The U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) and granted priority review for Lanadelumab (SHP643), an investigational treatment being evaluated for the prevention of angioedema attacks in patients 12 years and older with HAE.
“Every day, patients living with HAE struggle to manage their disease — not knowing when their next attack will occur,” said Andreas Busch, Ph.D., Executive Vice President, Head of Research and Development at Shire. “Lanadelumab if approved will be the first monoclonal antibody for HAE, a serious and potentially life threating disease. Lanadelumab provides a new mechanism of action inhibiting plasma kallikrein for the prevention of HAE attacks. Lanadelumab will offer patients a new option to help control this disease with the potential to change the treatment paradigm. The FDA’s decision underscores Shire’s serial innovation in HAE and commitment to improving treatment options for patients.”
The BLA for Shire’s investigational HAE treatment is supported by data from four clinical trials, including HELP™, the pivotal Phase 3 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).
“Physicians as well as patients in the HAE community are excited to see Lanadelumab moving forward for FDA review because there is now the real possibility of having a new way to prevent HAE attacks,” said Aleena Banerji, M.D., Massachusetts General Hospital, Boston, MA, and clinical trial investigator. “As an investigator, I am appreciative of the HAE patients who participated in the clinical trial to help advance science in a way that may transform the treatment of HAE.”
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 26 August 2018, based on the Prescription Drug User Fee Act V action date.