioedema biocryst.com ↗
Treatment with BCX4161 demonstrated a statistically significant mean attack rate reduction of 0.45 attacks per week versus placebo, p < 0.001. |
| 2017-10-09 | BioCryst Pharmaceuticals | Results BioCryst Announces Late Breaker Presentation of OPuS-1 Phase 2 Trial Results at the 23rd EADV Congress biocryst.com ↗
The trial met the primary efficacy endpoint and all other objectives established for the trial. |
All press releases naming this drug 15 releases
Evidence & citations 6 cited values
Every value below carries the sentence it was read from. 7 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | avoralstat | ClinicalTrials.gov intervention name — accepted as the source's own label NCT02670720 ↗ |
| Known as | BCX4161 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT01984788 ↗ |
| Action | Inhibit | “Plasma kallikrein inhibitor” CHEMBL4297502 ↗ |
| Modality | Small molecule | — CHEMBL4297502 ↗ |
| Route | Oral | “daily oral prophylactic treatment” NCT01984788 ↗ |
| Target | KLKB1 | “Plasma kallikrein inhibitor” CHEMBL4297502 ↗ |