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Sanofi’s olipudase alfa fares well in open-label extension ASMD studies

By Brian Buntz | February 9, 2022

SanofiSanofi (NSDQ:SNY) has announced that olipudase alfa offered a sustained improvement in patients with acid sphingomyelinase deficiency (ASMD) in investigational long-term follow-up studies.

ASDM is a rare progressive and potentially life-threatening disease with no authorized treatments.

Patients in the trial had improvements in terms of lung function and reduced spleen and liver volumes.

The research focused on non-central nervous system (non-CNS) manifestations of ASDM.

A sphingomyelin-phosphodiesterase replacement, olipudase alfa, has been the subject of seven clinical trials to date.
To date, treatment of ASDM, also known as ASM-deficient Niemann-Pick disease, depends on whether patients have a manifestation known as Niemann-Pick disease type A (NPD-A) or Niemann-Pick disease type B (NPD-B).

Patients with the first type may receive physical and occupational therapy and nutritional consultations.

Some individuals with type B disease receive treatment for dyslipidemia, nutritional consultations and, occasionally, blood transfusions.

A number of clinical trials are investigating the potential of enzyme replacement therapy in treating adults with the disease.

Sanofi notes that if olipudase alfa is approved, it would become the first therapy for ASDM.


Filed Under: clinical trials, Drug Discovery
Tagged With: acid sphingomyelinase deficiency, ASDM, olipudase alfa, Sanofi
 

About The Author

Brian Buntz

As the pharma and biotech editor at WTWH Media, Brian has almost two decades of experience in B2B media, with a focus on healthcare and technology. While he has long maintained a keen interest in AI, more recently Brian has made making data analysis a central focus, and is exploring tools ranging from NLP and clustering to predictive analytics.

Throughout his 18-year tenure, Brian has covered an array of life science topics, including clinical trials, medical devices, and drug discovery and development. Prior to WTWH, he held the title of content director at Informa, where he focused on topics such as connected devices, cybersecurity, AI and Industry 4.0. A dedicated decade at UBM saw Brian providing in-depth coverage of the medical device sector. Engage with Brian on LinkedIn or drop him an email at bbuntz@wtwhmedia.com.

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