Business Details
40 Years
experience
Leading Biotech Solutions for Rare Diseases
Bio Cryst Pharmaceuticals is a global biotechnology company dedicated to discovering, developing, and delivering innovative medicines for patients with rare diseases. Founded in 1986, we combine decades of scientific expertise with a patient-centric approach to address unmet medical needs. Our commitment to structure-guided drug design has led to breakthrough therapies for conditions like hereditary angioedema (HAE) and antiviral diseases. We prioritize collaboration with patient advocacy groups to ensure treatments align with real-world needs. With headquarters in Durham, North Carolina, and a Discovery Center in Birmingham, Alabama, our team leverages cutting-edge technology to advance novel oral and protein therapeutics.Our leadership in rare disease research is supported by strategic partnerships and a transparent, ethical corporate culture. We focus on delivering first-in-class or best-in-class treatments that improve quality of life globally. Our employees are empowered to innovate, ensuring resilience and excellence in every project. Through rigorous clinical trials and regulatory approvals, we bring transformative therapies to market. Bio Cryst Pharmaceuticals remains steadfast in its mission to empower patients by redefining what is possible in rare disease treatment.
Services
ORLADEYO Therapies
RAPIVAB Treatments
HAE Drug Development
Antiviral Research
Clinical Trials
Details
- Year established: 1986
Highlights and features
- We have over 35 years of experience in rare disease drug development.
- Our structure-guided drug design approach enables precision in therapy development.
- We maintain global partnerships to expand patient access to critical treatments.
- Our patient-centric model integrates advocacy insights into every research phase.
- We hold FDA approvals for groundbreaking therapies like ORLADEYO and RAPIVAB.
Services
ORLADEYO Therapies
ORLADEYO (berotralstat) is a once-daily oral therapy for preventing hereditary angioedema attacks in patients aged 12 and older. This groundbreaking medication reduces swelling episodes by targeting plasma kallikrein, improving patients' quality of life. Clinical trials demonstrated its efficacy in reducing attack frequency, leading to FDA approval in 2020.
RAPIVAB Treatments
RAPIVAB (peramivir injection) is an intravenous neuraminidase inhibitor for treating acute influenza in adults and children. Approved by the FDA since 2010, it provides rapid antiviral action against both influenza A and B strains. The drug received emergency use authorization during the H1N1 pandemic and remains a critical treatment option for severe flu cases.
Clinical Trials
Bio Cryst Pharmaceuticals conducts global clinical trials for rare disease therapies, including phase 3 studies for BCX9930 in complement-mediated diseases. Our research follows strict FDA guidelines, with trials listed on ClinicalTrials.gov. We prioritize patient safety and data transparency while advancing novel treatments for underserved populations.
Patient Advocacy
Our patient advocacy programs collaborate with rare disease organizations to amplify patient voices. We provide resources for disease awareness, policy development, and treatment access. The Global Patient Advocacy team works directly with patient communities to ensure therapies meet real-world needs through education and support initiatives.
Structure-Guided Design
Bio Cryst Pharmaceuticals's proprietary structure-guided drug design platform combines computational modeling with experimental data to create targeted therapies. This approach accelerates discovery of small-molecule and protein therapeutics for rare diseases. The technology has produced multiple clinical candidates, including next-generation factor D inhibitors for complementopathies.
People
Jon P. Stonehouse
Chief Executive Officer
Jon Stonehouse led Bio Cryst Pharmaceuticals as CEO from 2007 until 2025, bringing 25 years of pharmaceutical industry experience. Prior roles included senior positions at Merck KGaA and AstraZeneca. He holds a microbiology degree from the University of Minnesota and expanded Bio Cryst Pharmaceuticals's global footprint during his tenure.
Dr. Yarlagadda S. Babu
Chief Discovery Officer
Dr. Babu has served as Chief Discovery Officer since joining Bio Cryst Pharmaceuticals in 1986. He earned his PhD in organic chemistry and pioneered the company's structure-guided drug design platform. With over 30 years at Bio Cryst Pharmaceuticals, he leads discovery efforts for rare disease therapies from the Birmingham research center.
Dr. Hutson
Board Chair
Dr. Hutson chairs Bio Cryst Pharmaceuticals's board with 30+ years in pharmaceutical R&D leadership. Formerly Pfizer's Senior Vice President of Global R&D, she holds a physiology PhD from Vanderbilt University. She has guided Bio Cryst Pharmaceuticals's strategic direction since 2012 and chairs the board since 2023.
Machelle Sanders
Board Member
Machelle Sanders joined the board in 2022 after serving as North Carolina Secretary of Commerce. With 30+ years in biopharma operations, including Biogen's $8B MS franchise leadership, she brings manufacturing and regulatory expertise. She holds a biochemistry degree from North Carolina State University.
Dr. Sandra McKee
Board Member
Dr. McKee serves on Bio Cryst Pharmaceuticals's board with extensive FDA and oncology expertise. As former Deputy Center Director at the FDA's Oncology Center of Excellence, she shaped regulatory pathways for cancer therapies. She earned her MD from Tulane University and leads oncology strategy at AstraZeneca.
Questions
What rare diseases does Bio Cryst Pharmaceuticals focus on?
Bio Cryst Pharmaceuticals specializes in hereditary angioedema (HAE) and antiviral therapies, with emerging programs in complement-mediated diseases. Our research targets unmet needs in rare conditions through precision drug design.
How can patients access Bio Cryst Pharmaceuticals medications?
Our therapies are available through specialty pharmacies globally. For HAE prevention, ORLADEYO is distributed in the US, Japan, and Europe. Contact our medical information team for specific availability details.
Does Bio Cryst Pharmaceuticals conduct clinical trials?
Yes, we run global clinical trials for rare disease therapies. Current studies include BCX9930 for complementopathies. Trial information is available on ClinicalTrials.gov or through our medical affairs department.
How does Bio Cryst Pharmaceuticals support patient advocacy?
We partner with rare disease organizations to advance patient-centered care. Our advocacy programs provide educational resources, policy support, and research funding to improve treatment access and disease awareness.
What is structure-guided drug design?
This approach combines computational modeling with experimental data to create targeted therapies. Bio Cryst Pharmaceuticals uses this method to develop precise small-molecule and protein therapeutics for rare diseases more efficiently.
Where is Bio Cryst Pharmaceuticals headquartered?
Our global headquarters are in Durham, North Carolina, with a Discovery Center in Birmingham, Alabama. We maintain regional offices worldwide to support our international research and commercial operations.