Resources for Patients

The organizations listed below provide support to patients and families living with Pompe disease through educational information, updates on research and treatment advances, support groups, and more. Please note that the websites listed are maintained by the sponsoring organizations. Sanofi Genzyme is not responsible for maintaining these websites.

Acid Maltase Deficiency Association (AMDA)
www.amda-pompe.org
Formed to assist in funding research and to promote public awareness of acid maltase deficiency, another name for Pompe disease, this U.S. organization is a member of the International Pompe Association.

Association for Glycogen Storage Disease (AGSD)
www.agsdus.org
A parent- and patient-oriented support group based in the U.S. The AGSD was established for parents of and individuals with glycogen storage diseases to communicate, share their successes and concerns, share useful findings, provide support as needed, create an awareness of this condition for the public, and to stimulate research in the various forms of glycogen storage diseases.

Genetic Alliance
www.geneticalliance.org
A non-profit health organization committed to transforming health through genetics and promoting an environment of openness centered on the health of individuals, families, and communities.

Muscular Dystrophy Association (MDA)
www.mdausa.org
A non-profit health organization dedicated to providing comprehensive medical services to individuals affected by neuromuscular diseases. Pompe disease is one of the more than 40 neuromuscular diseases covered by MDA.

National Organization for Rare Disorders (NORD)
www.rarediseases.org
A unique federation of individuals and organizations working together to build a better world for people affected by rare diseases.

United Pompe Foundation
www.unitedpompe.com
An organization formed to assist patients and/or their families with medical costs and other expenses that may not be covered by insurance. The Foundation also hopes to raise public awareness of Pompe disease.

Lumizyme.com for Your Patients

To help address your patients' questions about Lumizyme, getting infusions, and other related topics, you can direct them to the Patients section of this website.

Visit the Lumizyme.com Patients section

INDICATION

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

IMPORTANT SAFETY INFORMATION

WARNING: RISK OF ANAPHYLAXIS, HYPERSENSITIVITY AND IMMUNE-MEDIATED REACTIONS, and RISK OF CARDIORESPIRATORY FAILURE
  • 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. Appropriate medical support and monitoring measures should be available during infusion.

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

Risk of Acute Cardiorespiratory Failure: Patients with acute underlying respiratory illness and compromised cardiac and/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. Appropriate medical support and monitoring measures should be available during infusion and some patients may require longer observation times.

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.

Risk of Antibody Development: As with all therapeutic proteins, there is potential for immunogenicity. There is some evidence to suggest that some patients who develop high and sustained IgG antibody titers may experience reduced clinical efficacy. Patients should be monitored for IgG antibody formation every 3 months for 2 years and then annually thereafter.

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.

USE IN SPECIFIC POPULATIONS

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

Please see the Full Prescribing Information, for complete details, including boxed WARNING

The Pompe Registry

To learn more about the Pompe Registry, visit www.registrynxt.com.

Contact Us

Sanofi Genzyme
50 Binney Street
Cambridge, MA 02142

Tel : 617-768-9000
Fax : 617 252 7600
Toll free : 800-745-4447