According to a story from Globe Newswire, olipudase alfa (marketed as Xenpozyme) has been approved by the European Commission for the treatment of non-CNS related symptoms of acid sphingomyelinase deficiency (ASMD), a rare disease. This covers both adult and pediatric patients with either type A or type A/B. Olipudase alfa is a form of enzyme replacement therapy and is the first therapy of this type to be approved for this indication. This therapy was first approved in Japan in March of this year.
About Acid Sphingomyelinase Deficiency
Acid sphingomyelinase deficiency (ASMD) 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.
Trial Findings
The approval follows encouraging results in trials. Patients treated with olipudase alfa saw improvements in lung function as well as reduced liver and spleen volumes. The drug was also well tolerated from a safety standpoint. In the trial 36 adult patients with either type A or type A/B received either a placebo or olipudase alfa for a 52 week period. Patients saw a 22 percent improvement in lung function vs just 3 percent for placebo and a 39.5 percent drop in spleen size.
A similar study was undertaken with pediatric patients. 20 patients were involved and received treatment for 64 weeks. Of the nine patients that could perform the baseline evaluation, lung function improved by 33 percent. Mean decrease in spleen volume was 49 percent.
