About NEXVIAZYME

An enzyme replacement monotherapy* for
LOPD

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The NEX chapter in Pompe treatment for LOPD patients

NEXVIAZYME is a monotherapy.* This means you only have to take a single medicine to treat your LOPD.* It’s also an M6P-enriched treatment. See pivotal study results below.

*Not including premedication or pretreatment your doctor may prescribe.

What is M6P?

M6P (Mannose 6-Phosphate) is a part of the molecule that binds to certain muscle cell
receptors and helps enzyme replacement therapy reach your muscle cells.

NEXVIAZYME 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.

What is Pompe disease?

How does NEXVIAZYME work?

Pompe is a degenerative muscle disease caused by a deficiency in the GAA enzyme.
In a normal muscle cell, GAA enzyme breaks down glycogen, a type of sugar, so the cell can it use it for energy. This happens in an area called the lysosome. To get there, GAA needs the help of M6P, which is a part of a molecule.
People with Pompe disease have very little GAA or none at all, which is why the body can’t break down glycogen in the muscle as it should. The glycogen builds up, causing ongoing muscle damage.
NEXVIAZYME is an M6P-enriched enzyme replacement therapy for late-onset Pompe disease (LOPD). It helps replace GAA enzyme for those whose bodies don’t produce enough. NEXVIAZYME is scientifically designed to be absorbed effectively by muscles.

NEX move: better breathing, greater endurance

NEXVIAZYME vs alglucosidase alfa

NEXVIAZYME showed meaningful improvement for breathing and walking when compared to alglucosidase alfa in a pivotal study (COMET trial).

Everyone (100 patients aged 16-78 years of age with LOPD) in the study had late-onset Pompe disease, but none of them had received any prior treatment. Some were given NEXVIAZYME, and others were given alglucosidase alfa. All 100 people stayed in the study for 49 weeks.

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) measures how much air you can force out in a deep breath
  • 6-minute walk test (6MWT) measures the distance you can walk within that time frame

The tests were given at the beginning and again at the end of the study.

After ~1 year (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.
This measurement was not tested to determine statistical superiority of NEXVIAZYME to alglucosidase alfa.

NEXVIAZYME demonstrated long-term stability

Demonstrated long-term stability

People on NEXVIAZYME were able to breathe better and walk farther at ~1 year (49 weeks), compared to when they started. In the open label extension study,* whether people started with or switched to NEXVIAZYME from alglucosidase alfa at ~1 year, they were able to maintain stability for ~3 years (145 weeks); no new safety concerns were observed.

After the first ~1 year (49 weeks) of the study, 44 people on alglucosidase alfa switched to NEXVIAZYME. Meanwhile, the 51 people already on NEXVIAZYME continued to take it. After ~3 years (145 weeks) into the open label extension study, most people on NEXVIAZYME were able to maintain their breathing and walking ability. *Open label: Both the doctors and patients knew what treatment people were taking

What was learned about safety

In a clinical trial, NEXVIAZYME demonstrated similar safety and risks to alglucosidase alfa. Over the 49-week study, people taking NEXVIAZYME had fewer serious side effects than those taking alglucosidase alfa.† No one had to stop taking NEXVIAZYME because of side effects, while 4 people had to stop taking alglucosidase alfa.

COMMON SIDE EFFECTS OF NEXVIAZYME INCLUDE:

  • headache
  • fatigue
  • dizziness
  • nausea
  • diarrhea
  • vomiting
  • joint pain
  • muscle pain
  • itching
  • shortness of breath
  • rash
  • “pins-and-needles” sensation
  • hives
  • headache
  • fatigue
  • dizziness
  • nausea
  • diarrhea
  • vomiting
  • joint pain
  • muscle pain
  • itching
  • shortness of breath
  • rash
  • “pins-and-needles” sensation
  • hives

These are the most common side effects, but there are other possible side effects. You should always tell your doctor about any changes in the way you feel, even if it’s not one of the listed side effects. You can also report side effects at
1-800-FDA-1088 or www.fda.gov/medwatch

When antibodies form, they can interfere with some medicines. There is a potential to develop antibodies, but for the patients in the trial it didn’t affect their efficacy. Patients in the clinical trial who did have high titers for antibodies had a higher risk of experiencing infusion-associated reactions.

NEXVIAZYME has a demonstrated safety profile with nearly 7 years of clinical data from 4 clinical trials, which includes people who switched from alglucosidase alfa.

†The Comet trial was not designed to demonstrate a statistically significant difference in the incidence of adverse reactions between NEXVIAZYME and alglucosidase alfa. 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

WHAT
MOVES
YOU?

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IMPORTANT SAFETY INFORMATION AND INDICATION

IMPORTANT SAFETY INFORMATION

WARNING: SEVERE HYPERSENSITIVITY REACTIONS, INFUSION-ASSOCIATED REACTIONS, and RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS

Hypersensitivity Reactions Including Anaphylaxis

If you are taking NEXVIAZYME, you should know that severe and potentially life threatening allergic-type reactions known as anaphylaxis and severe hypersensitivity reactions have occurred during and after NEXVIAZYME treatment. You should seek immediate medical care if signs and symptoms of anaphylaxis or hypersensitivity reactions occur. If such a reaction is severe enough, your doctor may decide to immediately discontinue the infusion and provide immediate medical care. Appropriate medical support measures may be administered during your infusion, and you may require close observation during and after NEXVIAZYME administration.

Infusion-Associated Reactions (IARs)

If you are taking NEXVIAZYME, you should know that severe IARs have occurred during and after NEXVIAZYME treatment. If severe IARs occur during your NEXVIAZYME infusion, your doctor may decide to immediately discontinue the infusion and provide appropriate medical care. If you have an acute underlying illness at the time of NEXVIAZYME infusion you may be at greater risk for IARs. If you have advanced Pompe disease you may have compromised heart and breathing function, which may put you at a higher risk of severe complications from IARs.

Risk of Acute Cardiorespiratory Failure in Susceptible Patients

If you are likely to develop fluid volume overload, or have acute underlying breathing problems or compromised heart or breathing function that may require fluid restriction, there may be a risk of worsening of your heart or breathing status during NEXVIAZYME infusion. Your doctor may decide that close observation during NEXVIAZYME administration may be necessary.

WARNINGS AND PRECAUTIONS

Hypersensitivity Reactions Including Anaphylaxis: See Boxed WARNING. Your doctor may decide to give you antihistamine, anti-fever and/or steroid medications before your infusions. Your doctor should consider the risks and benefits of restarting the infusion if you have a severe hypersensitivity reaction (including anaphylaxis) to NEXVIAZYME. If a mild or moderate hypersensitivity reaction occurs, your healthcare provider may slow the infusion rate or temporarily stop the infusion.

Infusion-Associated Reactions (IARs): See Boxed WARNING. Your doctor may decide to give you medications before your infusions to decrease the risk of IARs; however, IARs may still occur after receiving these medications. If mild or moderate IARs occur, your healthcare provider should consider decreasing the infusion rate or temporarily stopping the infusion which may help improve the symptoms.

Risk of Acute Cardiorespiratory Failure in Susceptible Patients: See Boxed WARNING.

ADVERSE REACTIONS

The most common adverse reactions (>5%) were headache, fatigue, diarrhea, nausea, joint pain, dizziness, muscle pain, itching, vomiting, shortness of breath, rash, “pins-and-needles” sensation, and hives.

Please see full Prescribing Information for complete details, including Boxed WARNING.

INDICATION

NEXVIAZYME (avalglucosidase alfa-ngpt) is used for the treatment of patients 1 year of age and older with late-onset Pompe disease [lysosomal acid alpha-glucosidase (GAA) deficiency].