Shire is asking for the therapy’s approval for patients aged 12 and older.
Priority review means the FDA must make its final decision on Shire’s Biologics License Application in eight months, instead of the standard 12. A decision is now expected by August 2018.
Hereditary angioedema, or HAE, is a rare genetic disorder that causes debilitating and sometimes life-threatening swelling in the body. These edema attacks can affect the abdomen, face, feet, genitals, hands and throat. On average, patients must take 20 days away from school or work per year to deal with the attacks.
Lanadelumab is an antibody designed to bind to and inhibit plasma kallikrein. High levels of this protein are the underlying cause of HAE.
“Lanadelumab, if approved, will be the first monoclonal antibody for HAE, a serious and potentially life-threatening disease,” Andreas Busch, the executive vice president of Shire, said in a press release. “Lanadelumab provides a new mechanism of action inhibiting plasma kallikrein for the prevention of HAE attacks. Lanadelumab will offer patients a new option to help control this disease with the potential to change the treatment paradigm.”
Lanadelumab’s application is supported by data from four clinical trials, including the Phase 3 HELP study (NCT02586805), which evaluated the effectiveness and safety of subcutaneous lanadelumab.
HELP was the largest prevention study in HAE. It included 125 patients aged 12 and older with types 1 and 2 HAE.
Trial results show that lanadelumab achieved its primary and secondary endpoints in all three treatment arms, compared with a placebo. Patients who received a 300-mg dose once every two weeks saw a mean reduction of 87 percent of their HAE attacks, compared with the placebo group.
In addition, an exploratory endpoint seemed to show that during the steady stage of the trial, a 91 percent attack reduction was achieved, with eight out of 10 patients reaching an attack-free state.
The therapy was also well-tolerated over the treatment period. The most common adverse event was pain at the injection site.
The Biologics License Application also included interim data from HELP’s extension study (NCT02741596). It covered 96 percent of the patients who completed the HELP study. The goal was to evaluate the long-term safety of repeated subcutaneous administration of lanadelumab over 14 months.
“Physicians as well as patients in the HAE community are excited to see lanadelumab moving forward for FDA review because there is now the real possibility of having a new way to prevent HAE attacks,” said trial investigator Aleena Banerji. “As an investigator, I am appreciative of the HAE patients who participated in the clinical trial to help advance science in a way that may transform the treatment of HAE.”
The FDA granted orphan drug status to lanadelumab in 2013, and the European Medicines Agency (EMA) in 2015. Lanadelumab has also received a breakthrough therapy designation from the FDA.
In February of this year, Health Canada granted priority review of a New Drug Submission requesting lanadelumab’s approval for patients older than 12 years.