European regulators will undertake an accelerated assessment of Shire’s hereditary angioedema (HAE) drug lanadelumab.
The firm is developing lanadelumab for the prevention of angioedema attacks in patients with the condition aged 12 years 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.
Lanadelumab is an investigational fully human monoclonal antibody that specifically binds and inhibits plasma kallikrein, thus potentially offering a new treatment approach.
The submission is based on data from the pivotal Phase III HELP study, which showed that subcutaneous administration of 300mg lanadelumab 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.
In this study, no treatment-related serious adverse events or deaths were reported, the most common side effecting observed being injection site pain (29.3 percent placebo versus 42.9 percent across lanadelumab arms).
Accelerated assessments filed under the centralised European procedure are evaluated within 150 days as opposed to the normal 210 days.
The drug has also been given a priority review in the US.