Avalglucosidase alfa

What is Avalglucosidase alfa?

• Avalglucosidase alfa (Nexviazyme) is a hydrolytic lysosomal glycogen-specific enzyme used to treat late-onset Pompe disease.

What are the uses of this medicine?

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

How does this medicine work?

• Pompe disease (also known as glycogen storage disease type II, acid maltase deficiency, and glycogenosis type II) is an inherited disorder of glycogen metabolism caused by a deficiency of the lysosomal enzyme acid α-glucosidase (GAA), which results in intralysosomal accumulation of glycogen in various tissues.
• Avalglucosidase alfa-ngpt provides an exogenous source of GAA.
• The M6P on avalglucosidase alfa-ngpt mediates binding to M6P receptors on the cell surface with high affinity.
• After binding, it is internalized and transported into lysosomes where it undergoes proteolytic cleavage that results in increased GAA enzymatic activity.
• Avalglucosidase alfa-ngpt then exerts enzymatic activity in cleaving glycogen.

Who Should Not Use this medicine ?

• This medicine have no usage limitations.

What drug interactions can this medicine cause?

• No formal drug interaction studies have been conducted with Nexviazyme.

Is this medicine FDA approved?

• Avalglucosidase alfa was approved for medical use in the United States in August 2021.

How should this medicine be used?

• Prior to Nexviazyme administration, consider pretreating with antihistamines, antipyretics, and/or corticosteroids.

Recommended dosage: For patients weighing :

• ≥30 kg, the recommended dosage is 20 mg/kg (of actual body weight) every two weeks.
• <30 kg, the recommended dosage is 40 mg/kg (of actual body weight) every two weeks.

Administration:

• Nexviazyme must be reconstituted and diluted prior to use.
• Nexviazyme is administered as intravenous infusion.
• The initial recommended infusion rate is 1 mg/kg/hour. Gradually increase the infusion rate every 30 minutes if there are no signs of infusion-associated reactions (IARs).

What are the dosage forms and brand names of this medicine?

This medicine is available in fallowing doasage form:

• As injection: 100 mg of avalglucosidase alfa-ngpt as a lyophilized powder in a single-dose vial for reconstitution.

This medicine is available in fallowing brand namesː

• Nexviazyme

What side effects can this medication cause?

The most common side effects of this medicine include:

• headache
• fatigue
• diarrhea
• nausea
• arthralgia
• dizziness
• myalgia
• pruritus
• vomiting
• dyspnea
• erythema
• paresthesia
• urticaria

What special precautions should I follow?

• Life-threatening hypersensitivity reactions, including anaphylaxis, have been reported in Nexviazyme-treated patients. Prior to Nexviazyme administration, consider pretreating with antihistamines, antipyretics, and/or corticosteroids. Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available during Nexviazyme administration.
• Antihistamines, antipyretics, and/or corticosteroids can be given prior to Nexviazyme administration to reduce the risk of infusion-associated reactions (IARs). Patients with an acute underlying illness at the time of Nexviazyme infusion appear to be at greater risk for IARs. If severe IARs occur, consider immediate discontinuation of Nexviazyme, initiation of appropriate medical treatment.
• Advise patients and caregivers that patients with underlying respiratory illness or compromised cardiac or respiratory function may be at risk of acute cardiorespiratory failure from volume overload during Nexviazyme infusion.

What to do in case of emergency/overdose?

• In case of overdose, call the poison control helpline of your country. In the United States, call 1-800-222-1222.

• Overdose related information is also available online at poisonhelp.org/help.
• In the event that the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services. In the United States, call 911.

Can this medicine be used in pregnancy?

• Available data from case reports of Nexviazyme use in pregnant women are insufficient to evaluate for a drug associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes.
• However, available data from postmarketing reports and published case reports on alglucosidase alfa (another hydrolytic lysosomal glycogen-specific enzyme replacement therapy) use in pregnant women have not identified a drug-associated risk of adverse pregnancy outcomes.
• Pregnant women exposed to Nexviazyme, or their healthcare providers, should report Nexviazyme exposure by calling 1-800-745-4447, extension 15500.

Can this medicine be used in children?

• The safety and effectiveness of Nexviazyme for the treatment of late-onset Pompe disease have been established in pediatric patients 1 year of age and older.

What are the active and inactive ingredients in this medicine?

Active ingredient:

• AVALGLUCOSIDASE ALFA

Inactive ingredients:

• GLYCINE
• HISTIDINE
• HISTIDINE MONOHYDROCHLORIDE MONOHYDRATE
• MANNITOL
• POLYSORBATE 80

Who manufactures and distributes this medicine?

Manufactured by:

• Genzyme Corporation
• Cambridge, MA

What should I know about storage and disposal of this medication?

• Refrigerate vials of Nexviazyme at 36°F to 46°F (2°C to 8°C ).
• Do not use Nexviazyme after the expiration date on the vial.

• Dailymed label info on Avalglucosidase alfa
• FDA Avalglucosidase alfa

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