Shire gets US market approval for rare inflammatory disease drug

pharming ruconest hereditary angioedema

The UK-US biotech giant’s drug Takhzyro has been approved by the FDA to prevent attacks of hereditary angioedema, a rare genetic disease that causes swelling around the body.

Hereditary angioedema is a potentially life threatening genetic condition that people can inherit from either of their parents. Takhzyro (lanadelumab-flyo) is a first-in-class monoclonal antibody drug that stops attacks of swelling in people with the condition by blocking the action of the protein kallikrein, which can trigger inflammation.

The drug was approved through the FDA’s fast-track priority review scheme. This was in response to a recent Phase III trial (the biggest of four trials testing the drug including 125 patients) in which it reduced the number of monthly attacks experienced by those with angioedema by 87% compared with placebo when given by self-administered injection every 2 weeks. It also worked well in patients who only had 1 injection per month, reducing attacks by 73% vs placebo. The drug will continue to be monitored for safety in an open study.

Swollen hand during a hereditary angioedema attack (By LucyHAE)
Swollen hand during a hereditary angioedema attack (By LucyHAE)

Shire, which was acquired by Takeda for £46B (€51B) in May this year, has a focus on hereditary angioedema and already has three drug approvals in this space globally including the bradykinin receptor antagonist Firazyr, another kallikrein inhibitor (with a different mechanism of action) Kalbitor, and C1-inhibitor Cinryze. Dutch biotech Pharming also has a C1 esterase inhibitor Ruconest.

Kallikrein is a relatively recent target for those seeking to develop drugs for the condition and in addition to Shire’s two drugs, British biotech Kalvista is also developing a kallikrein inhibitor for hereditary angioedema. C1 inhibitors are typically blood or animal-product derived and can be difficult to access for patients, which may at least partially explain the appeal of kallikrein inhibitors. Patients with hereditary angioedema also have varying symptoms and so having the option of more than one therapy is appealing from a tailored treatment point of view.

Images: Shutterstock, Lucy HAE

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