The US Food and Drug Administration (FDA) today announced approval of alglucosidase alfa (Lumizyme, Genzyme) for treatment of infantile-onset Pompe disease, including for patients younger than 8 years.
The agency has also eliminated a risk evaluation and mitigation strategy (REMS) called the Lumizyme ACE (Alglucosidase Alfa Control and Education) Program. The drug, a lysosomal glycogen-specific enzyme, was approved in 2010, but the REMS restricted its use to late, not infantile-onset Pompe disease in patients aged 8 years and older, a statement from FDA notes. "This approval provides access to Lumizyme for all Pompe disease patients, regardless of their age," the statement said.
The REMS was required to mitigate the potential risk for rapid disease progression in the patients with infantile-onset Pompe disease and patients with late-onset disease younger than 8 years, and to communicate the risks for anaphylaxis, severe allergic reactions, and severe skin and systemic immune-mediated reactions to prescribers and patients, the agency writes.
"At the time of Lumizyme's approval, there were insufficient data to support the safety and efficacy of Lumizyme in the infantile-onset Pompe population, so Lumizyme was approved for use only in late onset Pompe disease patients who are at least 8 years of age," the FDA said. "Pompe patients with infantile-onset disease and patients younger than 8 years of age continued treatment with Myozyme, which was already approved. Myozyme and Lumizyme, both manufactured by Genzyme Corporation, are produced from the same cell line at different production scales."
The FDA reviewed newly available information and determined that these agents are chemically and biochemically comparable, so their safety and effectiveness are also expected to be comparable, they note. In addition, a single-center clinical study of 18 patients with infantile-onset Pompe disease, aged 0.2 to 5.8 months at the time of first infusion, provides further support that infantile-onset patients treated with this drug will have a similar improvement in ventilator-free survival as those treated with Myozyme.
Because data were submitted supporting approval of Lumizyme for all patients with Pompe, the agency decided a REMS restricting its use to a specific age group is no longer necessary. "While the risk of anaphylaxis, severe allergic reactions, and severe cutaneous and immune mediated reactions for Lumizyme still exist, these risks are comparable to Myozyme and are communicated in labeling through the Warnings and Precautions, and a Boxed Warning," the statement said.
"REMS continue to be vital tools for the agency to employ as we work with companies to address the serious risks associated with drugs and monitor their appropriate and safe use in various health care settings," said Janet Woodcock, MD, director of the FDA's Center for Drug Evaluation and Research in the statement. "The agency remains committed to exercising a flexible and responsible regulatory approach that ensures REMS programs are being effectively and efficiently used and not resulting in an unnecessary burden on health care professionals and patients."
The statement adds that healthcare professionals and patients should also be aware that:
The warnings and precautions section of the Lumizyme product label and the clinical studies section of the Lumizyme label have been updated to include the safety information of the drug in patients with infantile-onset Pompe disease. This includes information from the currently approved Myozyme label and information from a new, uncontrolled study in which patients with infantile onset disease were treated with Lumizyme.
Lumizyme is approved with a boxed warning because of the risk for anaphylaxis, severe allergic reactions, immune-mediated reactions, and cardiorespiratory failure.
Healthcare professionals should continue to refer to the drug prescribing information for the latest recommendations on prescribing Lumizyme and report adverse events to the FDA's MedWatch program.
Distribution of Lumizyme will no longer be restricted. Healthcare professionals, healthcare facilities, and patients will no longer be required to enroll in the REMS program to be able to prescribe, dispense, or receive the drug.
The most commonly reported adverse effects for the drug were infusion-related reactions and included severe allergic reactions, hives, diarrhea, vomiting, shortness of breath, itchy skin, skin rash, neck pain, partial hearing loss, flushing, pain in extremities, and chest discomfort.
Pompe disease is a rare genetic disorder and occurs in an estimated 1 in every 40,000 to 300,000 births. Its primary symptom is heart and skeletal muscle weakness, progressing to respiratory weakness and death from respiratory failure.
The disease causes gene mutations to prevent production of sufficient amounts of the functional form of acid alpha-glucosidase, an enzyme necessary for proper muscle functioning. Without the enzyme action, glycogen builds up in the cells and, ultimately, weakens the heart and muscles. Lumizyme is believed to work by replacing the deficient acid alpha-glucosidase, thereby reducing the accumulated glycogen in heart and skeletal muscle cells.
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Cite this: FDA Expands Approval of Pompe Disease Drug - Medscape - Aug 01, 2014.