First Treatment for ASMD approved by FDA
The US FDA has announced approving Xenpozyme (Olipudase alfa), for intravenous infusion in pediatric and adult patients with Acid Sphingomyelinase Deficiency (ASMD). The medication is the first to treat symptoms that are not related to the central nervous system in patients with ASMD. ASMD is a rare genetic disease, that is caused by the lack of an enzyme needed to break down a complex lipid, called sphingomyelin, that accumulates in the liver, spleen, lung, and brain. Patients with ASMD suffer from enlarged abdomens that can cause pain, vomiting, feeding difficulties, and falls. The severe type of ASMD causes profound neurologic symptoms, and accordingly affected patients prematurely die at the age of 2 or 3 years. Other patients can survive into adulthood, however, they also die prematurely from respiratory failure. Xenpozyme is an enzyme replacement therapy that decreases sphingomyelin accumulation in the liver, spleen, and lung. The efficacy of Xenpozyme as ASMD treatment was shown in a randomized, double-blind, placebo-controlled study of 31 patients randomized between Xenpozyme and placebo. As the study had the placebo comparator and measured treatment benefits that could be detected during the study’s duration, the FDA concluded that Xenpozyme is effective. Overall, treatment with Xenpozyme improved lung function and reduced liver and spleen size.
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Reference: FDA Approves First Treatment for Acid Sphingomyelinase Deficiency, a Rare Genetic Disease. (2022). Retrieved 4 September 2022, from https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-acid-sphingomyelinase-deficiency-rare-genetic-disease