About NEXVIAZYME

Understand NEXVIAZYME and learn about the results of a pivotal study.

New to
treatment? Switching treatment? NEXVIAZYME may be your new choice

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.

How does NEXVIAZYME work?

When you have LOPD, the body does not have enough of the enzyme that breaks down glycogen in your muscle cells. So the glycogen builds up and causes muscle damage. NEXVIAZYME is an enzyme replacement therapy that’s designed to bind to muscle cells with a high affinity (a strong attachment). After this happens, NEXVIAZYME enters the cells, so it can help break down glycogen.

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—GAA enzymes break 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:

person breathing
Improve the breathing by averange of 2.9 percentage points in a breathing test

*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.

person walking
Improve the walking distance by an average of 106 feet during a 6mwt

*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 a new 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