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

Claire Richmond avatar

by Claire Richmond |

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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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Givlaari Beyond the Trials: Real-world Patient Experiences

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.

I’ve noticed that several members of the patient community seem hesitant to speak openly about their disappointment in outcomes after being treated with Givlaari. I’m not sure where this hesitation is coming from. Is there concern about dissuading others from trying the medication? Disappointment it didn’t work? Fear that healthcare providers won’t believe them?

After I spoke up about having continued attacks while taking Givlaari, a handful of people reached out to share their own experiences. They’ve each expressed their permission for me to share their feedback.

One woman messaged me to say she was diagnosed with AHP last year, and is eight months into a Givlaari regimen. Overall, she says she feels better, but still takes time off work to manage symptoms every month. She feels ill after the injection, and her porphyria symptoms begin to ramp up between doses, near the end of the four weeks.

Another texted with similar concerns. Her doctor is experimenting with dosing to prevent a drop-off in medication effectiveness after three weeks. The unpredictability of the disease continues to cause her distress, especially when it comes to travel and making plans. She ends up canceling often due to attack symptoms.

Recently, I was texting with a friend, and we began swapping treatment stories. She told me it’s been a year and a half since she began treatment with Givlaari, and she isn’t sure it’s helping. She talked to her doctor about discontinuing the treatment, but has decided to stay on it for now. She’s concerned about a lack of long-term data about Givlaari’s effectiveness.

As much as I wish each of these women living with AHP had benefited more from Givlaari, it was nice to know that I wasn’t alone in my uncertainty about whether it’s making a difference with my symptoms. Maybe over a longer period of time, it will have a greater effect.

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.

Comments

Thomas Stauch avatar

Thomas Stauch

Dear Claire,
thank you for sharing your experiences with siRNA (Givosiran) for AHP.
Since your "porphobilinogen (PBG) level remains elevated", as you say, the medication does not seem to reach the place of impact (the ALAS1 enzyme) in the liver. So maybe the internalisation of the agent into the liver cells might be blocked somehow. Did your therapists check for antibodies against asialoglycoprotein receptor (sometimes also called liver membrane antibodies)? These autoantibodies (sometimes appearing without clinical symptoms) could avert the uptake of Givosiran in the liver cell, so that the drug targeting is disturbed. Consequently, the enzyme activity will not be suppressed adequately and porphyrin precursors can still be produced (
Best regards
Thomas

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