The application requesting that lanadelumab be reviewed with priority as a new treatment for the prevention of angioedema attacks has been accepted by Health Canada, the medicine’s manufacturer, Shire, announced.
The New Drug Submission (NDS) is requesting the lanadelumab’s approval for patients older than 12 years, who live with hereditary angioedema (HAE). In an update on its key pipeline programs in rare diseases, Shire said that a decision is expected by the second half of 2018.
HAE is a rare genetic disorder in which recurring attacks of edema (swelling) affect several parts of the body, including the abdomen, face, hands and other body parts. The swelling can be painful and impair daily life activities.
Lanadelumab (SHP643) is an antibody designed to bind and inhibit plasma kallikrein. High levels of this protein are the underlying cause of HAE.
Lanadelumab is being studied as a potential treatment for the prevention of angioedema attacks in patients older than 12 with HAE. The therapy is formulated for subcutaneous administration and has a half-life of about 14 days in patients with HAE.
Priority review status is granted to drug therapies aiming to provide an effective treatment for serious conditions for which there are no drugs currently marketed in Canada.
Earlier this month, Shire also submitted a biologics license application (BLA) to the U.S. Food and Drug Administration (FDA) seeking marketing approval of lanadelumab as a therapy for HAE.
Both applications were supported by positive results from the HELP Phase 3 study (NCT02586805), which evaluated the effectiveness and safety of subcutaneous lanadelumab in patients 12 and older with HAE.
Results from the trial show that lanadelumab achieved its primary and secondary endpoints in all three treatment arms, compared to placebo. In fact, patients treated with a 300 mg dose once every two weeks, had a reduction of 87 percent in mean HAE attacks, compared to placebo controls. The reduction also was statistically significant when lanadelumab was administered monthly.
The investigational therapy candidate also was well-tolerated over the treatment period and the most common adverse event was pain in the injection site.
Nearly all patients who completed the HELP study (96 percent) also enrolled in a now ongoing, long-term HELP extension follow-up study (NCT02741596) to evaluate the long-term safety of repeated subcutaneous administration of lanadelumab for 14 months.
Lanadelumab received orphan drug status from the FDA in 2013 and the European Medicines Agency (EMA) in 2015, as well as breakthrough therapy designation from the FDA that same year.