The European Medicines Agency (EMA) has validated its marketing authorization application (MAA) for Lanadelumab (SHP643) from Shire plc – and Health Canada has completed screening and accepted the New Drug Submission (NDS) 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 HAE.
Andreas Busch, Ph.D., 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.”
The EMA MAA 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 (CHMP) 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 NDS for Priority Review shortens the review timeline from 300 to 180 days.
Filings are 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, the largest prevention study in HAE conducted to date, 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 and post-hoc analysis showed that during the steady state stage of the trial (day 70-182) a 91% attack reduction was achieved and nearly 8 out of 10 patients reached 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).
“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.”
Both the EMA MAA validation and Health Canada’s acceptance to review Lanadelumab reinforces Shire’s global leadership in rare disease innovation. In addition to receiving accelerated assessment from EMA and Priority Review from Health Canada, Lanadelumab has received priority review from the U.S. FDA and is expected to provide a decision by 26 August 2018, based on the Prescription Drug User Fee Act V action date. 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.