Lumizyme ACE Program®

Lumizyme is indicated for use in patients 8 years and older, with late (non-infantile) onset Pompe disease who do not have evidence of cardiac hypertrophy. Lumizyme is available only under a restricted distribution program called the Lumizyme ACE (Alglucosidase Alfa Control and Education).

The program is a risk evaluation and mitigation strategy designed to:

  • Ensure that the known risks of anaphylaxis and severe allergic reactions and the potential risks of severe cutaneous and systemic immune complex-mediated reactions associated with the use of Lumizyme are communicated to patients, caregivers, and prescribers.
  • Mitigate the potential risk of rapid disease progression in infantile-onset Pompe disease patients and late (non-infantile) onset Pompe disease patients less than 8 years of age for whom the safety and effectiveness of Lumizyme have not been evaluated.

Participation in the Lumizyme ACE Program is mandatory if you intend to prescribe or administer Lumizyme.

Program Requirements

Under the Lumizyme ACE Program, only trained and certified healthcare providers and facilities are able to prescribe, order, dispense, or administer Lumizyme. Only patients who are enrolled in and meet all the conditions of the program may receive Lumizyme treatment.

Learn more about Program Requirements

INDICATION

LUMIZYME®(alglucosidase alfa) is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (acid α-glucosidase (GAA) deficiency).

IMPORTANT SAFETY INFORMATION

WARNING: RISK OF ANAPHYLAXIS, HYPERSENSITIVITY AND IMMUNE-MEDIATED REACTIONS, and RISK OF CARDIORESPIRATORY FAILURE

See full prescribing information for complete boxed warning.

Life-threatening anaphylactic reactions and severe hypersensitivity reactions, presenting as respiratory distress, hypoxia, apnea, dyspnea, bradycardia, tachycardia, bronchospasm, throat tightness, hypotension, angioedema (including tongue or lip swelling, periorbital edema, and face edema), and urticaria, have occurred in some patients during and after alglucosidase alfa infusions. Immune-mediated reactions presenting as proteinuria, nephrotic syndrome, and necrotizing skin lesions have occurred in some patients following alglucosidase alfa treatment. Closely observe patients during and after alglucosidase alfa administration and be prepared to manage anaphylaxis and hypersensitivity reactions. Inform patients of the signs and symptoms of anaphylaxis, hypersensitivity reactions, and immune-mediated reactions and have them seek immediate medical care should signs and symptoms occur.

Infantile-onset Pompe disease patients with compromised cardiac or respiratory function may be at risk of serious acute exacerbation of their cardiac or respiratory compromise due to fluid overload, and require additional monitoring.

WARNINGS AND PRECAUTIONS

Anaphylaxis and Hypersensitivity Reactions: Life-threatening anaphylaxis and hypersensitivity reactions have been observed in some patients during and after treatment with alglucosidase alfa. If anaphylaxis or severe hypersensitivity reactions occur, immediately discontinue infusion and institute appropriate medical treatment.

Immune-Mediated Reactions: Monitor patients for the development of systemic immune-mediated reactions involving skin and other organs.

Risk of Acute Cardiorespiratory Failure: Patients with compromised cardiac or respiratory function may be at risk of acute cardiorespiratory failure. Caution should be exercised when administering alglucosidase alfa to patients susceptible to fluid volume overload.

Risk of Cardiac Arrhythmia and Sudden Cardiac Death during General Anesthesia for Central Venous Catheter Placement: Caution should be used when administering general anesthesia for the placement of a central venous catheter intended for alglucosidase alfa infusion.

Appropriate medical support and monitoring measures should be available during infusion.

ADVERSE REACTIONS

The most frequently reported adverse reactions (≥ 5%) in clinical trials were hypersensitivity reactions and included: anaphylaxis, rash, pyrexia, flushing/feeling hot, urticaria, headache, hyperhidrosis, nausea, cough, decreased oxygen saturation, tachycardia, tachypnea, chest discomfort, dizziness, muscle twitching, agitation, cyanosis, erythema, hypertension/increased blood pressure, pallor, rigors, tremor, vomiting, fatigue, and myalgia (6.1).

USE IN SPECIFIC POPULATIONS

Pregnancy: Based on animal data, alglucosidase alfa may cause fetal harm.

To report SUSPECTED ADVERSE REACTIONS, contact Genzyme at 1-800-745-4447 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch

Please see the full prescribing information for complete details, including boxed warning.

Online Training for the Lumizyme ACE Program®

Complete your training and certification for the Lumizyme ACE Program conveniently online.

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Lumizyme ACE Program® Enrollment

After completing the Lumizyme ACE Program® training, complete your enrollment by submitting these forms to Genzyme:

Reporting Adverse Reactions

You can report suspected adverse reactions to Genzyme by either:

Calling 800-745-4447, option 2 OR
Sending us a detailed description by fax at 617-761-8506 or by email

You can also report directly to the FDA's MedWatch program: call 800-FDA-1088 or visit the MedWatch site.