EMA Ready to Review Shire’s HAE Therapy for Approval

Rare Daily Staff

Shire said that the European Medicines Agency has validated its marketing authorization application for lanadelumab, the company’s experimental therapy to treat hereditary angioedema, a rare, genetic disorder that causes debilitating, painful, and sometimes life-threatening swelling in the body.

The company also reported that Health Canada has completed screening and accepted Shire’s New Drug Submission under Priority Review for the drug.

Lanadelumab is an experimental treatment being evaluated for the prevention of angioedema attacks in patients 12 years and older with HAE.

“HAE presents a significant burden on the lives of patients whose recurring attacks of swelling can be debilitating and painful,” said Andreas Busch, executive vice president and head of research and development for Shire. “Lanadelumab is the first monoclonal antibody under evaluation to prevent HAE attacks and has the potential to change the treatment paradigm for this rare disease, if approved.”

The EMA validation confirms the submission for lanadelumab is sufficiently complete and an accelerated assessment for the potential therapy will begin. In February 2018, the Committee for Medicinal Products for Human Use of the EMA granted an accelerated assessment for lanadelumab reducing the number of evaluation days required from 210 to 150. Similarly, Health Canada’s recent acceptance of the lanadelumab New Drug Submission for Priority Review shortens the review timeline from 300 to 180 days.

The filings are supported by data from four clinical trials including a pivotal late-stage efficacy and safety study, along with interim data from its extension study. The pivotal 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 and post-hoc analysis showed that during the steady state stage of the trial (day 70-182) a 91 percent attack reduction was achieved, and nearly 8 out of 10 patients reached an attack-free state.

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

March 29, 2018
Photo: Andreas Busch, executive vice president and head of research and development for Shire