- In January 2024, a groundbreaking gene therapy trial demonstrated remarkable success in treating hereditary angioedema (HAE), offering hope for a potential cure. Utilizing CRISPR technology, the therapy targets the kallikrein gene responsible for HAE attacks. Participants in the phase-one trial experienced significant symptom relief, with many remaining attack-free for 18 months post-treatment. This advancement underscores the transformative potential of gene editing in managing rare genetic disorders
- In August 2023, Takeda Pharmaceutical Company announced that the U.S. FDA approved an expanded indication for TAKHZYRO (lanadelumab-flyo), allowing its use in children aged 2 years and older for the prevention of HAE attacks. This approval marks a significant step in pediatric HAE management, providing younger patients with access to a proven prophylactic treatment
- In November 2023, BioCryst Pharmaceuticals presented new real-world data highlighting the efficacy of ORLADEYO (berotralstat) in reducing HAE attack rates among patients with normal C1-inhibitor levels. The findings revealed a median attack rate of zero over a 12-month period, emphasizing ORLADEYO's potential as a reliable prophylactic option for this patient subgroup
- In April 2023, BioCryst Pharmaceuticals shared long-term data from the APeX-S clinical trial, demonstrating that patients treated with ORLADEYO maintained consistently high attack-free status over 96 weeks. The study encompassed diverse patient demographics, reinforcing the drug's broad applicability and sustained effectiveness in HAE prophylaxis.
- In February 2025, BioCryst Pharmaceuticals announced positive interim results from the APeX-P trial, evaluating ORLADEYO in pediatric patients aged 2 to <12 years. The oral granule formulation was well-tolerated and led to early and sustained reductions in monthly HAE attack rates, highlighting its promise as a pediatric-friendly prophylactic treatment
