BLA for Novel Hereditary Angioedema Treatment Accepted by FDA

If approved, it would be the first monoclonal antibody for hereditary angioedema.
If approved, it would be the first monoclonal antibody for hereditary angioedema.

The Biologics License Application (BLA) for lanadelumab (SHP643; Shire) – an investigational treatment for the prevention of hereditary angioedema (HAE) attacks – has been accepted by the Food and Drug Administration (FDA).

Lanadelumab, a fully human monoclonal antibody that specifically binds and inhibits plasma kallikrein, is intended for patients aged ≥12 years. If approved, it would be the first long-acting monoclonal antibody for HAE. The BLA submission is supported by data from the Phase 3 HELP study (N=125) and interim data from its extension study. 

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Results showed that those who received a subcutaneous (SC) administration of lanadelumab 300mg once every 2 weeks had an 87% reduction in mean frequency of HAE attacks. An exploratory endpoint further demonstrated a 91% attack reduction during the steady state stage (Days 70 to 182) of which 8/10 patients achieved an attack-free state. 

No treatment-related serious adverse events or deaths were noted during the trial, however there was a higher rate of injection site pain seen in the combined lanadelumab arms vs placebo (42.9% vs 29.3%).

The FDA Prescription Drug User Free Act (PDUFA) action date for a decision on lanadelumab has been set for August 26, 2018.

For more information call (800) 536-7878 or visit Shire.com.