FDA Approves Shire’s First-of-Its-Kind Preventive Treatment for Hereditary Angioedema

Rare Daily Staff

The U.S. Food and Drug Administration approved Shire’s Takhzyro to prevent attacks of hereditary angioedema (HAE) in patients 12 years of age and older.

HAE is a rare, genetic and potentially life-threatening disorder that can result in recurrent attacks of edema (swelling) in various parts of the body.

Takhzyro is the only monoclonal antibody that provides targeted inhibition of plasma kallikrein, an enzyme that is chronically uncontrolled in people with HAE, to help prevent attacks.

In the phase 3 study supporting FDA approval, Takhzyro reduced the number of monthly HAE attacks an average of 87 percent vs. placebo when administered at 300 mg every two weeks and 73 percent vs placebo when administered at 300 mg every four weeks.

The most commonly observed adverse reactions associated with Takhzyro were injection site reactions consisting mainly of pain, erythema, and bruising at the injection site; upper respiratory infection; headache; rash; myalgia; dizziness; and diarrhea.

“With the approval of Takhzyro, HAE patients have an innovative treatment that works differently than current options to help prevent attacks,” said Andreas Busch, executive vice president, head of research and development at Shire. “Looking to the future, we continue to work towards our goal of a world in which those living with HAE can aim for zero attacks.”

As a result of Shire’s $5.6 billion acquisition of Dyax’s HAE portfolio in January 2016, the approval of Takhzyro will trigger payment to Dyax shareholders of $4 in cash per contingent value right. One right was issued for each share as part of that transaction.

August 24, 2018
Photo: Andreas Busch, executive vice president, head of research and development at Shire