EU regulators have validated Shire’s application to market its experimental hereditary angioedema (HAE) drug lanadelumab, indicating that there is sufficient data to begin a priority review.
The firm is developing lanadelumab for the prevention of angioedema attacks in patients with the condition aged 12 years and older.
HAE is a rare, genetic disorder that causes debilitating, painful and sometimes life-threatening swelling in the body, affecting about one in 10,000 to one in 50,000 people worldwide.
Lanadelumab is an investigational fully human monoclonal antibody that specifically binds and inhibits plasma kallikrein, thus potentially offering a new treatment approach.
“HAE presents a significant burden on the lives of patients whose recurring attacks of swelling can be debilitating and painful,” said Andreas Busch, head of Research and Development at 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 submission is based on data from the pivotal Phase III HELP study, which showed that subcutaneous administration of 300mg 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 eight out of 10 patients reaching an attack free state.
“As a physician treating patients with HAE, I would welcome new treatment options to help prevent attacks, as it is important to recognize the impact HAE can have on the quality of life of these individuals,” said Marcus Maurer, Prof. Dr. Med., Department of Dermatology and Allergy, Charité – Universitätsmedizin Berlin, Germany and clinical trial investigator.
“I am pleased to see the progress in the review of lanadelumab, that if approved, would offer a targeted mechanism of action inhibiting plasma kallikrein.”