Switching to Nexviazyme Not So Easy for Infantile-onset Pompe

Keara Engle avatar

by Keara Engle |

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A few weeks ago, I discussed how we were eyeing a medication switch to Nexviazyme (avalglucosidase alfa) for my 4-year-old son, Cayden. But switching to this new drug may not be as easy as his doctors and I were hoping.

Cayden has been receiving Lumizyme (alglucosidase alfa) infusions since he was 1 month old for his infantile-onset Pompe disease. While he remains stable on the Lumizyme, we were hoping to switch him to Nexviazyme for a few reasons, mostly because studies on the drug have shown promising results. The majority of patients who’ve tried it have shown improvement in pulmonary function, for instance.

Pompe disease has negatively affected Cayden’s lungs. A small cold often leads to pneumonia and lands him in the hospital for weeks or even months on end. We were hoping that the Nexviazyme would give his lungs the extra strength they need to fight off infections.

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His metabolic specialists submitted the request to switch Cayden to Nexviazyme a few weeks ago. However, our insurance company won’t allow this. It’s telling us it cannot OK the switch because the U.S. Food and Drug Administration has only approved Nexviazyme for patients with late-onset Pompe disease, which is not the type Cayden has.

While there have been some Nexviazyme studies on children with infantile-onset Pompe disease, the insurance doesn’t seem to care. It told us it won’t approve the medication switch because he doesn’t have a qualifying diagnosis.

This news was upsetting to hear. My son does have Pompe disease. Yes, he has infantile-onset, not late-onset, but he is still greatly affected by this disease and everything that comes along with it. I understand why the insurance company is wary of approving the switch, but as his mother, it just frustrates me. The people at the insurance company aren’t around us every day. They don’t see how Pompe affects just about any and every aspect of our lives.

Cayden’s specialists have one more idea they want to try in order to get him switched to Nexviazyme. Sanofi Genzyme, the company that created both Lumizyme and Nexviazyme, offers a patient assistance program to qualifying families. It helps cover the cost of the drug if your insurance company doesn’t approve it.

While this program sounds promising, I’m not getting my hopes up. We’re still waiting to hear back, and I’m not sure what kind of answer we’ll get. If it won’t cover the Nexviazyme infusions, we’re out of luck. These infusions are so expensive that there’s no way I could ever afford to pay out of pocket for them. But I am hoping that the company recognizes our reasons for wanting to switch and allows us to do so.

I’m anxious to see whether or not we will get Cayden switched to Nexviazyme. It would be great if we could, but if we can’t, at least we already have a medication he remains stable on. Not every rare disease has a treatment option, let alone more than one. So for now, I must be grateful for what we do have.

Note: Pompe Disease 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 Pompe Disease News or its parent company, BioNews, and are intended to spark discussion about issues pertaining to Pompe disease.


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