Shire to Seek FDA Marketing Approval for Lanadelumab as Therapy for Hereditary Angioedema

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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Approval request to FDA

The biopharmaceutical company Shire is expected to soon submit a biologics license application to the U.S. Food and Drug Administration (FDA) seeking marketing approval of its investigational monoclonal antibody lanadelumab as therapy for patients with hereditary angioedema.

The application is supported by positive results from the company’s Phase 3 HELP study (NCT02586805) that evaluated the effectiveness and safety of subcutaneous delivery of lanadelumab in patients 12 and older with hereditary angioedema. The study’s primary goal of determining the rate of investigator-confirmed hereditary angioedema attacks was analyzed after 26 weeks of treatment.

The therapy was administered in three different regimens – 300 mg every 2 weeks, 300 mg every 4 weeks or 150 mg every 4 weeks – and compared to a placebo control group.

The results showed that the trial achieved its primary and secondary endpoints in all three lanadelumab treatment arms compared to placebo.

Patients treated with 300 mg dose administered once every two weeks had a noteworthy 87% reduction in mean hereditary angioedema attacks compared to placebo controls. The reduction was also statistically significant when lanadelumab was administered monthly.

Lanadelumab was well-tolerated over the treatment period with no cases of therapy-induced serious adverse events. The most common adverse event was injection site pain (29.3% in the placebo group vs. 42.9 % in the combined lanadelumab groups).

Nearly all patients who completed the HELP study (96%) have enrolled in an ongoing, long-term HELP extension follow-up study (NCT02741596). This non-randomized, open-label trial will evaluate the long-term safety of repeated subcutaneous administration of lanadelumab for 14 months.

Lanadelumab is an investigational, fully human monoclonal antibody that works by specifically binding and inhibiting the plasma kallikrein. High levels of this protein are the underlying cause of hereditary angioedema.

“If approved, lanadelumab may offer patients a long-acting treatment option that significantly reduces HAE attacks when administered subcutaneously as infrequently as every 4 weeks,” Aleena Banerji, MD, clinical trial investigator at Massachusetts General Hospital, said in a press release.

“In the U.S., available treatment options include either injections for acute attacks or short-acting intravenous infusions administered twice a week,” Banerji added.

“The possibility of a new way to address the underlying cause of HAE to prevent attacks could transform how we treat the disease in the future,” said Marcus Maurer, MD, clinical trial investigator at the Charité-Universitätsmedizin in Berlin, Germany. “Patients with HAE want to live independently and without fear of an angioedema attack.”

Lanadelumab has previously received orphan drug designation from the FDA and the European Medicines Agency, and breakthrough therapy designation from the FDA.