Kallidinogenase

The FDA has approved novel therapies for Duchenne Muscular Dystrophy, including ITF Therapeutics' Duvyzat (givinostat), which targets histone deacetylases to potentially slow muscle deterioration.

Astria Therapeutics is initiating the ALPHA-ORBIT Phase 3 trial to evaluate navenibart for hereditary angioedema (HAE) with flexible dosing.

2024 marked significant advances in hereditary angioedema (HAE) treatment, with Intellia's gene-editing therapy NTLA-2002 showing a remarkable 95% reduction in monthly attacks. The year also saw important developments in multiple therapeutic candidates, including donidalorsen and garadacimab, while established treatments like Takhzyro demonstrated continued efficacy in adolescent populations.

The European Commission and the MHRA have approved CSL's Andembry (garadacimab) for preventing hereditary angioedema (HAE) attacks in patients aged 12 and older.

Astria Therapeutics' navenibart demonstrated a 90-95% reduction in mean monthly attack rates in HAE patients over six months.