FDA Accepts Shire’s Application for HAE Therapeutic, Grants Priority Review

Rare Daily Staff

Shire said the U.S. Food and Drug Administration accepted its application and granted priority review to lanadelumab, an experimental monoclonal antibody to treat the prevention of angioedema attacks in patients 12 years and older with the rare, genetic disorder hereditary angioedema.

Hereditary angioedema, or HAE, causes debilitating, painful and sometimes life-threatening swelling in the body. It is characterized by spontaneous and recurrent episodes of swelling of the skin in different parts of the body, as well as in the airways and internal organs. Almost all HAE patients suffer from bouts of severe abdominal pain, nausea, vomiting, and diarrhea caused by swelling of the intestinal wall.

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.

 “Every day, patients living with HAE struggle to manage their disease—not knowing when their next attack will occur,” said Andreas Busch, executive vice president and head of research and development at Shire. “Lanadelumab will offer patients a new option to help control this disease with the potential to change the treatment paradigm.

The application for Shire’s experimental HAE treatment is supported by data from four clinical trials. The pivotal late-stage study enrolled a total of 125 patients aged 12 years and over with type I/II HAE. The study demonstrated that subcutaneous administration of 300 mg 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 8 out of 10 patients reaching an attack free state.

There were no treatment-related serious adverse events or deaths reported. The most common adverse event was injection site pain.

Febraury 23, 2018
Photo: Andreas Busch, executive vice president and head of research and development at Shire.