Understand NEXVIAZYME and learn about the results of a pivotal study.
Switching treatment? NEXVIAZYME may be the right choice for you
Wherever you are in your late-onset Pompe disease (LOPD) journey, it’s good to explore what’s possible. Learn all about NEXVIAZYME and how it may improve your breathing and walking distance. As always, CareConnectPSS® and your healthcare provider can answer more questions.
You can reach CareConnectPSS® at 1-800-745-4447, option 3.
NEXVIAZYME is an M6P-enriched enzyme replacement therapy
Here’s how it works
When you have LOPD, the body doesn’t have enough of the enzyme that breaks down glycogen in your muscle cells. So glycogen builds up and causes muscle damage.
NEXVIAZYME is a scientifically advanced, M6P-enriched therapy that’s designed to be effectively absorbed by muscles to clear out more glycogen for people with LOPD.
In fact, NEXVIAZYME was created with muscle penetration specifically in mind. It has 15x more M6P than the current standard of care, alglucosidase alfa (Lumizyme), and was made to improve uptake of the enzyme into muscle cells.
Scroll through the image of a muscle cell below to get an idea of how NEXVIAZYME works. Helpful terms to know for this example—and for Pompe disease in general—are:
- Glycogen—A type of sugar that your muscle cells can store to break down for energy when needed.
- Lysosome—A saclike part of a cell that contains enzymes, which can break down particles such as glycogen. To do this, it needs GAA enzyme.
- GAA (acid alpha-glucosidase)—An enzyme that breaks down glycogen inside the lysosome. In Pompe disease, the GAA enzymes don’t work as expected.
What does NEXVIAZYME do?
The truth is, it's easy to take breathing and walking for granted—unless you have a progressive disease like LOPD. People living with LOPD know this. They also know the importance of staying informed about treatment. NEXVIAZYME has demonstrated results in a pivotal study.
The 2 tests used to measure how people were doing in the study are tests you may be familiar with if you’ve been living with LOPD:
- Forced vital capacity (FVC) which measures how much air you can breathe out in one forceful breath.
- 6-minute walk test (6MWT) which measures the distance you can walk within that time frame
The clinical study included 100 people with LOPD who had never been treated before. One group was given NEXVIAZYME, and the other was given alglucosidase alfa.
The tests were given at the beginning of the study and again at the end, after 49 weeks of treatment. You can talk to your doctor about what these trial results mean.
After 49 weeks on NEXVIAZYME, people were able to:
*Compared to when they began treatment
Those who took alglucosidase alfa improved their breathing by an average of 0.5 percentage points.* This resulted in an average of 2.4 percentage points measurable improvement for people treated with NEXVIAZYME compared with people taking alglucosidase alfa, although NEXVIAZYME was not statistically superior.
*Compared to when they began treatment
Those who took alglucosidase alfa improved their walking distance by an average of 7.2 feet.* People taking NEXVIAZYME walked an average of 98 feet further than those who were taking alglucosidase alfa. The study was not designed to test whether NEXVIAZYME was superior to alglucosidase alfa for this measure.
What was learned about safety
In the clinical trial, serious adverse reactions were reported in 1 patient treated with NEXVIAZYME and in 3 patients treated with alglucosidase alfa.
The most common side effects reported by people receiving NEXVIAZYME were headache, fatigue, diarrhea, nausea, joint pain, dizziness, muscle pain, itching, vomiting, shortness of breath, rash, “pins-and-needles” sensation, and hives.
In addition, 13 out of 51 people (25%) receiving NEXVIAZYME experienced mild to moderate infusion- associated reactions. These reactions included headache, diarrhea, itching, hives, and rash. No one experienced a severe infusion-associated reaction.
Please see additional Important Safety Information,
including Boxed WARNING.
These are not all of the possible side effects. If you experience any side effects, it’s important to tell your healthcare provider right away. You can also report side effects at 1-800-FDA-1088 1-800-FDA-1088 or www.fda.gov/medwatch.
Get ready to talk to your healthcare provider about NEXVIAZYME
Now that you’ve learned about another option, the next step is to find out if it may be right for you.
To have a more informed conversation with your healthcare provider, click the button below to complete the interactive self-assessment guide. This can be especially helpful if you’ve missed your regularly scheduled check-up due to Covid or concerns about Covid.
When you talk with your healthcare provider about changes you’ve noticed, goals and needs, you can ask if NEXVIAZYME can help you get where you want to be. Your healthcare provider can also help you understand what to expect with NEXVIAZYME.START WITH THE INTERACTIVE GUIDE