BioCryst reports long-term efficacy of ORLADEYO in HAE treatment

Published 16/06/2025, 12:10
BioCryst reports long-term efficacy of ORLADEYO in HAE treatment

RESEARCH TRIANGLE PARK, N.C. - BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX), a pharmaceutical company with impressive revenue growth of 41.67% over the last twelve months, announced new data showing sustained efficacy and safety of ORLADEYO (berotralstat) for hereditary angioedema (HAE) prevention across pediatric, adolescent, and adult populations. According to InvestingPro data, the company maintains a healthy gross profit margin of 64.25%, reflecting strong commercial execution.

The data, presented at the European Academy of Allergy and Clinical Immunology meeting in Glasgow from June 13-16, 2025, included findings from four studies evaluating the medication’s long-term performance. This clinical progress has contributed to BioCryst’s strong market performance, with the stock delivering a 68.07% return over the past year. InvestingPro analysis reveals that 5 analysts have revised their earnings upwards for the upcoming period, suggesting growing confidence in the company’s prospects.

In the pediatric APeX-P study examining children aged 2-11 years, researchers observed an 86% reduction in attacks requiring professional care after 12 weeks of treatment. The mean monthly attack rate decreased from 1.28 during standard care to 0.38 during the first four weeks of berotralstat treatment, with reductions sustained for up to 48 weeks.

The Berolife real-world study in France involving 82 patients showed the median monthly attack rate decreased from 1.0 to 0.44 after six months of treatment, with reductions maintained through 24 months.

Quality of life improvements were documented in pooled data from Phase 3 trials, with 60% of patients receiving berotralstat achieving clinically meaningful improvements at week 24 compared to 52.4% in the placebo group.

A qualitative focus group study with seven French patients reported reduced attack frequency and severity, minimal side effects, and easier incorporation into daily routines compared to previous treatments.

ORLADEYO is an oral therapy designed to prevent HAE attacks by decreasing plasma kallikrein activity. Common adverse reactions included abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease.

The medication is not indicated for treatment of acute HAE attacks, and dosages higher than the recommended 150 mg once daily are not recommended due to potential QT prolongation.

This information is based on a press release statement from BioCryst Pharmaceuticals. With a market capitalization of $2.17 billion and analysts predicting profitability this year, BioCryst shows promising financial health indicators. For deeper insights into BioCryst’s financial metrics and growth potential, including 12 additional ProTips and comprehensive valuation analysis, visit InvestingPro.

In other recent news, BioCryst Pharmaceuticals announced significant developments regarding its hereditary angioedema (HAE) treatment, ORLADEYO (berotralstat). The medication has received approval for reimbursement in the Netherlands, expanding its availability across major European countries. Additionally, Colombia’s National Institute of Drug and Food Surveillance has approved ORLADEYO for preventing HAE attacks in patients aged 12 and older. BioCryst also shared new data demonstrating the drug’s effectiveness in reducing attack rates among adolescents and patients with severe HAE. The findings, presented at the ISPOR conference, showed a substantial decrease in monthly attack rates after starting ORLADEYO. Furthermore, BioCryst has extended its consulting agreement with former CFO Anthony Doyle to ensure continuity in its financial operations. The company continues to focus on bringing ORLADEYO to more patients worldwide and improving the lives of those with rare diseases.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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