There’s some good news for the hereditary angioedema patient community this week!
Shire, a global biotechnology company focused on rare diseases, announced in the Journal of the American Medical Association (JAMA) publication its results from the Phase 3 HELP Study for its drug TAKHZYRO (lanadelumab), showing statistically significant reductions in monthly Hereditary Angioedema (HAE).
What is Hereditary Angioedema (HAE)?
“Angio” means pertaining to blood vessels, and “edema” is swelling caused by excess fluid trapped in the body’s tissues. People who have hereditary angioedema have a genetic defect on chromosome 11 that results in recurring attacks of unexplained, severe swelling underneath the skin.
Many things can trigger these attacks, including stress and physical injuries, but the swelling often occurs without a known trigger.
Click here to learn more about HAE!
A Clinically Meaningful Improvement
TAKHZYRO is a first-of-its-kind antibody that inhibits the activity of plasma kallikrein, an enzyme which is uncontrolled in people with HAE, to help prevent attacks.
“HAE can be an unpredictable disease which requires an individualised approach to treatment,” said Aleena Banerji, MD, Massachusetts General Hospital, Boston, Mass. and principal investigator of the clinical trial. “The findings from HELP support the use of lanadelumab as a subcutaneous prophylactic therapy to prevent HAE attacks in appropriate patients, helping address the need for new treatment options.”
The study met all primary and secondary endpoints, with all three treatment regimens demonstrating statistically significant results when compared to placebos.
“We are excited about the potential of lanadelumab.” said Donatello Crocetta, Head, Global Medical Affairs, Immunology at Shire. “Data from HELP demonstrate the efficacy of lanadelumab in preventing HAE attacks over the entire duration of the study, with many patients remaining attack-free during the 16-week steady state period. We remain focused on our work to help make lanadelumab available to patients living with HAE in additional countries around the world.”
Music to the ears of those affected by HAE!
As TAKHZYRO moves up the ladder to approval, these results are promising and gives hope to effective treatment for the near future.
