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Sanofi Reports Results of Olipudase alfa in Two Studies for Acid Sphingomyelinase Deficiency in Adults and Pediatric Patients

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Sanofi Reports Results of Olipudase alfa in Two Studies for Acid Sphingomyelinase Deficiency in Adults and Pediatric Patients

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  • The P-II/III study involves assessing olipudase alfa (up to 3mg/kg- IV- q2w) vs PBO in 36 adult patients with ASMD for 52wks. demonstrating reduction in spleen volume (39.5% vs 0.5%); reduction in SRS (8.0 vs 9.3)
  • The P-II study involves assessing of olipudase alfa (up to 3mg/kg- IV- q2w) in 20 pediatric patients aged <18yrs. across six countries for 64wks. demonstrating a 33% increment in predicted DLco in 9 patients who performed the test at baseline @52wks. and a 49% reduction in spleen volume assessed by MN
  • Olipudase alfa is an investigational enzyme replacement therapy designed to replace deficient/ defective ASM- allowing the breakdown of sphingomyelin with its expected global regulatory submission to be initiated in H2’21

Click here ­to­ read full press release/ article | Ref: Sanofi | Image: Reuters


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