BioCryst Pharmaceuticals' Drug in the FDA Approval Process
This section highlights FDA-related milestones and regulatory updates for drugs developed by BioCryst Pharmaceuticals (BCRX).
Over the past two years, BioCryst Pharmaceuticals has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
ORLADEYO. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
ORLADEYO (berotralstat) FDA Regulatory Timeline and Events
ORLADEYO (berotralstat) is a drug developed by BioCryst Pharmaceuticals for the following indication: Hereditary angioedema (HAE).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- ORLADEYO (berotralstat)
- Announced Date:
- August 25, 2026
- Indication:
- Hereditary angioedema (HAE)
Announcement
BioCryst Pharmaceuticals, Inc. 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.
Read Announcement- Drug:
- ORLADEYO (berotralstat)
- Announced Date:
- June 12, 2026
- Indication:
- Hereditary angioedema (HAE)
Announcement
BioCryst Pharmaceuticals, Inc announced new clinical data and real-world evidence for ORLADEYO® (berotralstat), the first and only targeted oral prophylactic therapy for patients with hereditary angioedema (HAE) aged 2 and older, in addition to new data from the Phase 1b/2 multicenter, dose-ranging, open-label ALPHA-STAR study of navenibart, an investigational, long-acting, monoclonal antibody plasma kallikrein inhibitor for prophylaxis to prevent attacks of HAE.
AI Summary
BioCryst Pharmaceuticals said new clinical and real-world data support ORLADEYO (berotralstat), its daily oral medicine for preventing hereditary angioedema (HAE) attacks in children and adults age 2 and older. In the 48-week APeX-P pediatric study, ORLADEYO was linked to early and lasting drops in the number of attacks that needed emergency or on-demand care. The company also said real-world evidence showed similar reductions in attack burden and healthcare use across different patient groups.
BioCryst also reported new Phase 1b/2 ALPHA-STAR data for navenibart, an investigational long-acting antibody designed to block plasma kallikrein. A post hoc analysis found consistent attack-rate reductions across patient subgroups, including differences in age, body mass index, and baseline attack rate. These findings support continued Phase 3 testing of navenibart as a possible treatment option for a broad HAE population.
Read Announcement- Drug:
- ORLADEYO (berotralstat)
- Announced Date:
- May 27, 2026
- Indication:
- Hereditary angioedema (HAE)
Announcement
BioCryst Pharmaceuticals, Inc announced that the company will present seven abstracts from its hereditary angioedema (HAE) portfolio at the Annual Meeting of the European Academy of Allergy and Clinical Immunology (EAACI) taking place in Istanbul, Turkey, from June 12-15, 2026..
AI Summary
BioCryst Pharmaceuticals said it will present seven abstracts from its hereditary angioedema (HAE) program at the EAACI Annual Meeting in Istanbul, Turkey, from June 12-15, 2026. The presentations include six abstracts with new clinical trial and real-world data for ORLADEYO (berotralstat) and one abstract with clinical trial results for navenibart.
ORLADEYO is the first and only oral medicine approved to help prevent HAE attacks in adults and children age 2 and older. It works by lowering plasma kallikrein activity, which helps reduce attacks. Navenibart is an experimental long-acting antibody that also targets plasma kallikrein and is being studied as a possible preventive treatment for HAE.
The company says navenibart may eventually offer dosing every 3 or 6 months, which could make treatment easier for patients. BioCryst’s EAACI presentations highlight its focus on both an established therapy and a next-generation option for long-term HAE care.
Read Announcement- Drug:
- ORLADEYO (berotralstat)
- Announced Date:
- February 11, 2026
- Indication:
- Hereditary angioedema (HAE)
Announcement
BioCryst Pharmaceuticals, Inc announced that the company will present nine abstracts from its hereditary angioedema (HAE) portfolio at the 2026 American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting taking place in Philadelphia from February 27-March 2, 2026.
AI Summary
BioCryst Pharmaceuticals will present nine abstracts from its hereditary angioedema (HAE) portfolio at the 2026 American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting in Philadelphia, February 27–March 2, 2026. The presentations highlight new data on prevention and long‑term control of HAE.
Eight poster presentations are scheduled for Friday, February 27, from 2:45–3:45 p.m. EST at the Convention Center, Level 2, Hall E. A late‑breaking abstract will be presented Sunday, March 1, from 9:45–10:45 a.m. EST in the same location. The late‑breaking poster (Poster #L59) reports ALPHA‑SOLAR interim results titled “Long‑Term, Sustained, Robust Hereditary Angioedema Attack Suppression with Navenibart Administered Every 3 and 6 Months.”
BioCryst’s portfolio includes ORLADEYO (berotralstat), an oral preventive therapy for HAE, and navenibart, an investigational monoclonal antibody targeting plasma kallikrein being studied for potential dosing every 3 or 6 months.
Read Announcement- Drug:
- ORLADEYO (berotralstat)
- Announced Date:
- December 12, 2025
- Indication:
- Hereditary angioedema (HAE)
Announcement
BioCryst Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved its New Drug Application (NDA) for the use of an oral pellet formulation of once-daily ORLADEYO® (berotralstat) for prophylactic therapy in pediatric patients with hereditary angioedema (HAE) aged 2 to <12 years.
AI Summary
BioCryst Pharmaceuticals announced that the FDA has approved its New Drug Application for an oral pellet formulation of once-daily ORLADEYO (berotralstat) as prophylactic therapy for pediatric patients with hereditary angioedema (HAE) aged 2 to less than 12 years. This makes ORLADEYO the first and only targeted oral prophylactic option for people with HAE aged 2 and older, offering a noninjectable choice designed to better suit young children and their families.
The approval was supported by interim results from the APeX-P trial, which showed ORLADEYO was well tolerated, had a consistent safety profile across the age group, and produced early and sustained reductions in monthly attack rates. The most common treatment-emergent adverse event was nasopharyngitis. The new pellet is sprinkle-like and can be poured into the mouth with water or milk or added to soft, non-acidic food, providing a child-friendly method of administration. BioCryst said the option helps families and clinicians tailor long-term prevention to children’s needs.
Read Announcement- Drug:
- ORLADEYO (berotralstat)
- Announced Date:
- October 23, 2025
- Indication:
- Hereditary angioedema (HAE)
Announcement
BioCryst Pharmaceuticals, Inc. announced that the company will present four abstracts at the Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI) taking place in Orlando, Fla., from November 6-10, 2025.
AI Summary
BioCryst Pharmaceuticals announced it will present four abstracts at the Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI) in Orlando, Fla., from November 6–10, 2025.
The data include a second interim analysis of the APeX-P trial evaluating long-term prophylaxis with an oral granule formulation of once-daily ORLADEYO® (berotralstat) in pediatric hereditary angioedema (HAE) patients aged 2 to <12 years. The company will also share findings on the psychosocial impact of HAE on young patients and their caregivers.
Poster presentations include:
• Hereditary Angioedema Attack Rates in Children 2–12 with ORLADEYO (ePoster #R089; Nov 7);
• Psychosocial Impact of HAE on Young Patients and Caregivers (ePoster #R102; Nov 7);
• Burden of Attack for Young Patients and Caregivers (ePoster #R109; Nov 8);
• Emergency Department Experiences of Young Patients and Caregivers (ePoster #R117; Nov 9).
All ePosters will be displayed in the exhibit hall and available online on demand for registered attendees beginning November 6 at 8:00 a.m. ET via the ACAAI website.
Read Announcement- Drug:
- ORLADEYO (berotralstat)
- Announced Date:
- June 16, 2025
- Indication:
- Hereditary angioedema (HAE)
Announcement
BioCryst Pharmaceuticals, Inc. ( announced new data on the long-term efficacy and safety of ORLADEYO® (berotralstat) for the prophylactic treatment of hereditary angioedema (HAE) in patients across all age groups.
AI Summary
BioCryst Pharmaceuticals recently presented new data on the long-term efficacy and safety of ORLADEYO® (berotralstat) for preventing hereditary angioedema (HAE) attacks in patients of all ages. The findings come from both clinical trials and real-world studies, highlighting that the medication consistently reduces the number and severity of HAE attacks in pediatric, adolescent, and adult populations. Patients experienced fewer attacks and significant improvements in daily functioning and overall quality of life. The data affirm that ORLADEYO is well tolerated, with a safety profile that remains consistent across age groups. These results add to the growing evidence supporting ORLADEYO as a reliable long-term prophylactic treatment option for those living with HAE, reinforcing its role in improving both the clinical outcomes and the everyday wellbeing of patients affected by this rare condition.
Read Announcement- Drug:
- ORLADEYO (berotralstat)
- Announced Date:
- June 13, 2025
- Indication:
- Hereditary angioedema (HAE)
Announcement
BioCryst Pharmaceuticals, Inc. 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.
AI Summary
BioCryst Pharmaceuticals announced that Colombia’s National Institute of Drug and Food Surveillance (INVIMA) has granted approval for its oral once-daily ORLADEYO® (berotralstat). The approval allows ORLADEYO to be used as a prophylactic treatment to prevent hereditary angioedema (HAE) attacks in adults and pediatric patients aged 12 years and older. This decision marks an important step in providing more treatment options for people living with HAE in Colombia and potentially across Latin America. BioCryst is collaborating with Pint Pharma to help bring the treatment to market in the region. The approval follows previous INVIMA decisions in other Latin American countries, underlining BioCryst’s commitment to expanding access to innovative therapies for HAE patients.
Read Announcement- Drug:
- ORLADEYO (berotralstat)
- Announced Date:
- June 2, 2025
- Indication:
- Hereditary angioedema (HAE)
Announcement
BioCryst Pharmaceuticals, Inc. announced that new data on oral, once-daily ORLADEYO® (berotralstat) for the prophylactic treatment of hereditary angioedema (HAE) will be presented at the European Academy of Allergy and Clinical Immunology (EAACI) meeting in Glasgow, United Kingdom, from June 13 to 16, 2025.
AI Summary
BioCryst Pharmaceuticals announced that it will present new data on ORLADEYO® (berotralstat), an oral, once-daily treatment for the prophylaxis of hereditary angioedema (HAE), at the European Academy of Allergy and Clinical Immunology (EAACI) meeting in Glasgow, United Kingdom, from June 13 to 16, 2025. The company plans to share four abstracts that cover various aspects of the therapy, including its impact on quality of life, positive patient feedback from focus groups, and long-term effectiveness and tolerability as observed in the Berolife Study. Additionally, interim results highlighting a reduction in the number of HAE attacks requiring intervention in pediatric patients will be presented. This upcoming presentation underscores ORLADEYO’s role as a pioneering oral therapy for both adult and adolescent patients with HAE and BioCryst’s ongoing commitment to improving care for those affected by this rare disease.
Read Announcement- Drug:
- ORLADEYO (berotralstat)
- Announced Date:
- May 30, 2025
- Indication:
- Hereditary angioedema (HAE)
Announcement
BioCryst Pharmaceuticals, Inc announced new data which highlights the reduction in the percentage of days with hereditary angioedema (HAE) symptoms among young children initiating berotralstat in the APeX-P trial, and the broad safety and efficacy outcomes observed across all age groups of patients taking ORLADEYO to prevent HAE attacks.
AI Summary
BioCryst Pharmaceuticals recently announced promising new data from their APeX-P trial. The study showed that young children with hereditary angioedema (HAE) who started treatment with berotralstat experienced a significant drop in the percentage of days with HAE symptoms—from an average of 11% before treatment to 4% after 12 weeks. This reduction indicates that the treatment may help children experience fewer symptoms and improved daily life.
In addition to these findings in children, the results also highlighted the broad safety and effectiveness of ORLADEYO (berotralstat) across all age groups. Patients from adolescents to adults taking this once-daily oral medication reported consistent benefits in preventing HAE attacks. The data underscores the potential of this therapy to offer meaningful, long-term relief, ensuring more attack-free days for individuals living with HAE regardless of their age.
Read Announcement- Drug:
- ORLADEYO (berotralstat)
- Announced Date:
- May 16, 2025
- Indication:
- Hereditary angioedema (HAE)
Announcement
BioCryst Pharmaceuticals, Inc. announced new real-world evidence on the use of oral, once-daily ORLADEYO® (berotralstat) in adolescents and people with severe HAE showing significant and sustained reductions in HAE attack rates through 18 months of follow-up after beginning treatment with ORLADEYO in both patient populations.
AI Summary
BioCryst Pharmaceuticals announced promising real-world evidence for its oral, once-daily therapy ORLADEYO® (berotralstat) in treating hereditary angioedema (HAE) in adolescents and patients with severe disease. The study showed significant and sustained reductions in HAE attack rates during 18 months of follow-up after starting treatment. Adolescents experienced up to 1.85 fewer attacks per month, while those with severe HAE saw reductions of up to 6.43 attacks per month compared to baseline levels.
These results, presented at the 2025 ISPOR conference, indicate that ORLADEYO can offer improved attack control over time. This new evidence provides additional reassurance for physicians and patients managing HAE, suggesting that long-term treatment with ORLADEYO is effective in reducing the frequency of painful HAE attacks in diverse patient populations.
Read Announcement- Drug:
- ORLADEYO (berotralstat)
- Announced Date:
- May 14, 2025
- Target Action Date:
- September 12, 2025
- Indication:
- Hereditary angioedema (HAE)
Announcement
BioCryst Pharmaceuticals, Inc. FDA grants Priority Review of application, with PDUFA target action date of September 12, 2025–
AI Summary
BioCryst Pharmaceuticals recently received FDA Priority Review for its New Drug Application for ORLADEYO®, an oral once-daily therapy intended to prevent hereditary angioedema attacks in pediatric patients aged 2 to 11 years. The FDA’s decision comes with a Prescription Drug User Fee Act (PDUFA) target action date set for September 12, 2025. If approved, ORLADEYO® would be the first targeted oral prophylactic treatment for children under 12, offering a more convenient option than current therapies. The application was supported by positive interim data from the APeX-P clinical trial, which showed that the treatment was well tolerated and effective in reducing monthly attack rates in this young population. BioCryst’s initiative aims to address a significant need for early intervention in hereditary angioedema, potentially improving the quality of life for affected children and their families.
Read Announcement- Drug:
- ORLADEYO (berotralstat)
- Announced Date:
- May 14, 2025
- Indication:
- Hereditary angioedema (HAE)
Announcement
BioCryst Pharmaceuticals, Inc announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for the use of oral, once-daily ORLADEYO® (berotralstat) in pediatric patients with hereditary angioedema (HAE) aged 2 to 11 years.
AI Summary
BioCryst Pharmaceuticals announced that the FDA has accepted its New Drug Application (NDA) for the use of ORLADEYO® (berotralstat) as a once-daily oral treatment for pediatric patients aged 2 to 11 with hereditary angioedema (HAE). The therapy, if approved, would be the first targeted oral prophylactic treatment for children under 12 with HAE.
The FDA has granted Priority Review for this application, with a Prescription Drug User Fee Act (PDUFA) target action date of September 12, 2025. This decision is based on encouraging interim data from the APeX-P clinical trial, which is the largest trial for a prophylactic therapy in this age group. The promising results indicate that ORLADEYO is well tolerated and effectively reduces the frequency of HAE attacks, offering a more convenient and safer option for young children and their families.
Read Announcement- Drug:
- ORLADEYO (berotralstat)
- Announced Date:
- February 12, 2025
- Indication:
- Hereditary angioedema (HAE)
Announcement
BioCryst Pharmaceuticals, Inc. 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.
AI Summary
BioCryst Pharmaceuticals announced that Infarmed in Portugal has recommended ORLADEYO® (berotralstat) for the routine prevention of recurrent hereditary angioedema (HAE) attacks in eligible patients aged 12 and older. This new recommendation means that Portuguese patients will now have access to the first oral, once-daily therapy designed specifically to reduce the frequency of HAE attacks. By easing the treatment process with a simple daily capsule, ORLADEYO offers a convenient, modern option that could improve the quality of life for those affected by this rare condition. The treatment works by decreasing the activity of plasma kallikrein, an enzyme that contributes to HAE attacks. This positive recommendation from Infarmed follows the earlier European Commission marketing authorization and adds to the growing availability of this innovative prophylactic treatment across multiple countries.
Read Announcement- Drug:
- ORLADEYO (berotralstat)
- Announced Date:
- February 10, 2025
- Indication:
- Hereditary angioedema (HAE)
Announcement
BioCryst Pharmaceuticals, Inc. announced that the company will present five abstracts featuring new clinical and real-world outcomes with oral, once-daily ORLADEYO® (berotralstat) for the prophylactic treatment of hereditary angioedema (HAE) at the 2025 American Academy of Allergy, Asthma & Immunology (AAAAI) / World Allergy Organization (WAO) joint congress.
AI Summary
BioCryst Pharmaceuticals, Inc. announced it will present five abstracts at the 2025 American Academy of Allergy, Asthma & Immunology (AAAAI) / World Allergy Organization (WAO) joint congress. The abstracts highlight new clinical and real-world outcomes for ORLADEYO® (berotralstat), an oral, once-daily medication designed to prevent attacks in patients with hereditary angioedema (HAE).
One key abstract is a late-breaking study presenting initial results from the APeX-P trial, which evaluates the effectiveness of ORLADEYO in pediatric patients aged 2 to less than 12 years. The other abstracts provide insights into attack rate reductions, patient reported outcomes, and treatment preferences. The presentations, scheduled for March 2 at the San Diego Convention Center, will share valuable data that could impact the management of HAE across different age groups.
Read Announcement- Drug:
- ORLADEYO (berotralstat)
- Announced Date:
- November 18, 2024
- Indication:
- Hereditary angioedema (HAE)
Announcement
BioCryst Pharmaceuticals, Inc announced that the Health Services Executive (HSE) in Ireland has recommended ORLADEYO® (berotralstat) for the routine prevention of recurrent attacks of hereditary angioedema (HAE) in eligible patients 12 years and older.
AI Summary
BioCryst Pharmaceuticals, Inc. announced that Ireland’s Health Service Executive (HSE) has recommended ORLADEYO® (berotralstat) for the routine prevention of recurrent hereditary angioedema (HAE) attacks in eligible patients aged 12 years and older. This recommendation provides HAE patients in Ireland with the first oral, once-daily treatment option aimed at reducing the frequency of their attacks.
The HSE decision supports a modern approach to HAE management by giving physicians greater flexibility in prescribing a reliable prophylactic therapy. With ORLADEYO already having received European Commission marketing authorization in 2021 and being licensed in 44 countries, the new recommendation underlines its growing acceptance as a valuable treatment option. This development is expected to improve quality of life for HAE patients by offering them an easy-to-use and effective treatment alternative.
Read Announcement- Drug:
- ORLADEYO (berotralstat)
- Announced Date:
- October 10, 2024
- Indication:
- Hereditary angioedema (HAE)
Announcement
BioCryst Pharmaceuticals, Inc announced that the company will present four abstracts on oral, once-daily ORLADEYO® (berotralstat) for the prophylactic treatment of hereditary angioedema (HAE) in patients 12 years and older at the Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI).
AI Summary
BioCryst Pharmaceuticals, Inc. announced that it will present four abstracts at the Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI) in Boston from October 24-28, 2024. The abstracts will focus on the use of the company’s oral, once-daily therapy, ORLADEYO® (berotralstat), for the prophylactic treatment of hereditary angioedema (HAE) in patients 12 years and older. Data to be presented is expected to show high patient adherence and persistence, sustained reductions in HAE attacks, and a patient preference for this convenient, oral treatment compared to other methods. The findings underscore key benefits of ORLADEYO® in real-world settings and highlight its potential to improve the quality of life for those managing HAE through an effective and patient-friendly treatment option.
Read Announcement
BioCryst Pharmaceuticals FDA Events - Frequently Asked Questions
As of now, BioCryst Pharmaceuticals (BCRX) has not received any FDA approvals for its therapy in the last two years.
In the past two years, BioCryst Pharmaceuticals (BCRX) has reported FDA regulatory activity for ORLADEYO (berotralstat).
The most recent FDA-related event for BioCryst Pharmaceuticals occurred on August 25, 2026, involving ORLADEYO (berotralstat). The update was categorized as "Approved," with the company reporting: "BioCryst Pharmaceuticals, Inc. 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."
Currently, BioCryst Pharmaceuticals has one therapy (ORLADEYO (berotralstat)) targeting the following condition: Hereditary angioedema (HAE).
More FDA Event Resources from MarketBeat
Companies With Recent FDA Events
FDA Event Stage Terminology & Abbreviation Guide
- NDA: New Drug Application
- ANDA: Abbreviated New Drug Application
- sNDA: Supplemental New Drug Application
- BLA: Biologics License Application
- sBLA: Supplemental Biologics License Application
- FDA Approved: Approved by the FDA
- EMA: European Medicines Agency
- CE Mark: European Union Certification
- NMPA: China National Medical Products Administration
- MHLW: Japanese Ministry of Health
- FDA Meeting: Consultation with FDA
- Pre-IND: Pre-Investigational New Drug Meeting
- Breakthrough Therapy: Special FDA designation for promising therapies
- Fast Track: Accelerated FDA approval pathway
- Orphan Drug: Designation for rare disease treatments
- RPD: Rare Pediatric Disease Designation
- RMAT: Regenerative Medicine Advanced Therapy
- DSMB Review: Data Safety Monitoring Board Review
- IDMC Review: Independent Data Monitoring Committee
- MAA: MHRA Marketing Authorization Application
- RTF: Refusal to File (Rejected Application)
- 510(k): FDA Clearance for Medical Devices
- Rolling Submission: Staggered regulatory review process
FDA progress for NASDAQ:BCRX last updated on 8/25/2026 by MarketBeat.com Staff. We continuously monitor for new FDA events and market data.
