Reflections – The Day After

Hi, All

Yesterday – 15 November 2021 – I received my first infusion of Nexviazyme – the recently-approved Enzyme Replacement Therapy (ERT) from Sanofi-Genzyme.

This followed nine years of Lumizyme infusions (also a product of Sanofi-Genzyme).

This milestone transition followed over two years of discussions, evaluations, research, negotiation, and a whole new battery of baseline studies in preparation to move on from Lumizyme. This long process included me abandoning my former Pompe specialist and finding a new one who would be more responsive to my changing condition and needs.

In late 2018 – early 2019, I began to plateau on the beneficial effects of Lumizyme, a fairly common experience among Pompe patients who have received that drug for seven or eight years. Together with my doctors, I considered increasing my Lumizyme dose, as many Pompe patients have done after plateauing. By the time we actually faced that decision, a new drug was already in clinical trials under the lab name, “Neo-GAA” – an acronym meaning “new GAA.” GAA is the abbreviation for the enzyme, acid alfa glucosidase, the deficiency of which causes Pompe Disease.

My new Pompe specialist informed me that she had experienced denials from insurance companies when she tried to adjust the Lumizyme dosage for other Pompe patients who had plateaued. Because higher doses of Lumizyme deviated from the prescribed standard of care, some insurance companies would not approve an alternate dose. Because Neo-GAA was nearing the end of its clinical trials, my doctor invited me to consider waiting for the new drug. She felt that, because my body was already not responding as favorably to Lumizyme as it once had, administering more was probably just delaying the inevitable. Because Neo-GAA was an entirely different composition, my doctor felt that my body would likely respond more favorably. So we decided to wait.

Neo-GAA was originally scheduled for FDA approval in May 2021. For unexplained reasons, the FDA decided to delay that decision by three months. In August 2021, the FDA approved Neo-GAA at which time it was assigned the new brand name, Nexviazyme (avalglucosidase alfa).

A month later, I met with my Pompe specialist, and she agreed to write the orders for the switch from Lumizyme to Nexviazyme. I began this blog at about that time to document this process. Readers can review the blog posts to see how that process unfolded. It took a while.

 But yesterday, the stars aligned, and I received my first Nexviazyme infusion.

Today, I want to share with you the noticeable differences between the way my body has historically reacted to Lumizyme and the way I’m responding to Nexviazyme.

Ever since my first Lumizyme infusion in 2012, I have had a consistent response to that drug. Lumizyme infusions took about four hours to administer. Beginning about an hour or two into the procedure, I would feel a gradual onset of fatigue. The physical fatigue was accompanied by a state of mental and emotional sensitivity. It’s difficult to think of a suitable comparison but I would become sensitive to light and sound. I would also find it difficult to reason clearly or make decisions. After my first few Lumizyme infusions, I would set the stage for my homecoming by having my dinner planned and prepared so I wouldn’t have to cook or decide what I was eating. It was also common for me to get home, turn down the lights and maybe light some candles, and put on soft music. I would avoid anything, like TV news, that would trigger anxiety. I would basically retreat from the outside world and, if possible, remain so for the day following my infusion. That heightened sensitivity would typically last a full 24 hours.

I have a few Pompe friends who made the switch from Lumizyme to Nexviazyme over the past few weeks and they have reported feeling energized after receiving their first Nexviazyme infusion. Some have speculated that this may be due, in part, to the fact that Nexviazyme is mixed with a dextrose solution (versus the saline solution used with Lumizyme). Dextrose is a sugar which would reasonably give patients a mild energy boost. One Pompe friend asked her doctor about this theory and her doctor said that dextrose wouldn’t really explain much of an energy boost. Whatever the case, I went into yesterday curious to see if I would have a similar reaction.

Yesterday was a very long day, relative to my normal patterns. I was up at 5:00am and went through my normal morning routine. Because I was expecting a long stay at the infusion center, my sister came by in the morning and picked up COPPOLA, my service dog, so he wouldn’t be confined all day. I left for the infusion center at 9:00am and stopped to get some lunch to bring with me. I arrived at the infusion center a little before 10:00am and my infusion was underway by 10:40am. The infusion ended at 3:00pm and I had to remain at the center for a two-hour observation period. I left at 5:00pm and was home by 5:15pm.

I was very tired … but not at all in the way I’m used to after a Lumizyme infusion. I was just tired from a long day. I put away all my gear, my sister dropped by to return COPI, and I settled in. After getting a drink and catching my breath, I began to notice a distinct difference – not just compared to a Lumizyme evening but to any other evening. My mental acuity was much sharper. After about an hour at home, I felt motivated to prepare dinner, sort through emails, return messages, and check in with my family. My energy wasn’t only up, I was feeling exhilarated and social – VERY different from a regular evening and certainly different from an evening following a Lumizyme infusion.

I slept great last night and today, that mental acuity and energy have been sustained.

One element that, for me, is consistent with both Lumizyme and Nexviazyme is my dream time afterwards. Following Lumizyme infusions, I would have super-vivid technicolor immersive dreams with epic storylines. When I woke during the night last night, I was delighted to find that Nexviazyme infusions are followed by similar dreams. Fortunately, I’m not a nightmare person. I have marvelous life-affirming dreams … where I’m not bound by the constraints of Pompe Disease. In my dreams, I can run and leap and fly. So being able to enjoy turbo-charged versions of dreams like that after infusions is a perk!

Enzyme Replacement Therapy (ERT) is not really about an immediate response. ERT nourishes the deficient system of a Pompe patient with normal levels of enzymes that enable our bodies to efficiently process glycogen. ERT is a long-term project. So ultimately, the efficacy of this new drug – Nexviazyme – will be revealed over months and years. But after nine years of feeling completely depleted after ERT, if this new treatment means I’m going to feel mentally and emotionally uplifted, I can get excited about that. I already am!

So, this story will unfold as it does. But after one Nexviazyme infusion, my report is that I feel great … better than I’ve felt in a long time. And since this blog is intended to inform fellow Pompe patients of what they might expect when facing the transition from Lumizyme to Nexviazyme, my report is decidedly favorable.

~ Brian

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