According to a story from Globe Newswire, The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recently announced a positive opinion in regards to the experimental treatment olipudase alfa (also called Xenpozyme). This is a form of enzyme replacement therapy being developed as a treatment for acid sphingomyelinase deficiency (ASMD), a rare disease. The treatment is intended to treat manifestations of the illness not related to the central nervous system.

About Acid Sphingomyelinase Deficiency (ASMD)

Acid sphingomyelinase deficiency was previously thought to be two diseases: Niemann-Pick disease type A and Niemann-Pick disease type B. More recent research has determined that these two illnesses were in fact extreme spectrums of a single illness. What was previously known as type A is a very severe disease that begins causing problems in infancy; patients experience failure to thrive, jaundice, enlarged liver, and progressive degeneration of the nervous system. Most patients don’t live beyond 3 years of age. What was previously called type B is less severe. This form can present anywhere from early childhood to later on in life. Symptoms include enlarged liver and spleen, problems with lung function, lung infections, inhibited growth, low platelet count, and abnormal cholesterol and lipid levels. Unlike type A, the central nervous system is usually spared in type B.  Nevertheless, life-threatening complications still appear. There are no disease-altering therapies available for ASMD. To learn more about this disease, click here.

This recommendation from the committee follows encouraging results from two clinical trials in which olipudase alfa was able to demonstrate several beneficial effects for patients, such as reduction in liver and spleen volumes as well as improvements in lung function. It also demonstrated a favorable safety profile in both adult and child patients.

About Olipudase Alfa

Olipudase alfa has also earned PRIority MEdicines (PRIME) designation, and was under EMA accelerated assessment for review. It is form of enzyme replacement therapy intended to substitute for defective or deficient acid sphingomyelinase, an enzyme which is critical for breaking down sphingomyelin. In the disease, build up of sphingomyelin can cause serious, life-threatening organ damage. It has only been studied in patients with type B and type A/B forms of acid sphingomyelinase deficiency.