European Medicines Agency (EMA) has validated Shire’s marketing authorization application (MAA) for its lanadelumab, which was also accepted by Health Canada after it’s completed screening under Priority Review for this investigational compound.
Lanadelumab is an investigational treatment being evaluated for the prevention of angioedema attacks in patients 12 years and older with hereditary angioedema (HAE), a rare, genetic disorder estimated to affect about 1 in 10,000 to 1 in 50,000 people worldwide.
“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, 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. We look forward to working with regulatory bodies to bring a new treatment option to HAE patients.”
“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., Allergy specialist, in Germany, and a 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.”
Shire said that lanadelumab has also received priority review from the U.S. FDA and is expected to provide a decision by August 26, 2018. Lanadelumab has also received priority review and orphan drug designation from the Therapeutic Goods Administration in Australia. In 2017 alone, Shire received breakthrough therapy, orphan drug or fast track designations by the FDA for five of its rare disease therapies and anticipates continued progress of key regulatory milestones for late-stage programs.
Image: A sign sits in front of Shire’s manufacturing facility in Lexington, Massachusetts July 18, 2014. REUTERS/Brian Snyder