FDA approved lumizyme for all ages of patients with Pompeii's disease

Source: Ding Xiangyuan August 1, 2014, FDA of the United States announced that it approved the use of glucosidase α (lumizyme) in the treatment of infantile paroxysmal Pompeii disease, including patients under 8 years old In addition, the lumizyme ace risk assessment and mitigation strategy (REMS) program was cancelled Pompeii's disease is a rare genetic disease that is estimated to occur in one out of every 40000 to 300000 births The main symptom of this disease is the weakness of heart and skeletal muscle, but it develops to the weakness of respiration until death due to respiratory failure The disease is caused by mutations that prevent the body from producing enough functional enzymes, acid alpha glucosidase (GAA) This enzyme is necessary for normal muscle function GAA is used by heart and muscle cells to convert glycogen into energy Without this enzyme, glycogen accumulates in cells, eventually shrinking the heart and muscles Lumizyme works by replacing defective GAA, thereby reducing glycogen accumulation in heart and skeletal muscle cells Lumizyme, a lysosomal specific glycogen enzyme, was approved by the FDA in 2010, along with a REMS to limit its use in patients 8 and older with delayed (non infant) Pompeii REMs requires reducing the potential risk of rapid disease progression in infants with paroxysmal Pompeii's disease and in patients under 8 years of age, and communicating with the prescriber and patients about the risks of allergies, severe allergic reactions, severe skin and systemic immune-mediated reactions When lumizyme was approved, there was not enough data to support the safety and effectiveness of lumizyme in the treatment of infantile paroxysmal Pompeii, so lumizyme was only approved for patients at least 8 years old with delayed Pompeii FDA reviewed the most available information and determined that lumizyme and Myozyme are comparable in chemical and biological learning Therefore, the safety and effectiveness of lumizyme and Myozyme are expected to be comparable In addition, a single center clinical study involving 18 infants with paroxysmal Pompeii disease (0.2 to 5.8 months old at the time of the first injection) provided further support, and infants treated with lumizyme will have similar improvement in ventilator free survival as those treated with Myozyme Since the data were submitted to support the approval of lumizyme for use in all patients with Pompeii's disease, it is no longer necessary to limit its use in REMS for specific age groups However, the risks of allergies, severe allergic reactions, severe skin and systemic immune-mediated reactions caused by lumizyme still exist, so these risks are the same as those of Myozyme, and need to be communicated through warnings and precautions and a black box warning in the label "When we work with pharmaceutical companies to address serious drug-related risks and monitor their appropriate and safe use in a variety of health care settings, REMS is still a key tool used by the FDA," said Woodcock, MD, director of the FDA's Center for drug evaluation and research "FDA remains committed to implementing a flexible and responsible regulatory approach to ensure that the REMS program is used effectively and efficiently, with no unnecessary burden on healthcare professionals and patients." Health care professionals and patients should also pay attention to the following points: the warning and precautions part of lumizyme product label and the clinical research part of lumizyme label have been updated, including the safety information of drugs used for infants with paroxysmal Pompeii disease This includes information from the currently approved Myozyme label and new, non controlled studies involving infants with seizures treated with lumizyme Lumizyme is approved with a black box warning due to its risk of allergies, severe allergic reactions, severe skin and systemic immune-mediated reactions Health care professionals should continue to refer to the latest recommended lumizyme prescription information and report adverse events to the FDA pharmacovigilance program Lumizyme's distribution will no longer be restricted Health care professionals, health care facilities, and patients are no longer required to participate in lumizyme REMS programs that can be issued, distributed, or accepted by lumizyme The most commonly reported side effects of lumizyme are infusion related reactions, including severe anaphylaxis, urticaria, diarrhea, vomiting, shortness of breath, pruritus, rash, neck pain, partial hearing loss, blushing, limb pain, and chest discomfort Myozyme and lumizyme are marketed by jianzan in Cambridge, Massachusetts.