Shire’s Takhzyro has been approved in the US for the prevention of attacks of hereditary angioedema (HAE) in patients 12 years of age and older.
HAE is a rare, genetic disorder that causes debilitating, painful and sometimes life-threatening swelling in the body, affecting about one in 10,000 to one in 50,000 people worldwide.
Takhzyro (lanadelumab) is an investigational fully human monoclonal antibody that specifically binds and inhibits plasma kallikrein, thus potentially offering a new treatment approach.
Data from the pivotal Phase III HELP study showed that subcutaneous administration of the drug once every two weeks resulted in an 87 percent reduction in the mean frequency of HAE attacks.
In addition, an exploratory endpoint, which will require further confirmatory studies, showed that during the steady state stage of the trial (day 70-182) a 91 percent attack reduction was achieved with eight out of 10 patients reaching an attack free state.
The marketing application for lanadelumab was assigned an accelerated assessment by European regulators back in February.