FDA Approves First Oral Preventive Treatment for Pediatric HAE, With Jolanta Bernatoniene, PhD скачать в хорошем качестве

FDA Approves First Oral Preventive Treatment for Pediatric HAE, With Jolanta Bernatoniene, PhD

The US Food & Drug Administration (FDA) has approved berotralstat for long-term prophylaxis of hereditary angioedema (HAE) in children aged 2 to 11 years, making it the first and only oral preventive therapy available for this pediatric population. In this interview, Bernatoniene discusses why this approval represents a major advance in pediatric HAE care, addressing long-standing gaps created by injectable-only treatment options. She explains how berotralstat’s once-daily oral formulation, administered as small pellets that can be sprinkled on food, reduces treatment burden for children, families, and clinicians. Bernatoniene also reviews key efficacy and safety findings from the APeX-P trial, including sustained reductions in HAE attack rates, early onset of benefit, and reassuring cardiovascular safety data. The conversation explores how this approval may influence clinical practice, guideline updates, and long-term prophylactic strategies for younger patients, as well as ongoing studies evaluating long-term safety. Key Highlights: 00:07: Significance of the berotralstat approval for pediatric HAE 02:38: Significant efficacy findings from the APeX-Trial 03:32: Monitoring PT prolongation risk at doses above 150 mg 06:27: Ongoing or planned trials on berotralstat Read more about the approval here: https://www.hcplive.com/view/fda-appr... More allergy coverage: https://www.hcplive.com/clinical/allergy #FDA #Berotralstat #HAE #PediatricHAE #HereditaryAngiodema #RareDisease #Prophylaxis