I have mixed feelings about a potential gene-editing therapy for HAE

Note: This column describes the author’s own experiences with various angioedema medications and thoughts about gene therapy. Not everyone will have the same response to treatment. Consult your doctor before starting or stopping a therapy.

Gene therapy is a hot topic in medicine these days. It can be exciting as a potential treatment or cure, but venturing into the unknown is also a bit scary.

Angioedema News recently reported that the U.S. Food and Drug Administration (FDA) will decide by March 2027 whether to approve a one-time gene-editing therapy called lonvoguran ziclumeran (lonvo-z) for hereditary angioedema (HAE). My initial reaction was “Wow!” But after all my medication trials and tribulations, I am also concerned.

Earlier this year, I tried a new HAE maintenance medication that was recommended by my doctor, but unfortunately, I did not respond well to it. It left me with more symptoms and HAE attacks than usual this summer.

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Other physicians have suggested intravenous immunoglobulin therapy for my chronic inflammatory demyelinating polyneuropathy and probable Sjögren’s disease, but I am not keen to try it, given the possible flu-like side effects. My mental and physical stamina have already decreased significantly over the past year, so I’m hesitant to try any medication that could potentially increase my fatigue.

After experiencing significant side effects from the new HAE medication I tried this summer, I inquired with the manufacturer and was told that those symptoms had been noted in patients with HAE type 3, also called HAE with normal C1-INH, which I have. However, while the medication is approved for HAE patients ages 12 and older, the approval was based on a Phase 3 clinical trial that only involved people with HAE types 1 and 2.

Therefore, I was curious about which types of HAE have been included in the testing of lonvo-z. The Phase 3 HAELO trial, which is the primary basis of the drug approval application, enrolled only people with HAE types 1 and 2.

That could certainly help my adopted daughter, Leah, who has HAE type 2, and my son Dorian, who’s been diagnosed with HAE type 1. But would it help those of us with HAE type 3, or other types of angioedema? We may have to wait for additional testing and trials.

Results from the HAELO trial showed that lonvo-z reduced swelling attack rates by 87%, and 62% of patients who received the gene therapy remained attack-free for six months. Additionally, none of the participants given lonvo-z have needed further preventive treatment as of the latest follow-up. These results are amazing!

The most common side effects reported were infusion-related reactions, which, for a one-time therapy, I would be totally prepared to experience. I am anxiously awaiting the FDA’s decision.

I would love to hear your thoughts and concerns about this topic. Let’s chat about it on the new Angioedema News Forums.

Note: Angioedema News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Angioedema News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to angioedema.