- The approval is based on P-lll COMPACT pivotal study and OLE study assessing Haegarda (C1 Esterase Inhibitor, SC) vs PBO in patients aged ≤17yrs. with symptomatic HAE.
- Results of COMPACT pivotal study: 95% reduction in the number of HAE attacks, 99% reduction in the use of rescue medication. COMPACT OLE results: all patients experienced a>50% reduction in the number of attacks/ mos. with a 97% reduction in the median number of attacks/ mos. (0.11). All had less than 1 attack/4wks. period and four had >1attack/yr. (one subject was attack free) while no one discontinues the treatment due to AEs
- Haegarda is a self-administered, plasma-derived concentrate of C1-esterase inhibitor and the only SC therapy approved in the US for routine prophylaxis to prevent HAE attack in patients aged ≥6yrs.
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