BioCryst announces new data on efficacy, safety of ORLADEYO

BioCryst (BCRX) Pharmaceuticals announced new data on the long-term efficacy and safety of ORLADEYO for the prophylactic treatment of hereditary angioedema, HAE, in patients across all age groups. Title: “Berotralstat Use Reduced Number of HAE Attacks Requiring Treatment or Professional Care in Pediatric Patients: Interim Results from APeX-P.” Key results: Eighty-six percent reduction in attacks requiring professional care: attacks dropped from 22 during the 12-week SOC period to three following 12 weeks of berotralstat treatment; Early/rapid and sustained reduction in the rate of HAE attacks requiring on-demand treatment; Berotralstat was well tolerated across all cohorts; the most common treatment-emergent adverse events were nasopharyngitis, upper respiratory tract infection, and headache. Title: Assessment of the Effectiveness and Tolerability of Berotralstat for Long-term Prophylaxis in Hereditary Angioedema: Findings from the Berolife Study. Key results: Significant reduction in monthly HAE attacks was observed at six months; Berotralstat was well tolerated throughout the study period; Adverse events were consistent with previous clinical trial data. Title: Impact of Berotralstat on Quality of Life in Patients with Hereditary Angioedema. Key results: Significant improvements in AE-QoL total and domain scores were observed with berotralstat versus placebo at week 24. Benefits sustained through week 96, indicating lasting improvement in daily life; At week 24, 60 percent of patients receiving berotralstat achieved the Minimal Clinically Important Difference in AE-QoL total score, compared to 52.4 percent in the placebo group. Title: Patients With HAE Report Positive Perceptions Following Berotralstat Treatment: Results from a Focus Group. Key findings: Participants reported notable improvements in disease control, including reduced frequency and severity of HAE attacks; Minimal side effects were observed; Berotralstat was described as less burdensome and easier to incorporate into daily routines compared to previous therapies