Shire Plans Lanadelumab BLA Submission Following Positive Phase III HAE Results
Shire said it plans to submit a Biologics License Application (BLA) to the FDA for its long-acting plasma kallikrein-targeting monoclonal antibody lanadelumab by early 2018, on the back of positive data from the Phase III HELP study in patients with hereditary angioedema (HAE).
Lanadelumab (SHP643; formerly DX-2930) is in development for preventing angioedema attacks in HAE patients. The global 26-week placebo-controlled HELP trial involved 152 HAE patients aged 12 years, randomized to receive subcutaneous injections of either placebo or one of two doses of lanadelumab administered every 2 weeks or every 4 weeks. The study met its primary and all secondary endpoints and demonstrated that subcutaneous administration of lanadelumab once every 2 weeks resulted in a statistically significant 87% reduction in the mean frequency of HAE attacks, irrespective of baseline attack rate.
Shire noted that for each of the three lanadelumab regimens studied, whether administration was every 2 weeks or every 4 weeks, significantly higher proportions of lanadelumab-treated patients remained attack-free throughout the entire 26-week period compared with placebo patients. Of the 90% of patients who completed the HELP trial, 96% elected to enroll into the ongoing long-term HELP™ Study Extension long-term safety study.
"If approved, lanadelumab may offer patients a long-acting treatment option that significantly reduces HAE attacks when administered subcutaneously as infrequently as every 4 weeks," commented Aleena Banerji, M.D., clinical trial investigator at the Massachusetts General Hospital. “In the U.S., available treatment options include either injections for acute attacks or short-acting intravenous infusions administered twice a week."
"The possibility of a new way to address the underlying cause of HAE to prevent attacks could transform how we treat the disease in the future," added Prof. Marcus Maurer, M.D., clinical trial investigator at the Charité-Universitätsmedizin in Berlin, Germany. "Patients with HAE want to live independently and without fear of an angioedema attack."
Lanadelumab has been granted orphan drug designation by the FDA and the European Medicines Agency, and breakthrough therapy designation by FDA.