Adults With Acid Sphingomyelinase Deficiency Have Sustained Improvements in Clinical Outcomes With up to 5 Years of Olipudase Alfa Enzyme Replacement Therapy: <scp>ASCEND</scp> Trial Final Results
) increased (mean 50.1% ± 10.8% at baseline vs. 66.5% ± 13.3% at final assessment; mean change from baseline of 35.9% ± 27.5% (p < 0.0001). Mean baseline spleen volume of 11.5 ± 4.6 multiples of normal (MN) decreased to 4.8 ± 2.1 MN at final assessment, mean change from baseline -57.5% ± 10.1% (p < 0.0001) and mean baseline liver volume (1.5 ± 0.4 MN) decreased to 0.95 ± 0.23 MN at final assessment, (mean change from baseline -36.8% ± 11.5%, p < 0.0001). Plasma lyso-sphingomyelin levels decreased by 72% from baseline to final assessment. Overall, improvements in clinical parameters occurred regardless of baseline severity. No new safety issues emerged during the trial extension and 98% of treatment emergent adverse events were mild/moderate. Improvements in visceral ASMD disease with olipudase alfa treatment will significantly impact the disease burden for those with this progressive multiorgan disorder.
Authors
- Norberto Guelbert (ORCID: https://orcid.org/0000-0003-3860-4750)
- Ebubekir Şenateş (ORCID: https://orcid.org/0000-0002-5804-7552)
- Michel Tchan (ORCID: https://orcid.org/0000-0001-9700-7898)
- Melissa Wasserstein (ORCID: https://orcid.org/0000-0003-2031-7817)
- Olivier Lidove (ORCID: https://orcid.org/0000-0003-4847-7839)
- Takayuki Ikezoe (ORCID: https://orcid.org/0000-0002-7858-278X)
- Antonio Barbato (ORCID: https://orcid.org/0000-0002-4660-821X)
- Monica Kumar (ORCID: https://orcid.org/0000-0002-1905-5955)
- Julia B. Hennermann (ORCID: https://orcid.org/0000-0003-2639-1233)
- Eugen Mengel
- Nicole M. Armstrong (ORCID: https://orcid.org/0000-0002-2479-1076)
- Paulina Mabe
- Beth L. Thurberg
- Yong Kim (ORCID: https://orcid.org/0000-0002-1387-2962)
- Robin Lachmann
- Renata C. Gallagher
- Jesus Villarrubia
- Carla E. Hollak (ORCID: https://orcid.org/0000-0003-0464-1078)
- Roberto Giugliani
- Maurizio Scarpa
- Shashi Bhushan
Institutions
- University of Hohenheim (DE)
- University of Udine (IT)
- Universidade Federal do Rio Grande do Sul (BR)
- Fukushima Medical University (JP)
- Morpho (United States) (US)
- University of California, San Francisco (US)
- Johannes Gutenberg University Mainz (DE)
- Morristown Medical Center (US)
- Westmead Hospital (AU)
- Sanofi (France) (FR)
- University Medical Center of the Johannes Gutenberg University Mainz (DE)
- Amsterdam Neuroscience (NL)
- Sanofi (United States) (US)
- Groupe Hospitalier Diaconesses Croix Saint-Simon (FR)
- Federico II University Hospital (IT)
- National Institute on Population Medical Genetics (BR)
- C3I (United States) (US)
- Children's Hospital at Montefiore (US)
- Clinica Santa Maria (CL)
- National Hospital for Neurology and Neurosurgery (GB)
- Hospital Universitario Ramón y Cajal (ES)
- University College London (GB)
- Istanbul Medeniyet University (TR)
- Cordoba University (US)
- University of Amsterdam (NL)
Publication Details
- Journal
- Journal of Inherited Metabolic Disease
- Published
- 2026-04-28
- DOI
- https://doi.org/10.1002/jimd.70192
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Sanofi