berotralstat

also known as BCX7353 · BCX-7353 DIHYDROCHLORIDE · BCX7353.2HCl · Berotralstat dihydrochloride · BEROTRALSTAT HYDROCHLORIDE · Orladeyo

Small molecule targets KLKB1 via inhibition

Developed for: hereditary angioedema
Investigated by: BioCryst Pharmaceuticals

Regulatory milestones approvals, filings & regulatory actions · 5 recorded

Milestone	Jurisdiction	Brand	Indication	Date	Sentence it was read from
Filed angioedema, hereditary, 4 3 milestones
Filed	Japan (PMDA)	—	angioedema, hereditary, 4	2020-02-03	“submitted a new drug application (JNDA) to the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) for approval of oral, once daily berotralstat” biocryst.com ↗
Filed	US (FDA)	—	angioedema, hereditary, 4	2019-12-11	“FDA has accepted and filed its new drug application (NDA) for the approval of oral, once daily berotralstat (BCX7353) for the prevention of hereditary angioedema (HAE) attacks.” biocryst.com ↗ “submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for approval of oral, once daily berotralstat (BCX7353)” biocryst.com ↗ “New drug application (NDA) submitted to U.S Food and Drug Administration in December 2019” biocryst.com ↗
Filed	EU (EMA)	—	angioedema, hereditary, 4	—	“EMA has validated its marketing authorization application (MAA) submission for approval of oral, once-daily berotralstat (BCX7353)” biocryst.com ↗
Approved Indication not stated 2 milestones
Approved	EU (EMA)	Orladeyo	—	2021-04-30	europa.eu ↗
Approved	US (FDA)	ORLADEYO	—	2020-12-03	fda.gov ↗

Trials 11

today

US approved 2020

EU approved 2021

NCT05453968 · Ph 3 NCT04933721 · Ph 3 NCT03873116 · Ph 3 NCT03472040 · Ph 2/3 NCT03485911 · Ph 3 NCT03240133 · Ph 2 NCT03202784 · Ph 1 NCT03136237 · Ph 1 NCT02870972 · Ph 2 NCT02819102 · Ph 1 NCT02448264 · Ph 1

201520162017201820192020202120222023202420252026202720282029203020312032

Phase	Registry id	Dates	Indication	Sponsor	Status	Outcome
Phase 14 trials · 1 met primary
Phase 1	NCT03202784	Feb → Aug 2017	hereditary angioedema	BioCryst Pharmaceuticals	Completed	No outcome recorded
Phase 1	NCT03136237	Feb → Aug 2017	hereditary angioedema	BioCryst Pharmaceuticals	Completed	No outcome recorded
Phase 1	NCT02819102	Mar → Jun 2016	hereditary angioedema	BioCryst Pharmaceuticals	Completed	No outcome recorded
Phase 1	NCT02448264	May → Nov 2015	hereditary angioedema	BioCryst Pharmaceuticals	Completed	Met primary
Phase 22 trials
Phase 2	NCT03240133	Jul 2017 → Jan 2019	hereditary angioedema	BioCryst Pharmaceuticals	Completed	Mixed
Phase 2	NCT02870972	Aug 2016 → Aug 2017	hereditary angioedema	BioCryst Pharmaceuticals	Completed	Mixed
Phase 2/31 trial
Phase 2/3	NCT03472040	Feb 2018 → Apr 2022	hereditary angioedema	BioCryst Pharmaceuticals	Completed	No outcome recorded
Phase 34 trials · 1 met primary
Phase 3	NCT05453968	Oct 2022 → Sep 2024 overdue	hereditary angioedema	BioCryst Pharmaceuticals	Active not recruiting	No outcome recorded
Phase 3	NCT04933721	Jul 2021 → Aug 2031 expected	hereditary angioedema	BioCryst Pharmaceuticals	Enrolling by invitation	No outcome recorded
Phase 3	NCT03873116	Dec 2018 → Jul 2021	hereditary angioedema	BioCryst Pharmaceuticals	Completed	No outcome recorded
Phase 3	NCT03485911	Feb 2018 → Apr 2019	hereditary angioedema	BioCryst Pharmaceuticals	Completed	Met primary
Comparator or background therapy1 trial
—	NCT04428632 Background	—	hereditary angioedema	BioCryst Pharmaceuticals	Approved for marketing	No outcome recorded

News releases announcing trial results or a regulatory action · 82

Date	Issuer	Release
2026-08-25	BioCryst Pharmaceuticals	Regulatory BioCryst Announces Approval of ORLADEYO® for Pediatric HAE Patients in Japan biocryst.com ↗ BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that Japan’s Ministry of Health, Labor and Welfare (MHLW) has granted marketing approval for once-daily ORLADEYO ® (berotralstat) for prophylactic therapy in pediatric patients with hereditary angioedema (HAE) aged 2 to <12 years.
2026-06-12	BioCryst Pharmaceuticals	Results BioCryst Presents New Clinical Data and Real-World Evidence Demonstrating Impact of HAE Portfolio at the 2026 European Academy of Allergy and Clinical Immunology Annual Meeting biocryst.com ↗ today announced new clinical data and real-world evidence for ORLADEYO ® (berotralstat)
2026-02-11	BioCryst Pharmaceuticals	Results BioCryst to Present New HAE Data from ORLADEYO® (berotralstat) and Navenibart at the 2026 American Academy of Allergy, Asthma & Immunology Annual Meeting biocryst.com ↗ Oral Berotralstat Reduces the Rate of Moderate and Severe Attacks
2025-12-12	BioCryst Pharmaceuticals	Regulatory BioCryst Announces FDA Approval of ORLADEYO® (berotralstat) Oral Pellets, First and Only Oral Prophylactic Treatment for Patients with HAE Aged 2 to biocryst.com ↗
2025-11-06	BioCryst Pharmaceuticals	Results BioCryst Presents New Data at ACAAI Highlighting Burden of HAE on Pediatric Patients and Caregivers and Sustained Reduction of Attacks in Children Treated with One Year of ORLADEYO® (berotralstat) biocryst.com ↗
2025-06-16	BioCryst Pharmaceuticals	Results BioCryst Presents New Data on the Long-term Efficacy and Safety of ORLADEYO® (berotralstat) Across all Ages at EAACI biocryst.com ↗ These data from both clinical trials and real-world settings continue to strengthen the evidence that ORLADEYO is an effective and well-tolerated long-term prophylactic treatment for HAE.
2025-06-13	BioCryst Pharmaceuticals	Regulatory ORLADEYO® (berotralstat) Approved in Colombia biocryst.com ↗ BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the National Institute of Drug and Food Surveillance (INVIMA) in Colombia has granted approval for oral, once-daily ORLADEYO ® (berotralstat) for the prophylaxis of hereditary angioedema (HAE) attacks in adults and pediatric patients 12 years of age or older.
2025-06-06	BioCryst Pharmaceuticals	Regulatory BioCryst Achieves Reimbursement for ORLADEYO Across all Major European Countries biocryst.com ↗ This reimbursement approval in the Netherlands marks national reimbursement for ORLADEYO across all major European countries, providing HAE patients with access to the first oral, once-daily preventive therapy.
2025-05-30	BioCryst Pharmaceuticals	Results BioCryst Highlights Real-world Data Showing ORLADEYO® (berotralstat) Consistently Reduces HAE Attacks Across All Ages biocryst.com ↗ New data shared at the 14th C1-inhibitor Deficiency & Angioedema Workshop supports berotralstat use at all stages of life
2025-05-16	BioCryst Pharmaceuticals	Results BioCryst Presents New Real-world Evidence Showing Significant and Sustained Reductions in HAE Attack Rates in Adolescents and People with Severe HAE Following Initiation of ORLADEYO® (berotralstat) biocryst.com ↗ These two groups experienced far fewer attacks per month compared to baseline after starting ORLADEYO.
2025-05-14	BioCryst Pharmaceuticals	Regulatory BioCryst Announces FDA Acceptance of NDA for ORLADEYO® (berotralstat) Oral Granules in Patients with Hereditary Angioedema Aged 2 to 11 Years biocryst.com ↗
2025-02-24	BioCryst Pharmaceuticals	Results BioCryst Announces Positive Results from APeX-P Trial for ORLADEYO® (berotralstat) in Pediatric Patients with Hereditary Angioedema Aged 2 to biocryst.com ↗ BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced positive results from an interim analysis of the ongoing APeX-P clinical trial evaluating an oral granule formulation of once-daily ORLADEYO ® (berotralstat) in pediatric patients with hereditary angioedema (HAE) aged 2 to <12 years.
2025-02-12	BioCryst Pharmaceuticals	Regulatory BioCryst Launches ORLADEYO® (berotralstat) in Portugal biocryst.com ↗ BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that Infarmed in Portugal has recommended ORLADEYO ® (berotralstat) for the routine prevention of recurrent attacks of hereditary angioedema (HAE) in eligible patients 12 years and older.
2024-10-24	BioCryst Pharmaceuticals	Results BioCryst Presents New Real-World Evidence Showing High Adherence and Persistence Rates with ORLADEYO® (berotralstat) biocryst.com ↗
2024-10-14	BioCryst Pharmaceuticals	Results BioCryst to Present New Real-World Evidence Showing Significant Reductions in Medical Visits and Hospitalizations After Starting ORLADEYO® (berotralstat) biocryst.com ↗ Here, our analysis reveals that significant reductions in healthcare resource utilization are achieved across multiple outcomes – such as reductions in hospitalizations and medical visits, including those related to HAE attacks.
2024-09-06	BioCryst Pharmaceuticals	Results BioCryst Presents New ORLADEYO® (berotralstat) Data at 7th Bradykinin Symposium biocryst.com ↗ Non-serious gastrointestinal adverse events were reported in 12.5 percent of patients.
2024-07-09	BioCryst Pharmaceuticals	Regulatory ORLADEYO® (berotralstat) Approved in Peru biocryst.com ↗ BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the General Directorate of Medicines, Supplies and Drugs (DIGEMID) in Peru has granted approval for oral, once-daily ORLADEYO ® (berotralstat) for the prophylaxis of hereditary angioedema (HAE) attacks in adults and pediatric patients 12 years of age or older.
2024-06-02	BioCryst Pharmaceuticals	Results BioCryst Presents New Real-world Evidence Showing Reductions in Attack Rates in HAE Patients with Normal C1-Inhibitor after Beginning ORLADEYO® (berotralstat) Treatment biocryst.com ↗ BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced new real-world evidence showing that patients with hereditary angioedema (HAE) who have normal C1-inhibitor (HAE-nC1-INH) level and function had a reduction in monthly attack rates after starting oral, once-daily ORLADEYO ® (berotralstat).
2024-05-13	BioCryst Pharmaceuticals	Regulatory ORLADEYO® (berotralstat) Approved in Mexico biocryst.com ↗ With this decision by COFEPRIS, ORLADEYO is now approved in four countries in the pan-Latin America region.
2024-05-09	BioCryst Pharmaceuticals	Results BioCryst Presents New Real-world Evidence Showing Significant Reductions in Healthcare Resource Utilization Among Patients with HAE Following Initiation of ORLADEYO® (berotralstat) biocryst.com ↗ The data presented here represent the first documentation that prescribing ORLADEYO significantly reduces healthcare resource utilization.
2024-04-17	BioCryst Pharmaceuticals	Regulatory BioCryst Announces Approval of ORLADEYO® (berotralstat) by the Brazilian Health Regulatory Agency biocryst.com ↗ We are excited to announce that ORLADEYO is now approved in the region’s largest market, following the positive regulatory decisions we received in Chile and Argentina last year.
2024-02-23	BioCryst Pharmaceuticals	Results BioCryst Presents New Real-world Data Showing Rapid, Substantial and Sustained HAE Attack Rate Reductions After Beginning ORLADEYO® (berotralstat) Treatment biocryst.com ↗
2024-02-19	BioCryst Pharmaceuticals	Regulatory BioCryst Launches ORLADEYO® (berotralstat) in Italy biocryst.com ↗ BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the Italian Medicines Agency, Agenzia Italiana del Farmaco (AIFA), has finalized reimbursement and recommended ORLADEYO ® (berotralstat) for the routine prevention of recurrent attacks of hereditary angioedema (HAE) in eligible patients 12 years and older.
2023-12-19	BioCryst Pharmaceuticals	Results BioCryst Announces Publication of Data from Open-label Extension of the APeX-2 Pivotal Trial of ORLADEYO® (berotralstat) biocryst.com ↗ As detailed in this manuscript, long-term prophylaxis with ORLADEYO has enabled patients with HAE to better manage their condition, including reducing the number of HAE attacks they experience and demonstrating clinically meaningful improvement in their quality of life.
2023-11-29	BioCryst Pharmaceuticals	Regulatory BioCryst Announces Approval of ORLADEYO® (berotralstat) by the National Administration of Drugs, Foods, and Medical Devices (ANMAT) in Argentina biocryst.com ↗
2023-11-21	BioCryst Pharmaceuticals	Regulatory BioCryst Launches ORLADEYO® (berotralstat) in Spain biocryst.com ↗ BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the Spanish Ministry for Health (Ministerio de Sanidad) has granted marketing authorization for oral, once-daily ORLADEYO ® (berotralstat) for the routine prevention of recurrent hereditary angioedema (HAE) attacks in HAE patients 12 years and older.
2023-11-10	BioCryst Pharmaceuticals	Results BioCryst Presents New Real-world Data Showing Reduced Attack Rates in Patients with HAE with Normal C1-inhibitor Following Long-term Treatment with ORLADEYO® (berotralstat) biocryst.com ↗ The company also announced a new post-hoc analysis from the APeX-S clinical trial that showed a sustained reduction in HAE attacks compared to patients’ self-reported baseline attack rates.
2023-07-17	BioCryst Pharmaceuticals	Results BioCryst to Participate in the 2023 US HAEA National Summit biocryst.com ↗ Once-daily oral berotralstat led to improvements in work productivity and activity impairment in patients with hereditary angioedema: Results from the APeX-2 randomized Phase III trial
2023-05-22	BioCryst Pharmaceuticals	Regulatory BioCryst Announces Approval of ORLADEYO® (berotralstat) by the Public Health Institute of Chile biocryst.com ↗ BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the Public Health Institute (ISP) of Chile has granted marketing authorization for oral, once-daily ORLADEYO ® (berotralstat) for the prophylaxis of hereditary angioedema (HAE) attacks in patients 12 years of age or older.
2023-04-27	BioCryst Pharmaceuticals	Results BioCryst Presents New Long-term Data Demonstrating Consistently High Attack-free Status Among Hereditary Angioedema Patients with ORLADEYO® (berotralstat) biocryst.com ↗ Long-term prophylaxis with ORLADEYO continues to help patients achieve consistently high attack-free days regardless of gender, age and prior HAE therapy after 96 weeks of treatment
2023-02-24	BioCryst Pharmaceuticals	Results BioCryst Presents New Long-term and Real-world Data Demonstrating Sustained Reductions in Hereditary Angioedema Attack Rates and Improvement in Quality of Life with ORLADEYO® (berotralstat) biocryst.com ↗ The final results from the long-term APeX-S study show that ORLADEYO was consistently well-tolerated, with no new safety signals observed, and attack reduction was sustained through 96 weeks.
2022-11-28	BioCryst Pharmaceuticals	Regulatory BioCryst Announces Approval of ORLADEYO® (berotralstat) by the Israeli Ministry of Health biocryst.com ↗ BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the Israeli Ministry of Health has granted marketing authorization for oral, once-daily ORLADEYO ® (berotralstat) to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years of age and older in Israel.
2022-11-10	BioCryst Pharmaceuticals	Results BioCryst Presents Real-World Data Showing Rapid and Sustained HAE Attack Rate Reduction After Beginning ORLADEYO® (berotralstat), Regardless of Prior Prophylactic Therapy biocryst.com ↗ “These data are particularly exciting because, consistent with our long-term clinical program data, they show that patients on ORLADEYO sustain, and even improve, attack control the longer they are on therapy,” said Dr. Ryan Arnold, chief medical officer of BioCryst.
2022-08-18	BioCryst Pharmaceuticals	Regulatory BioCryst Announces Approval of ORLADEYO® (berotralstat) in Saudi Arabia biocryst.com ↗
2022-07-01	BioCryst Pharmaceuticals	Results BioCryst Presents New Data Demonstrating Consistently Low Attack Rates Among HAE Patients After Switching to ORLADEYO® (berotralstat) biocryst.com ↗ Analysis from APeX-S showed 98 percent attack-free days during treatment with ORLADEYO after switching from an injectable therapy
2022-06-07	BioCryst Pharmaceuticals	Regulatory BioCryst Announces Approval of ORLADEYO® (berotralstat) by Swissmedic biocryst.com ↗ BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that Swissmedic has granted marketing authorization for oral, once-daily ORLADEYO ® (berotralstat) for the routine prevention of recurrent hereditary angioedema (HAE) attacks in patients 12 years and older in Switzerland.
2022-06-06	BioCryst Pharmaceuticals	Regulatory BioCryst Announces Health Canada has Authorized ORLADEYO® (berotralstat), the Only Oral Treatment for the Prevention of Hereditary Angioedema Attacks biocryst.com ↗ BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that Health Canada has approved oral, once-daily ORLADEYO ® (berotralstat) for the routine prevention of recurrent hereditary angioedema (HAE) attacks in patients 12 years and older.
2022-02-24	BioCryst Pharmaceuticals	Results BioCryst Presents New Data Demonstrating Sustained Reductions in Attack Rates and Improvement in Quality of Life Among HAE Patients Following Long-Term Treatment with ORLADEYO® (berotralstat) biocryst.com ↗ The 96-week data from APeX-2 showcase the durable, long-term efficacy of ORLADEYO and build on the strong reductions in attack rates that have previously been reported, with all patients experiencing an average of 94 percent attack-free days at Week 96.
2022-02-01	BioCryst Pharmaceuticals	Results BioCryst to Present New ORLADEYO® (berotralstat) Data at 2022 American Academy of Allergy, Asthma & Immunology Annual Meeting biocryst.com ↗ “The long-term data from our APeX-2 pivotal trial continues to demonstrate that ORLADEYO delivers sustained reductions in attack rates and improvement in quality of life among patients living with HAE,” said Dr. William Sheridan, chief medical officer of BioCryst.
2021-11-05	BioCryst Pharmaceuticals	Results BioCryst Presents New Data Showing Sustained Reduction of HAE Attack Rates and Improved Patient Satisfaction After Patients Switch to ORLADEYO® (berotralstat) biocryst.com ↗ The data from APeX-S showed that HAE patients who switched to ORLADEYO from an injectable prophylactic therapy had more than 80 percent attack free months,
2021-09-15	BioCryst Pharmaceuticals	Regulatory NICE Recommends BioCryst’s ORLADEYO® (berotralstat), the First Oral, Once-daily Therapy, to Prevent Attacks in HAE Patients in the UK biocryst.com ↗ With this recommendation, HAE patients in England, Wales and Northern Ireland will have access to the first oral, once-daily therapy for routine prevention of recurrent HAE attacks.
2021-09-09	BioCryst Pharmaceuticals	Regulatory BioCryst Announces Approval of ORLADEYO® (berotralstat) in United Arab Emirates biocryst.com ↗ BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the Ministry of Health and Prevention (MOHAP) in the United Arab Emirates (UAE) has granted marketing authorization for oral, once-daily ORLADEYO ® (berotralstat) for the prevention of recurrent attacks in patients with hereditary angioedema (HAE) 12 years and older.
2021-08-25	BioCryst Pharmaceuticals	Regulatory BioCryst Announces Acceptance of Regulatory Applications for ORLADEYO® (berotralstat) by Health Canada and Swissmedic biocryst.com ↗ BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the new drug submission for ORLADEYO ® (berotralstat) has been accepted for review by Health Canada for the prevention of recurrent attacks in patients with hereditary angioedema (HAE) 12 years and older.
2021-07-10	BioCryst Pharmaceuticals	Results BioCryst Reports 96-week Data from APeX-2 Showing ORLADEYO®(berotralstat) Reduced HAE Attack Rate by 80 Percent from Baseline biocryst.com ↗ Median attack rates also decreased from 2.7 attacks/month at baseline to 0.0 attacks per month in 16 of 17 months through the same period.
2021-06-16	BioCryst Pharmaceuticals	Regulatory BioCryst Announces Acceptance and Accelerated Review of the ORLADEYO™ (berotralstat) Marketing Application by the Israeli Ministry of Health biocryst.com ↗
2021-05-12	BioCryst Pharmaceuticals	Regulatory BioCryst Receives UK Approval of ORLADEYO™ (berotralstat), First Oral, Once-daily Therapy to Prevent Attacks in Hereditary Angioedema Patients biocryst.com ↗
2021-04-30	BioCryst Pharmaceuticals	Regulatory BioCryst Receives European Commission Approval of ORLADEYO™ (berotralstat), First Oral, Once-daily Therapy to Prevent Attacks in Hereditary Angioedema Patients biocryst.com ↗ BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the European Commission (EC) has approved oral, once-daily ORLADEYO ™ (berotralstat) for the prevention of recurrent hereditary angioedema (HAE) attacks in HAE patients 12 years and older.
2021-04-14	BioCryst Pharmaceuticals	Regulatory BioCryst Announces Approval of Japanese NHI Price Listing of ORLADEYO™ (berotralstat) for Prophylactic Treatment of Hereditary Angioedema biocryst.com ↗ BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the Japanese National Health Insurance System (NHI) has approved the addition of oral, once-daily ORLADEYO ™ (berotralstat) to the NHI drug price list on April 21, 2021.
2021-03-11	BioCryst Pharmaceuticals	Regulatory Early Access to BioCryst’s Berotralstat Granted for HAE Patients in France biocryst.com ↗ This cohort ATU allows patients with HAE in France to receive treatment with berotralstat before the drug is granted marketing authorization by the European Commmission (EC).
2021-03-02	BioCryst Pharmaceuticals	Regulatory BioCryst Submits Marketing Authorization Application for UK Approval of ORLADEYO™ (berotralstat), an Oral, Once-daily Therapy to Prevent Attacks in Patients with Hereditary Angioedema biocryst.com ↗ The MAA was submitted under the MHRA’s new European Commission Decision Reliance Procedure (ECDRP).
2021-02-25	BioCryst Pharmaceuticals	Regulatory BioCryst Receives Positive CHMP Opinion for ORLADEYO™ (berotralstat), an Oral, Once-daily Therapy to Prevent Attacks in Patients with Hereditary Angioedema biocryst.com ↗ The CHMP positive opinion is based on data from the pivotal APeX-2 clinical trial and supporting data from the APeX-S trial.
2021-01-22	BioCryst Pharmaceuticals	Regulatory BioCryst Announces Approval of ORLADEYO™ (berotralstat) in Japan for the Prophylactic Treatment of Hereditary Angioedema biocryst.com ↗ ORLADEYO is the first and only prophylactic HAE medication approved in Japan.
2020-12-03	BioCryst Pharmaceuticals	Regulatory BioCryst Announces FDA Approval of ORLADEYO™ (berotralstat), First Oral, Once-daily Therapy to Prevent Attacks in Hereditary Angioedema Patients biocryst.com ↗ BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the U.S. Food and Drug Administration (FDA) has approved oral, once-daily ORLADEYO™ (berotralstat) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older.
2020-11-30	BioCryst Pharmaceuticals	Results Allergy Publishes Results from BioCryst’s APeX-J Trial of Oral, Once-Daily Berotralstat for the Prevention of HAE Attacks biocryst.com ↗ The APeX-J trial met its primary endpoint of a reduction in the rate of HAE attacks for berotralstat 150 mg compared to placebo during the 24-week period (p=0.003).
2020-11-13	BioCryst Pharmaceuticals	Results BioCryst Presents Data Showing Sustained Attack Rate Reductions, Improved Patient Satisfaction and Quality of Life for HAE Patients Taking Berotralstat in APeX-2 Trial biocryst.com ↗ BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) today presented new clinical data that further evaluates the attack rate reductions, patient satisfaction and quality of life of hereditary angioedema (HAE) patients in the APeX-2 trial over 48 weeks.
2020-10-30	BioCryst Pharmaceuticals	Regulatory MHRA Grants HAE Patients Early Access to BioCryst’s Berotralstat in United Kingdom biocryst.com ↗ BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted oral, once-daily berotralstat a positive scientific opinion through the Early Access to Medicines Scheme (EAMS).
2020-10-22	BioCryst Pharmaceuticals	Results Journal of Allergy and Clinical Immunology Publishes Results from BioCryst’s APeX-2 Pivotal Trial of Oral, Once-Daily Berotralstat for the Prevention of HAE Attacks biocryst.com ↗ In the trial, after 24 weeks, both the 110-mg and 150-mg doses of berotralstat significantly reduced HAE attack rates compared with placebo and were safe and well tolerated, with greater attack rate reductions observed for the 150-mg dose.
2020-06-06	BioCryst Pharmaceuticals	Results Long-term (48-week) Data Show Treatment with Berotralstat Provides Robust and Durable Reductions in HAE Attacks and Improvements in Quality of Life Scores biocryst.com ↗ In APeX-S, patients completing 48 weeks of treatment on 150 mg of berotralstat (n=73) had a median attack rate of zero attacks per month in six of the 12 months, including month 12 (week 48).
2020-05-26	BioCryst Pharmaceuticals	Results BioCryst to Present New Berotralstat Data at European Academy of Allergy and Clinical Immunology Digital Congress biocryst.com ↗ Berotralstat (BCX7353) Treatment Demonstrates Robust and Durable Reduction in the Rate of Hereditary Angioedema (HAE) Attacks Over 48 Weeks of the Phase 3 APeX-2 Study
2020-03-30	BioCryst Pharmaceuticals	Regulatory European Medicines Agency Validates Marketing Authorization Application for Oral, Once-Daily Berotralstat (BCX7353) to Prevent HAE Attacks biocryst.com ↗ BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) announced that the European Medicines Agency (EMA) has validated its marketing authorization application (MAA) submission for approval of oral, once-daily berotralstat (BCX7353) for the prevention of hereditary angioedema (HAE) attacks.
2020-03-16	BioCryst Pharmaceuticals	Results Berotralstat (BCX7353) Significantly Reduced Use of Acute On-Demand Medicine in HAE Patients in APeX-2 biocryst.com ↗ Additional new data show that the percentage of HAE attacks requiring re-treatment with multiple doses of on-demand therapy was lower for patients receiving berotralstat (150 mg) than placebo.
2020-03-05	BioCryst Pharmaceuticals	Regulatory BioCryst Reports Fourth Quarter and Full Year 2019 Financial Results and Upcoming Key Milestones biocryst.com ↗ Berotralstat NDA accepted by FDA; PDUFA date December 3, 2020
2020-02-18	BioCryst Pharmaceuticals	Regulatory FDA Accepts BioCryst’s NDA for Oral, Once Daily Berotralstat (BCX7353) to Prevent HAE Attacks biocryst.com ↗ BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) announced that the U.S. Food and Drug Administration (FDA) has accepted and filed its new drug application (NDA) for the approval of oral, once daily berotralstat (BCX7353) for the prevention of hereditary angioedema (HAE) attacks.
2020-02-03	BioCryst Pharmaceuticals	Regulatory BioCryst Submits Japanese New Drug Application for Oral, Once Daily Berotralstat biocryst.com ↗ BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that it has submitted a new drug application (JNDA) to the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) for approval of oral, once daily berotralstat for the prophylactic treatment of hereditary angioedema (HAE).
2020-01-12	BioCryst Pharmaceuticals	Results BioCryst to Provide Berotralstat and BCX9930 Program Updates at 38th Annual J.P. Morgan Healthcare Conference biocryst.com ↗ APeX-J trial in Japan met its primary endpoint (p=0.003) for prevention of HAE attacks, and berotralstat was safe and generally well-tolerated
2019-12-11	BioCryst Pharmaceuticals	Regulatory BioCryst Submits New Drug Application for Oral, Once Daily Berotralstat (BCX7353) to Prevent HAE Attacks biocryst.com ↗ BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) announced it has submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for approval of oral, once daily berotralstat (BCX7353) for the prevention of hereditary angioedema (HAE) attacks.
2019-11-06	BioCryst Pharmaceuticals	Results New Clinical Trial Results and Market Research Support Significant Commercial Opportunity for Oral, Once Daily BCX7353 in HAE biocryst.com ↗ BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced 48-week results from its APeX-S and APeX-2 trials and comprehensive market research which support the significant commercial opportunity for oral, once daily BCX7353 in HAE.
2019-05-21	BioCryst Pharmaceuticals	Results BioCryst’s Oral BCX7353 Meets Primary Endpoint in Phase 3 APeX-2 Trial biocryst.com ↗ Fifty percent of patients receiving 150 mg BCX7353 in APeX-2 had a ≥ 70 percent reduction in their HAE attack rate compared to baseline, compared to 15 percent of placebo patients (p=0.002).
2019-02-23	BioCryst Pharmaceuticals	Results BioCryst Reports ZENITH-1 Results With Oral BCX7353 Which Confirm Rapid Onset of Action, Sustained Activity and Robust Dose Response for Treatment of Acute HAE Attacks biocryst.com ↗ BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today reported additional topline data from the Phase 2 ZENITH-1 trial, including new data from the 250 mg and 500 mg dose cohorts.
2019-02-05	BioCryst Pharmaceuticals	Results BioCryst to Present ZENITH-1 Results at American Academy of Allergy, Asthma & Immunology Annual Meeting biocryst.com ↗ Oral Plasma Kallikrein Inhibitor BCX7353 is Safe and Effective as an On-Demand Treatment of Angioedema Attacks in Hereditary Angioedema (HAE) Patients: Results of the ZENITH-1 Trial
2018-11-16	BioCryst Pharmaceuticals	Results BioCryst Presents Data Highlighting the Rapid and Sustained Plasma Concentrations of BCX7353 in HAE Patients at the Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology biocryst.com ↗
2018-09-04	BioCryst Pharmaceuticals	Results CORRECTION -- BioCryst Reports Positive Results Across Multiple Endpoints in ZENITH-1 Trial of Oral BCX7353 as Acute Therapy for Hereditary Angioedema (HAE) Attacks biocryst.com ↗ Safety and efficacy data support advancement of 750 mg dose as first oral therapy for acute HAE attacks
2018-09-04	BioCryst Pharmaceuticals	Results BioCryst Reports Positive Results Across Multiple Endpoints in ZENITH-1 Trial of Oral BCX7353 as Acute Therapy for Hereditary Angioedema (HAE) Attacks biocryst.com ↗ BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today announced initial results from the ZENITH-1 trial showing that a single 750 mg oral dose of BCX7353 was well tolerated and superior to placebo (p<0.05) against the majority of efficacy endpoints evaluated in HAE patients suffering an acute attack.
2018-08-06	BioCryst Pharmaceuticals	Regulatory U.S. FDA Grants Fast Track Designation for BioCryst’s BCX7353 biocryst.com ↗ the Company has been granted Fast Track Designation by the U.S. Food and Drug Administration ( FDA ) for BCX7353 for the prevention of angioedema attacks in patients with hereditary angioedema (HAE).
2018-07-25	BioCryst Pharmaceuticals	Results BioCryst Announces Publication of APeX-1 Clinical Trial Results for BCX7353 in The New England Journal of Medicine biocryst.com ↗ The trial demonstrated that oral administration of BCX7353 at a dose of 125 mg or more resulted in a significantly lower rate of attacks compared with placebo.
2018-06-25	BioCryst Pharmaceuticals	Regulatory BioCryst Reports Agreement with PMDA on Phase 3 Clinical Trial and Regulatory Requirements for Marketing Authorization of BCX7353 in Japan biocryst.com ↗ today announced that the Company has reached agreement on the design of a Phase 3 trial and regulatory requirements for marketing authorization of BCX7353 for Hereditary Angioedema (“HAE”) with the Pharmaceuticals and Medical Devices Agency (“PMDA”) in Japan .
2018-05-24	BioCryst Pharmaceuticals	Regulatory BioCryst's BCX7353 Receives European Regulatory Designations for the Treatment of Hereditary Angioedema biocryst.com ↗ In addition, the United Kingdom’s Medicines and Healthcare products Regulatory Agency (“MHRA”) has granted a Promising Innovative Medicine (“PIM”) designation to BCX7353.
2017-10-09	BioCryst Pharmaceuticals	Results BioCryst Reports Positive Interim Results from its APeX-1 Trial biocryst.com ↗ BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today announced results from an interim analysis of its Phase 2 APeX-1 trial in hereditary angioedema (HAE).
2017-10-09	BioCryst Pharmaceuticals	Results BioCryst Reports Additional Positive Results From the Second Interim Analysis of Its APeX-1 Trial biocryst.com ↗ We are delighted to see that a daily dose of 125 mg of BCX7353 results in a high level of efficacy with an improved tolerability profile compared to the 350 mg dose observed in the first interim analysis.
2017-10-09	BioCryst Pharmaceuticals	Regulatory BioCryst Expands Development of BCX7353 to Explore Treatment of Acute HAE Attacks biocryst.com ↗ The Company has received initial regulatory approvals in Europe to initiate the ZENITH-1 exploratory clinical trial this summer.
2017-10-09	BioCryst Pharmaceuticals	Results BioCryst Announces Successful Phase 1 Clinical Trial of BCX7353 biocryst.com ↗ BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today announced that the randomized, placebo-controlled, Phase 1 clinical trial of orally-administered BCX7353 in healthy volunteers successfully met all of its objectives.
2017-10-09	BioCryst Pharmaceuticals	Results BioCryst Announces Positive Results from its APeX-1 Phase 2 Trial in HAE biocryst.com ↗ BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today announced final results from its Phase 2 APeX-1 clinical trial in hereditary angioedema (HAE).

All press releases naming this drug 117 releases

Date	Issuer	Release
Show all 117 releases opens the full record — a long page

Evidence & citations 11 cited values

Every value below carries the sentence it was read from. 16 sources stand behind the page.

Field	Value	Cited text
Known as	berotralstat	“BACKGROUND: Berotralstat (BCX7353) is an oral, once-daily inhibitor of plasma kallikrein recently approved for prevention of angioedema attacks in adults and adolescents with...” PMID 34161665 ↗ Jun 2021 4 “Berotralstat (BCX7353) is an oral, once-daily, highly selective inhibitor of plasma kallikrein in development for prophylaxis of angioedema attacks in HAE patients.” PMID 33247955 ↗ Dec 2020 NCT04428632 ↗ NCT05453968 ↗ NCT04933721 ↗
Known as	BCX-7353 DIHYDROCHLORIDE	ChEMBL registry synonym — accepted as the source's own label CHEMBL5189739 ↗
Known as	BCX7353	ClinicalTrials.gov intervention name — accepted as the source's own label NCT03202784 ↗ 8 NCT03472040 ↗ NCT03136237 ↗ NCT03485911 ↗ NCT02448264 ↗ NCT02870972 ↗ NCT02819102 ↗ NCT03873116 ↗ NCT03240133 ↗
Known as	BCX7353.2HCl	ChEMBL registry synonym — accepted as the source's own label CHEMBL5189739 ↗
Known as	Berotralstat dihydrochloride	ChEMBL registry synonym — accepted as the source's own label CHEMBL5189739 ↗
Known as	BEROTRALSTAT HYDROCHLORIDE	ChEMBL registry synonym — accepted as the source's own label CHEMBL5189739 ↗
Known as	Orladeyo	ChEMBL registry synonym — accepted as the source's own label CHEMBL5189739 ↗
Action	Inhibit	“inhibitor of plasma kallikrein” PMID 30044938 ↗ Jul 2018 1 “Plasma kallikrein inhibitor” CHEMBL5189739 ↗
Modality	Small molecule	“BCX7353 is a potent oral small-molecule inhibitor of plasma kallikrein” PMID 30044938 ↗ Jul 2018
Route	Oral	“BCX7353 capsules administered orally” NCT02448264 ↗
Target	KLKB1	“Plasma kallikrein inhibitor” CHEMBL5189739 ↗