NNPDF Celebrates Monumental Milestone with the First U.S. Approval for ASMD

September 2022

The National Niemann-Pick Disease Foundation joins with patients and families in the U.S. and the entire global Niemann-Pick community in celebrating this historic advance in research: the first-ever U.S. approval of a treatment for ASMD!

For more than 20 years, NNPDF has worked to support patients and families impacted by ASMD (acid sphingomyelinase deficiency) by raising awareness and advocating for research that can lead to life-changing therapies. While we have faced many challenges in the effort to bring new treatment options to patients who desperately need them, today we can see a sign of hope for the ASMD community and for all patients living with Niemann-Pick disease.  

The U.S. Food and Drug Administration (FDA) has announced the approval of Xenpozyme (olipudase alfa) for intravenous infusion in pediatric and adult patients with ASMD. Xenpozyme is an enzyme replacement therapy that has been shown to help reduce sphingomyelin accumulation in the liver, spleen, and lung. The efficacy of Xenpozyme for the treatment of ASMD was demonstrated in a controlled study of 31 patients. Based on results from the study, the FDA was able to conclude that Xenpozyme is effective and that treatment helped to improved lung function and reduced liver and spleen size in patients.

NNPDF would like to take this opportunity to recognize the hard work of many people including regulatory and industry leaders who have made this milestone possible. We applaud Sanofi for their commitment to moving this program forward. We also want to emphasize that this approval would not have been possible without the patients and families who participate in clinical research and who use their voices and stories to raise awareness of the need for treatments and support.

More than anything, this life-changing moment is an example of what we can accomplish when we work together. Hopefully, this will bring new levels of momentum and dedication to promising research moving forward. The Niemann-Pick community is resilient and thanks to the bravery and persistence of patients and families, people living with ASMD in the U.S., Europe and Japan can now have access to an approved treatment.

We are so happy to see this amazing progress and will use this to fuel us on our journey to finding approved treatment options for all people impacted by all types of Niemann-Pick.

Supporting one another. Supporting our community.