Article

Acid Sphingomyelinase Deficiency Receives First FDA Approved Therapy

Author(s):

Olipudase alfa (Xenpozyme) was developed by Sanofi to treat symptoms unrelated to the central nervous system in pediatric and adult patients with the life-threatening condition.

Acid Sphingomyelinase Deficiency Receives First FDA Approved Therapy

The rare genetic disease, acid sphingomyelinase deficiency (ASMD), was just granted its first approved treatment by the US Food and Drug Administration (FDA). Olipudase alfa (Xenpozyme) was developed by Sanofi to treat symptoms unrelated to the central nervous system in pediatric and adult patients with the life-threatening condition.

Caused by the inability to break down the complex liquid sphingomyelin due to an insufficient amount of the necessary enzyme, ASMD affects the liver, spleen, lung and brain, which can make it life-threatening. These patients experience painful symptoms, and even those that survive into adulthood can face respiratory failure that lead to premature death.

“ASMD has a debilitating effect on people’s lives and there is a critical need to increase treatment options for patients who suffer from this rare disease,” Christine Nguyen, MD, Deputy Director, Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, FDA Center for Drug Evaluation and Research, said in a statement.

Leading up to this decision, the FDA granted olipudase alfa orphan drug designation, as well as fast track, breakthrough therapy, and priority review designations. Additionally, the agency aimed to encourage further development of new drugs and biologic therapies that target rare diseases in children by awarding Sanofi a rare pediatric disease priority review voucher.

“The challenges involved with developing treatments for rare diseases are significant and unique. We believe patients who suffer from ASMD, their families and their physicians will welcome this long-awaited advancement,” Nguyen stated.

Related Videos
Christine N. Kay, MD | Image Credit: Atsena Therapeutics
Christine N. Kay, MD: Interim Data on ATSN-201 Shows Promise for XLRS | Image Credit: Vitreo Retinal Associates
How Artificial Intelligence is Being Used in Lung Imaging, with Rachel Eddy, PhD
Roger A. Goldberg, MD: Pooled Visual Function Data of NT-501 for MacTel | Image Credit: Bay Area Retina Associates
Developing Risk Assessment Tools for Viruses in School
Using Microbiomes to Diagnose Ventilator-Associated Pneumonia
What Do Patients Need to Learn About their Hypersensitivity Pneumonitis?
Discussing Use of Vaping Among Students, Conversations About Vaccines
© 2024 MJH Life Sciences

All rights reserved.