We’re Eyeing a Medication Switch to Nexviazyme

Recently, my 4-year-old son, Cayden, had an appointment with his metabolic specialist. This doctor manages everything related to his Pompe disease and is part of a team of doctors that diagnosed him when he was just 1 month old. They also oversee everything related to his enzyme replacement infusions.

Since his diagnosis, Cayden has been receiving Lumizyme (alglucosidase alfa) infusions, the only approved treatment he could access at the time. However, things have changed in the past four years, and a new medication called Nexviazyme (avalglucosidase alfa) has been approved. The results seem promising. I made sure to have a discussion about Nexviazyme during Cayden’s recent appointment.

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I asked the metabolic doctor if Nexviazyme might be an option for Cayden in the near future. The doctor gave me a lot of great information and said we’d work on getting Cayden switched by the summer. While the U.S. Food and Drug Administration approved Nexviazyme a couple years ago, it was only for patients ages 1 year or older with late-onset Pompe disease. Cayden is well above that age and has infantile-onset Pompe, not late-onset.

Due to the difference in onsets, Cayden would have to receive the drug off-label. This isn’t a huge deal, considering he is already receiving Lumizyme infusions off-label. Instead of the standard biweekly Lumizyme dose of 20 mg/kg, Cayden receives 40 mg/kg. We increased the dosage when he was about 18 months old after a study found that a higher dose may be better for children with infantile-onset Pompe disease.

The problem with using medications off-label is that insurance companies aren’t always happy to accept the requests. But Cayden’s doctors are willing to put up a fight if needed, and they can easily prove why he would benefit from a switch in medication. Getting the Nexviazyme approved by our insurance company may take time, which is why our target is summertime.

While switching to Nexviazyme sounds like a great opportunity, I’m a little bummed about one detail. Because it’s a new medication, Cayden will have to receive the first couple infusions at the daytime infusion clinic located over two hours away, where his specialists are.

I understand the importance of monitoring him during administration of the new medication, but I will miss our home infusions. We have built such a strong bond with our home infusion nurse, and not seeing her every week will feel weird. Hopefully, if all goes well, Cayden will be able to go back to home infusions in no time.

We are crossing our fingers for Cayden to do well with the switch to Nexviazyme. I am so grateful to live at a time when more than one treatment option exists for such a rare disease, and I’ll forever be thankful for the amazing people who took part in clinical trials to make it happen.

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.