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First ASMD treatment Xenpozyme to land in Korea
by Eo, Yun-Ho Nov 24, 2022 05:51am
The first-ever pediatric storage disorder treatment is expected to be introduced to Korea. According to industry sources, Sanofi Genzyme’s treatment for acid sphingomyelinase deficiency (ASMD) ‘Xenpozyme (olipudase alfa)’ is undergoing processes for marketing authorization by the Ministry of Food and Drug Safety. Starting with Japan in March, the drug was also approved in Europe in July and by the US FDA in August and received Breakthrough Therapy designation in the countries. The efficacy of Xenpozyme, the only existing ASMD treatment, was identified through the ASCEND and ASCEND-Peds trials. The ASCEND trial evaluated the efficacy and safety of Xenpozyme in 36 adult patients with ASMD type A/B or type B. The patients were randomized to receive Xenpozyme or placebo for 52 weeks (primary analysis). At Week 52, Xenpozyme improved lung function from baseline by 22% in terms of the predicted diffusing capacity of the lung for carbon monoxide (DLco). Compared with the 3% improvement shown in the placebo group, the difference between the two treatment arms of 19% was statistically significant. Also, at Week 52, patients treated with Xenpozyme had a mean reduction in spleen volume by 39.5% compared with the 0.5% increase in the placebo group. All patients that were treated with Xenpozyme showed an improvement in one or two primary endpoints. The single-arm ASCEND-Peds trial studied 20 pediatric patients younger than 12 years of age with ASMD type A/B or type B. The primary objective of the trial was to evaluate the safety and tolerability of Xenpozyme for 64 weeks, and the explored efficacy endpoints of progressive lung disease, spleen, and liver enlargement, and platelet count were also explored in the trial. 9 patients who could take the test for diffusing capacity of the lung for carbon monoxide in the trial showed a 33% improvement in diffusing capacity after 1 year. The patients also showed a mean reduction in spleen volume of 49%. ASMD is caused by the lack of an enzyme needed to break down a complex lipid, called sphingomyelin, which accumulates in the liver, spleen, lung, and brain. Patients with ASMD experience enlarged abdomens that can cause pain, vomiting, feeding difficulties, and falls at 3 to 6 months of birth. The most severely affected patients have profound neurologic symptoms and rarely survive beyond two to three years of age.