▎WuXi AppTec Content Team Editor
Today, the US FDA announced the approval of Xenpozyme (olipudase alfa), an innovative enzyme replacement therapy from Genzyme, a subsidiary of Sanofi, for the intravenous infusion of acid sphingomyelinase deficiency ( acid sphingomyelinase deficiency, ASMD) in adult and pediatric patients. This drug is the first FDA-approved drug for non-central nervous system symptoms in ASMD patients.
ASMD patients lack the enzyme that breaks down sphingomyelin, which causes this complex lipid molecule to accumulate in the liver, spleen, lungs and brain, resulting in an enlarged abdomen and Causes symptoms such as pain, vomiting, difficulty eating and falling easily. Patients often have abnormal liver and blood test values. Those with the most severe disease develop neurological symptoms and most do not survive beyond 2-3 years of age. The remaining patients may survive into adulthood, but eventually die prematurely from respiratory failure.
Xenpozyme is an enzyme replacement therapy to replace missing or defective acid sphingomyelinase. This drug degrades sphingomyelin, which helps reduce the accumulation of this lipid in the liver, spleen and lungs. Xenpozyme has obtained Fast Track designation, Breakthrough Therapy designation, Priority Review designation and Orphan Drug designation from the US FDA. In addition, this drug was also obtained in March and June this year, respectively.
Ministry of Health, Labour and Welfare (MHLW)
Approved by the European Commission (EC) for the treatment of non-central nervous system symptoms in adults and pediatric patients with ASMD.
Image source: 123RF
Xenpozyme’s FDA approval was based on a randomized, double-blind, placebo-controlled trial of 31 ASMD patients. Overall, the drug was effective in improving lung function and reducing the size of the liver and spleen. The most common side effects of Xenpozyme include headache, cough, fever, joint pain, diarrhea, and low blood pressure, with the potential for severe allergic reactions.
“ASMD can have a debilitating impact on patients’ lives, so there is a critical need to increase treatment options for those suffering from this rare disease,” FDA Center for Drug Evaluation and Research Dr Christine Nguyen, Deputy Director, Office of Rare Diseases, Pediatrics, Urology and Reproductive Medicine (ORPURM) at (CDER) said, “The challenges of developing drugs for rare diseases are enormous and unique. We believe that patients suffering from ASMD and his family and doctors will welcome this long-awaited development.”