The Swiss Agency for Therapeutic Products (Swissmedic) has validated the marketing authorization application (MAA) for Lanadelumab (SHP643). The validation of the MAA confirms that the Lanadelumab MAA submission is complete and that the formal review process will begin. Lanadelumab is an investigational treatment being evaluated for the prevention of angioedema attacks in patients 12 years and older with HAE. Lanadelumab was also designated orphan drug status by Swissmedic.
Andreas Busch, Ph.D., Executive Vice President, Head of Research and Development at Shire plc said, “Today’s announcement represents another important step forward as we continue our work to make Lanadelumab available to the global HAE community. For those living with HAE, the recurring attacks of swelling can be debilitating. Lanadelumab, if approved, has the potential to change the HAE treatment landscape by directly targeting plasma kallikrein to inhibit excessive bradykinin formation, which stops the blood vessel permeability that causes these swelling attacks.”
Lanadelumab Regulatory Status
- U.S. Food and Drug Administration (FDA) accepted Shire’s biologics license application (BLA) and granted priority review for Lanadelumab in February 2018. The FDA is expected to provide a decision on Lanadelumab by 26 August 2018, based on the Prescription Drug User Fee Act V action date.
- European Medicines Agency (EMA) validated the marketing authorization application for Lanadelumab in March 2018. EMA had previously granted Lanadelumab an accelerated assessment reducing the number of evaluation days required, from 210 to 150.
- Health Canada accepted the New Drug Submission (NDS) for Lanadelumab under Priority Review in March 2018 shortening the review timeline from 300 to 180 days.
- Therapeutic Goods Administration in Australia granted Lanadelumab priority review and orphan drug designation in February 2018.
Regulatory 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 ongoing 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 versus placebo. In addition, an exploratory endpoint and post-hoc analysis which would require confirmatory studies, showed that during the steady state stage of the trial (day 70-182), a 91% attack reduction was achieved versus placebo, 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 commonly reported treatment-related adverse events in patients treated with Lanadelumab during the entire treatment period were injection site pain (29.3% placebo vs. 42.9 % combined Lanadelumab arms), viral upper respiratory tract infection, headache, injection site erythema, injection site bruising, and dizziness. Most adverse events were mild to moderate in severity.