NEXVIAZYME is the first new enzyme replacement therapy for late-onset Pompe disease (LOPD) in 15 years. It’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.

In a clinical study of 100 LOPD patients who had not been on treatment before, and who were given either NEXVIAZYME or alglucosidase alfa, NEXVIAZYME helped improve their breathing and walking distance compared to when they began the study.

After 49 weeks on NEXVIAZYME, compared to when they began treatment

People were able to improve their breathing capacity by an average of 2.9 percentage points in a breathing test:

• 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.

People on NEXVIAZYME improved their walking distance by an average of 106 feet during a 6-minute walk test:

• Those taking alglucosidase alfa improved their walking distance by an average of 7.2 feet.
• People taking NEXVIAZYME walked an average of 98 feet farther than those who were taking alglucosidase alfa. The study was not designed to test whether NEXVIAZYME was superior to alglucosidase alfa for this measure.

The tests were given at the beginning of the study and again at the end, after 49 weeks of treatment.

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.

Click here for Important Safety Information

These are not all 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 or www.fda.gov/medwatch.

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 has something called “high binding affinity” for a receptor in your muscle cell called M6P receptor. That receptor is like a lock on a door. M6P is like the key to opening that lock. NEXVIAZYME has “keys” on its surface that fit in the “lock.” In fact, NEXVIAZYME has about 15 times the number of keys that alglucosidase alfa has. In a clinical study, NEXVIAZYME was not shown to be statistically superior to alglucosidase alfa.

NEXVIAZYME is given every 2 weeks by intravenous (IV) infusion. The recommended dosage of NEXVIAZYME is either 20 mg or 40 mg for each kilogram of body weight—your healthcare provider will calculate the appropriate dosage for you. The infusion usually takes approximately 4-5 hours for those receiving 20 mg/kg and approximately 5-7 hours for those receiving 40 mg/kg. There could be additional time if you need any pretreatment. Also, infusion times may vary based on your response to therapy and comfort. Your healthcare provider will give you more details about what to expect during and after your infusion as well as how to prepare.

You’ll most likely want to bring a book, work, and/or electronic devices to make the most of your time. If you’re switching to NEXVIAZYME from alglucosidase alfa, your treatment schedule may stay the same.