EMA and Health Canada accept marketing applications for SHP 643 for the prevention of angioedema attacks .- Shire plc.

Shire plc announced that the European Medicines Agency (EMA) has validated its marketing authorization application (MAA) for lanadelumab (SHP 643) and also reports that Health Canada has completed screening and accepted the New Drug Submission (NDS) under Priority Review for this investigational compound. Lanadelumab is an investigational treatment being evaluated for the prevention of angioedema attacks in patients 12 years and older with hereditary angioedema (HAE), a rare, genetic disorder estimated to affect about 1 in 10,000 to 1 in 50,000 people worldwide.

Filings are supported by data from four clinical trials, including HELP, the pivotal Phase III efficacy and safety study, along with interim data from its extension study. HELP, the largest prevention study in HAE conducted to date, enrolled a total of 125 patients aged 12 years and over with type I/II HAE. The HELP study demonstrated that subcutaneous administration of 300 mg lanadelumab once every two weeks resulted in an 87% reduction in the mean frequency of HAE attacks. In addition, an exploratory endpoint and post-hoc analysis showed that during the steady state stage of the trial (day 70-182) a 91% attack reduction was achieved and nearly 8 out of 10 patients reached an attack free state. In this study, no treatment-related serious adverse events or deaths were reported. The most common adverse event was injection site pain (29.3% placebo vs. 42.9 % combined lanadelumab arms). .