NEXVIAZYME (avalglucosidase alfa-ngpt)

NEXVIAZYME (avalglucosidase alfa-ngpt) is an enzyme replacement therapy (ERT) used to treat patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency). Pompe disease is a rare genetic disorder characterized by the accumulation of glycogen in the muscles, which impairs muscle function. NEXVIAZYME works by replacing the deficient enzyme in patients, helping to reduce glycogen buildup and improve muscle function. This medication is administered through intravenous infusion and is typically prescribed under a specialist’s care. NEXVIAZYME offers a significant advancement in managing Pompe disease, improving patients’ quality of life.

How It Works

NEXVIAZYME contains avalglucosidase alfa, a recombinant human enzyme that mimics the naturally occurring lysosomal enzyme, acid alpha-glucosidase (GAA). In patients with Pompe disease, a deficiency in this enzyme leads to the accumulation of glycogen in tissues, particularly in muscles, which causes progressive muscle weakness.

Mechanism of Action:

1. Enzyme Replacement: NEXVIAZYME delivers an active form of GAA to the lysosomes of cells. It is designed to bind to the mannose-6-phosphate receptors on the cell surface, facilitating its entry into cells.
2. Breakdown of Glycogen: Once inside the lysosomes, the enzyme breaks down glycogen into glucose, reducing its harmful accumulation.
3. Muscle Function Improvement: By reducing glycogen buildup, NEXVIAZYME improves muscle strength and slows disease progression, helping patients manage their symptoms more effectively.

NEXVIAZYME targets the underlying cause of Pompe disease and has shown promise in improving respiratory function and walking ability in patients with late-onset forms of the disease.

Side Effects

While NEXVIAZYME can offer significant benefits, it may also cause side effects, which vary from patient to patient.

Common Side Effects:

• Headache: Mild to moderate headaches are a common side effect.
• Fatigue: Some patients may feel unusually tired or weak after treatment.
• Nausea: Gastrointestinal discomfort, including nausea, can occur.
• Diarrhea: Loose or frequent bowel movements have been reported.
• Fever: Some patients experience a rise in body temperature post-infusion.

Serious Side Effects:

• Hypersensitivity Reactions: Including anaphylaxis or infusion-related reactions, such as difficulty breathing, rash, or swelling.
• Respiratory Distress: Shortness of breath or wheezing may occur, particularly in patients with pre-existing respiratory issues.
• Increased Blood Pressure: Some patients have experienced elevated blood pressure following infusions.
• Severe Allergic Reactions: Severe reactions, including swelling of the face, hives, or itching.

Patients should be closely monitored during and after administration for any serious adverse reactions. In cases of severe allergic responses, the infusion should be stopped immediately, and appropriate medical treatment should be initiated.

Indications

NEXVIAZYME is specifically indicated for the treatment of patients with late-onset Pompe disease. This genetic disorder results in a deficiency of the enzyme acid alpha-glucosidase (GAA), which is necessary for breaking down glycogen. When glycogen accumulates, it damages muscles and other tissues, leading to progressive weakness and respiratory failure.

Approved Indications:

1. Late-Onset Pompe Disease (LOPD): NEXVIAZYME is used to treat patients diagnosed with late-onset Pompe disease by helping to manage the symptoms of muscle weakness and respiratory dysfunction.

Contraindications

NEXVIAZYME is contraindicated in certain patients who may experience severe adverse reactions.

Contraindications:

1. Severe Hypersensitivity to Avalglucosidase Alfa: Patients who have had life-threatening hypersensitivity or anaphylactic reactions to NEXVIAZYME or any of its components should avoid the drug.
2. Severe Infusion-Related Reactions: Those who have experienced serious infusion-related reactions despite appropriate medical intervention should not continue treatment.
3. Patients with Active Infection: NEXVIAZYME should be used with caution in patients with an active infection or those with an underlying compromised immune system.
4. Severe Respiratory Compromise: Patients with severe respiratory issues should be evaluated closely, as the drug may cause worsening of respiratory symptoms.

Price in Different Countries

The cost of NEXVIAZYME can vary significantly depending on the healthcare system, availability, and insurance coverage in each country. Below is a table comparing the price of NEXVIAZYME in different regions.

Country	Price (Per Infusion)	Reference
United States	$25,000 – $30,000	GoodRx
United Kingdom	£18,000 – £25,000	NHS
Canada	CAD $20,000 – $28,000	Canada Pharmacy
Australia	AUD $35,000	PBS Australia
India	INR ₹15,00,000 – ₹20,00,000	1mg

Top 5 Global Brands

NEXVIAZYME is produced by Sanofi, a global leader in healthcare and pharmaceuticals, but there are other key players in the field of enzyme replacement therapies and Pompe disease management.

1. Sanofi

• The manufacturer of NEXVIAZYME, Sanofi is a multinational pharmaceutical company that focuses on developing treatments for rare genetic diseases, among others.

2. Amicus Therapeutics

• Known for their focus on rare and orphan diseases, Amicus Therapeutics develops therapies for lysosomal storage disorders and other rare conditions.

3. Genzyme (a Sanofi Company)

• Genzyme is another division of Sanofi, specialized in rare diseases, including enzyme replacement therapies for conditions like Pompe disease.

4. BioMarin Pharmaceutical

• BioMarin develops treatments for rare diseases, including enzyme replacement therapies, and is a recognized leader in genetic disorders.

5. Shire (Now part of Takeda Pharmaceuticals)

• Shire, now integrated with Takeda, has a focus on rare diseases and produces treatments for several lysosomal storage disorders.