BCRX
RESEARCH TRIANGLE PARK - BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced the presentation of six posters, including the first interim real-word evidence from the APeX-N trial, and new data highlighting the value of shared decision making (SDM) between healthcare providers (HCPs) and their hereditary angioedema (HAE) patients to provide optimal patient outcomes.
The company is presenting the posters at the 7th Bradykinin Symposium in Berlin from September 5-6, 2024.
APeX-N interim results: Oral berotralstat for HAE prophylaxis in Europe
HCP and patient perspectives: HAE long-term prophylaxis and shared decision-making
Adverse health outcomes and perspectives of androgen use in HAE
Evaluation of adherence to berotralstat in patients with hereditary angioedema
Tolerability and effectiveness of berotralstat for long-term prophylaxis in HAE
Effectiveness and safety of berotralstat in HAE with normal C1-inhibitor
APeX-N interim results
APeX-N is a European multi-center observational study assessing the safety (primary objective), effectiveness and quality of life (secondary objectives) of berotralstat 150 mg in routine clinical use. This interim analysis included 56 patients from the United Kingdom, France, Germany and Sweden.
Non-serious gastrointestinal adverse events were reported in 12.5 percent of patients. Seven percent (n=4) of patients discontinued treatment (three due to unsatisfactory response, one to participate in a clinical trial). One patient had a severe HAE attack but continued treatment.
'These initial data from APeX-N in Europe reinforce and closely replicate the clinical trial and real-world evidence of berotralstat as the first oral prophylaxis for HAE,' said Dr. Sorena Kiani, consultant immunologist at Royal Free London NHS Foundation.
HCP and patient perspectives: HAE long-term prophylaxis and shared decision-making
This study, conducted in Germany, explored the dynamics between HCPs and patients in HAE management, identifying barriers to SDM and strategies to improve it. Ten HCPs participated in 60 minute interviews and simulated patient consultations. Eight HAE patients participated in 30 minute interviews. Participants then convened in structured focus groups to discuss their findings.
The participants identified a need for enhanced HCP awareness of patient perspectives, more comprehensive HCP-patient conversations and improved education about HAE treatment management.
'The findings from this study support the need for further insights to develop future guidance and HAE management strategies to facilitate successful shared decision making and improved patient quality of life,' said Dr. Emel Aygoren-Pursun, department for children and adolescents, University Hospital Frankfurt, Germany.
About ORLADEYO (berotralstat)
ORLADEYO (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years and older. One capsule of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein.
U.S. Indication and Important Safety Information
INDICATION
ORLADEYO (berotralstat) is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older.
Limitations of use
The safety and effectiveness of ORLADEYO for the treatment of acute HAE attacks have not been established. ORLADEYO should not be used for the treatment of acute HAE attacks. Additional doses or dosages of ORLADEYO higher than 150 mg once daily are not recommended due to the potential for QT prolongation.
IMPORTANT SAFETY INFORMATION
An increase in QT prolongation was observed at dosages higher than the recommended 150 mg once-daily dosage and was concentration dependent.
The most common adverse reactions (10% and higher than placebo) in patients receiving ORLADEYO were abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease.
A reduced dosage of 110 mg taken orally once daily with food is recommended in patients with moderate or severe hepatic impairment (Child-Pugh B or C).
Berotralstat is a substrate of P-glycoprotein (P-gp) and breast cancer resistance protein. P-gp inducers (eg, rifampin, St. John's wort) may decrease berotralstat plasma concentration, leading to reduced efficacy of ORLADEYO. The use of P-gp inducers is not recommended with ORLADEYO.
ORLADEYO at a dose of 150 mg is a moderate inhibitor of CYP2D6 and CYP3A4. For concomitant medications with a narrow therapeutic index that are predominantly metabolized by CYP2D6 or CYP3A4, appropriate monitoring and dose titration is recommended. ORLADEYO at a dose of 300 mg is a P-gp inhibitor. Appropriate monitoring and dose titration is recommended for P-gp substrates (eg, digoxin) when coadministering with ORLADEYO.
The safety and effectiveness of ORLADEYO in pediatric patients
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