Givlaari Has Reduced Acute Porphyria Attacks for Many — But Not Me

Note: This column describes the author’s own experiences with Givlaari. Not everybody will have the same response to treatment. Consult your doctor before starting or stopping any therapy.

I used to believe in a wonder drug for my rare disease. And I wasn’t alone in telling myself I’d be saved by a new treatment option for acute hepatic porphyria (AHP).

In 2019, the U.S. Food and Drug Administration approved Givlaari (givosiran), a monthly injection intended to prevent acute porphyria attacks, for adults with AHP, after it proved highly effective in clinical trials. On social media and in online forums, I read the accounts of many people living with AHP who said they had made miraculous recoveries — reclaiming their lives in the process, taking vacations, going back to school, restarting their careers, and planning for families.

I didn’t know if the medication would work as well for me as it did for others, but I badly wanted my own story of restitution. I became preoccupied with thoughts of how this innovative therapy might change my life.

Last August, I decided to start the shots. And then I waited, anticipating improvements. It’s now been 11 months. In that time, I’ve experienced seven severe attacks, four emergency room visits, and one hospitalization. My porphobilinogen (PBG) level remains elevated, and I continue managing twice-monthly flares. It’s not the response I expected based on published reports of significantly improved attack rates.

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That doesn’t mean I’m ungrateful for the additional AHP treatment and the brilliant minds who developed it. Our rare disease community is fortunate, as are the many patients who’ve benefited from Givlaari. However, in the midst of these success stories, I found it important to temper my own expectations while my story is written.

In the face of constant and recurring extreme pain, it’s natural to seek an escape, and this drug has proven to be helpful in preventing acute attacks for many. I’m ready to admit that I got caught up in the excitement and bought into the idea of overcoming my disability. For anyone feeling disappointed by their response to treatment, it’s good to remember that it was never meant to be a miraculous cure. It was developed as a preventive medication that helped curb attack rates in clinical trials.

Maybe it’s naive to believe there could be a one-size-fits-all solution for everybody. What I know for sure is that Givlaari is promising, and it represents the start of something important for our community.

Note: Porphyria 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 Porphyria News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to porphyria.