Genzyme is pleased to share with you an update regarding the recent United States (U.S.) Food and Drug Administration’s (FDA) approval of an expanded label for Lumizyme® (alglucosidase alfa).
In January 2014 Genzyme submitted a Supplemental Biologics License Application (sBLA) to the FDA in support of a label expansion for Lumizyme. The purpose of the application was to expand the indication for Lumizyme to permit its use in all Pompe patients.
On August 1, 2014, the U.S. Food and Drug Administration (FDA) approved the supplement to expand the indication for Lumizyme. The expanded Lumizyme label is based on data that included biochemical analysis as well as clinical data from an investigator sponsored trial in Taiwan in infantile-onset patients. Additional supportive data was included from the ADVANCE trial of patients 12 months and older previously treated with Myozyme and switched to Lumizyme as part of the trial. This data demonstrated that alglucosidase alfa 4000L (Lumizyme) is comparable to alglucosidase alfa 160L (Myozyme®). Lumizyme is now indicated for patients with Pompe disease. There is no limitation as to age and phenotype. Patients currently receiving commercial Lumizyme are not affected at all by this change. All newly diagnosed Pompe patients regardless of age now have access to Lumizyme, should ERT be considered.
This approval removes the Risk Mitigation Evaluation Strategy (REMS) required to treat patients receiving Lumizyme. Patients, Healthcare Providers, and Facilities treating late-onset patients are no longer required to participate in the Lumizyme Alglucosidase Alfa Control and Education (ACE) Program® prior to prescribing or receiving Lumizyme. However, Lumizyme continues to have a box warning regarding certain serious adverse reactions. It is, therefore, extremely important for Healthcare Providers and patients to be educated about the possible reactions associated with Lumizyme and what to do if a reaction occurs.
While this information may not directly impact you or your patients, Genzyme wanted to share with you the press release announcing the label expansion, the new label and the patient communications that we have distributed through various channels.
Should you have any additional questions please feel free to contact Genzyme’s Medical Information department at 1-800-745-4447 (toll-free) or 617-768-9000, and choose option #2 or your local Genzyme representative.
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
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.
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.
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.