BioCryst Unveils New Findings on ORLADEYO’s Efficacy in HAE
Introduction to BioCryst's Promising Data
Research Triangle Park, N.C. — BioCryst Pharmaceuticals, Inc. (NASDAQ: BCRX) has unveiled important real-world evidence showcasing the use of oral, once-daily ORLADEYO® (berotralstat). This groundbreaking data indicates significant and sustained reductions in hereditary angioedema (HAE) attack rates among adolescents and individuals grappling with severe forms of this condition. The evidence encompasses follow-up assessments across an extended 18-month period after commencing treatment with ORLADEYO.
Detailed Presentation of Research Findings
The pivotal results were presented through two informative posters at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) 2025 conference held in Montreal. The findings reflect a comprehensive analysis derived from patient data collected by BioCryst’s exclusive pharmacy, detailing the experiences of both adolescents and severely affected patients.
Understanding the Study Parameters
The first poster, titled "Real-World Hereditary Angioedema Attack Rates Before and After Berotralstat Initiation Among Patients with C1 Inhibitor Deficiency (Type I/II) and ?8 Attacks/month," elaborated on how 56 U.S. patients suffering from C1-inhibitor deficiency benefited from ORLADEYO treatment.
- Patients showed notable reductions in HAE attack frequencies during the respective 90-day follow-up intervals. Attack rates were recorded between 1.24 and 1.90 per month, in stark contrast to baseline figures, which ranged from 7.78 to 8.23 attacks per month.
- Particularly significant were the results at various milestones: 6.25 fewer attacks per month at 12 months, and a further 6.43 reductions noted at the 18-month mark, both with high statistical significance (p<0.001).
Adolescents' Outcomes with ORLADEYO
The second poster addressed treatment impacts on adolescents aged 12 to 17 years receiving ORLADEYO. Findings captured from 99 patients documented critical attack frequency changes:
- The adolescent group experienced a decline in attack rates to between 0.36 and 0.76 attacks per month, compared to a baseline mean of 2.07 to 2.30 attacks.
- Results indicated a noteworthy decrease of 1.56 attacks per month at the 12-month assessment, with reductions increasing to 1.85 attacks at the 18-month follow-up, showcasing statistical significance across the board (p<0.001).
Expert Insights on the Results
Dr. Raffi Tachdjian, an associate clinical professor of medicine and pediatrics, emphasized that the positive outcomes showcased in the new evidence provide re-assurance to both healthcare professionals and patients involved in the treatment of HAE. ORLADEYO appears to effectively manage and reduce attacks, which is especially crucial for younger individuals with severe conditions.
Further Observations on ORLADEYO
Moreover, Dr. Donald S. Fong, BioCryst’s Chief Medical Officer, reiterated the commitment to developing substantial evidence supporting ORLADEYO's efficacy across diverse patient demographics. These findings enhance understanding of ORLADEYO's potential as a dependable treatment option regardless of patient age or the severity of HAE attacks.
About ORLADEYO
ORLADEYO (berotralstat) stands as the first oral medication specifically designed to preemptively manage hereditary angioedema attacks in patients aged 12 years and older. Administering just one capsule daily effectively mitigates HAE attack occurrences by inhibiting plasma kallikrein activity.
Indications and Important Safety Information
ORLADEYO is indicated for the prophylaxis of HAE attacks in adults and pediatric patients aged 12 and above. However, it is essential to note that the medication is not intended for treating acute HAE attacks, and the safety and effectiveness of ORLADEYO have yet to be established for patients under 12 years.
Patients with moderate or severe hepatic impairment may require a reduced dosage. Furthermore, caution should be exercised with concomitant medications, as ORLADEYO interacts with several medications, potentially affecting their efficacy.
About BioCryst Pharmaceuticals
BioCryst Pharmaceuticals is a renowned biotechnology company dedicated to enhancing the lives of individuals affected by rare diseases, particularly hereditary angioedema. The company's substantial expertise in drug design propels its mission to develop outstanding small-molecule and protein therapeutics for challenging health conditions. To learn more about their innovative products and commitments, please visit their official website.
Frequently Asked Questions
What is ORLADEYO used for?
ORLADEYO is utilized for the prophylaxis of hereditary angioedema attacks in patients aged 12 and older.
How does ORLADEYO work?
This medication acts by inhibiting plasma kallikrein, which is crucial in preventing HAE attacks.
What were the results from the recent studies?
The studies revealed significant and sustained reductions in monthly HAE attack rates among both adolescents and severely affected individuals after starting ORLADEYO.
Are there any side effects associated with ORLADEYO?
Common side effects include abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease.
How can I get more information about BioCryst?
For further information, you can visit BioCryst Pharmaceuticals' official website or contact their customer service.
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