FDA accepts BLA for SHP 643 to treat hereditary angioedema

Shire plc announced the FDA accepted the Biologics License Application (BLA) and granted priority review for lanadelumab (SHP 643). Lanadelumab is an investigational treatment being evaluated for the prevention of angioedema attacks in patients 12 years and older with the rare, genetic disorder, hereditary angioedema (HAE).

The burden of HAE on patients is significant. On average, HAE patients take 20 days away from school or off from work per year. HAE results in recurring attacks of edema (swelling) that can be debilitating and painful in various parts of the body, including the abdomen, face, feet, genitals, hands and throat. Attacks that obstruct the airways (asphyxiation) are potentially life-threatening.

The BLA for Shire's investigational HAE treatment is 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 is the largest prevention study in HAE conducted to date, and 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, which will require further confirmatory studies, showed that during the steady state stage of the trial (day 70-182) a 91% attack reduction was achieved with 8 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 adverse event was injection site pain (29.3% placebo vs. 42.9% combined lanadelumab arms).

The FDA grants Priority Review designation to drugs that have the potential to provide significant improvements in the safety or effectiveness for the treatment, diagnosis or prevention of a serious disease. Drugs with Priority Review designation have an accelerated review target of eight months, instead of the standard of 12 months. The FDA is expected to provide a decision on lanadelumab by August 26, 2018, based on the Prescription Drug User Fee Act V action date.