Lumizyme is produced using recombinant DNA technology, a process that allows scientists to alter the genetic make-up of an organism in order to produce human proteins such as enzymes. This highly complex biomanufacturing process was developed after many years of research and testing. It takes place at Sanofi Genzyme facilities in three stages that last several months.
Lumizyme is a therapeutic protein. Production begins by inserting the human gene that controls production of the GAA enzyme into CHO (Chinese hamster ovary) cells. Once the CHO cells have the gene, they will begin to manufacture the human GAA enzyme. The cells are kept under special conditions in large tanks called bioreactors. Each day, liquid is removed from the bioreactor, and the enzyme those cells have produced is collected for purification.
Lumizyme must meet very high standards for purity and safety. At Sanofi Genzyme, the enzyme is purified using a process called column chromatography, a method of separating and isolating the parts of a mixture to remove unwanted substances. As the enzyme moves through multiple chromatography columns, it becomes more purified.
After purification, the enzyme is put into sterile glass vials. After the vials are filled, they are placed into a freeze dryer for about 48 hours. In the freeze dryer, water evaporates off the enzyme and leaves a cake-like dry substance. In this form, the enzyme is more stable. Multiple tests are conducted through the manufacturing process to help ensure Lumizyme meets the highest standards. Each vial is inspected before it is released and made available for administering to patients.
Making Lumizyme is a complex process that takes several months and could only be accomplished after many years of development and testing.
LUMIZYME® (alglucosidase alfa) is an enzyme replacement therapy for patients with Pompe disease (acid α-glucosidase (GAA) deficiency).
WARNING: RISK OF ANAPHYLAXIS, HYPERSENSITIVITY AND IMMUNE-MEDIATED REACTIONS, AND RISK OF CARDIORESPIRATORY FAILURE
Anaphylaxis and Hypersensitivity Reactions: Life-threatening anaphylaxis and hypersensitivity reactions have been observed in some patients during and after treatment with alglucosidase alfa. If such a reaction is severe enough, your doctor may decide to immediately discontinue the infusion and provide you with immediate medical care. Appropriate medical support and monitoring measures should be available during infusion.
Immune-Mediated Reactions: You or your child may be monitored for the development of systemic immune-mediated reactions while receiving Lumizyme. If these reactions occur, your doctor may discontinue the infusion and initiate appropriate medical treatment.
Risk of Acute Cardiorespiratory Failure: Infant Pompe patients with heart or breathing problems who are experiencing an acute respiratory condition may be at risk for increasing the seriousness of these problems as a result of Lumizyme administration due to the infusion fluid, and your child’s doctor may require additional monitoring for these infants.
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 Lumizyme infusion. Ventricular arrhythmias and slow heart rate resulting in cardiac arrest or death have been observed in infant Pompe patients with cardiac hypertrophy during general anesthesia for central venous catheter placement. Appropriate medical support and monitoring measures should be available during infusion.
Risk of Antibody Development: Patients with infantile-onset Pompe disease (IOPD) should be managed by a clinical specialist knowledgeable in immune tolerance induction in Pompe disease to optimize treatment. Some patients who develop high and sustained IgG antibody levels, including certain IOPD patients, may have a reduced response to Lumizyme.
Monitoring: Laboratory Tests: Patients should be monitored for IgG antibody formation every 3 months for 2 years and then annually thereafter.
The most frequently reported adverse reactions during Lumizyme studies in patients were allergy reactions and included: anaphylaxis, rash, fever, flushing/feeling hot, hives, headache, excessive sweating, nausea, cough, less oxygen in the blood, fast heart rate, rapid breathing, chest discomfort, dizziness, muscle twitching, agitation, bluish or purple skin, redness of skin, high blood pressure/increased blood pressure, facial paleness, chills, tremor, vomiting, fatigue, and muscle pain.
Please see the Full Prescribing Information for complete details, including boxed WARNING.