Genzyme's Xenpozyme approved for Acid Sphingomyelinase Deficiency | iPharmaCenter

Sanofi announced that the U.S. Food and Drug Administration (FDA) approved Xenpozyme (olipudase alfa) for treating acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients. It is indicated for treating non-central nervous system (non-CNS) manifestations of ASMD.




The approval was based on ASCEND and ASCEND-Peds clinical trials.

In the ASCEND trial, 31 adult patients with ASMD type A/B or type B were randomized between placebo and Xenpozyme.

  • Twelve patients treated with Xenpozyme had improvement in the diffusing capacity of the lung for carbon monoxide (DLco), from 49.1% to 59.4%. The improvement in DLco in the placebo group was 48.5% to 49.9%. There was a nominally statically significant improvement between Xenpozyme and the placebo group.

  • In terms of mean reduction in spleen volume, there was a reduction of 38.9% at week 52 in the Xenpozyme arm. There was an increase of 0.5% in patients on placebo, with a difference of 39.4% between both arms.

  • There was an improvement in platelet count by 18.3% from the baseline versus 2.7% in patients on the placebo.

  • The most commonly reported adverse events were headache, cough, diarrhea, hypotension, and ocular hyperemia.

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ASCEND-Peds is a single-arm trial with eight pediatric patients younger than 12 years. After 52 weeks of treatment

  • There was an improvement in predicted DLco by 45.9%.

  • In eight patients, spleen volume was reduced by 46.7%.

  • There was a mean reduction in liver volume by 38.1%.

  • The platelet count was improved by 37.6% from baseline.

  • The most commonly reported adverse events were pyrexia, cough, diarrhea, rhinitis, abdominal pain, vomiting, headache, urticaria, nausea, rash, arthralgia, pruritus, fatigue, and pharyngitis.

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ASMD is previously known as Niemann-Pick disease types A, A/B, and B. It is a rare disease with significant morbidity and mortality. Xenpozyme received Breakthrough Therapy designation, which will be given for treating severe and life-threatening conditions. It is evaluated under Priority Review. Previously, it was approved in Japan under the SAKIGAKE designation. In June, it was approved in Europe.

Less than 120 patients were diagnosed with ASMD in the U.S, out of which two-thirds are pediatric patients.



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