Shots:  

• 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.

­ Ref: PRNewswire | Image: CSL Behring