Clinical Trial Results Look Promising For Experimental Niemann-Pick Disease Type C Treatment

According to a story from BioSpace, the biopharmaceutical company Orphazyme recently announced the results from its Phase 2/3 clinical trial of its investigational drug arimoclomol. This therapy is in development for the treatment of Niemann-Pick disease type C, and the results from the trial suggest that it could be an effective treatment. Orphazyme is committed to the development of therapies designed to treat rare diseases.

About Niemann-Pick disease type C

Niemann-Pick disease type C is a form of lysosomal storage disease which is characterized by a deficiency not in an enzyme, but most typically in a type of transporter protein that prevents water soluble molecules from moving within a cell. It is caused by mutations of either the NPC1 or NPC2 gene. There is broad disparity in the severity and presentation of symptoms in Niemann-Pick disease type C, making symptoms an unreliable method for diagnosis. They may appear in childhood or as late as a patient’s sixth decade of life. Such symptoms include spleen and/or liver enlargement, jaundice, severe depression, ataxia, epilepsy, difficulty speaking and swallowing, dystonia, poor muscle tone, bipolar disorder, microcephaly, progressive loss of hearing, progressive dementia, and psychosis. Most treatment is supportive, but there are some medicines that can delay disease progression and prolong life. Lifespan is connected to the onset of symptoms, with those with the earliest symptoms usually dying sooner. To learn more about Niemann-Pick disease type C, click here.

About The Results So Far

The study was a year in duration and involved a total of 50 patients. In the trial, arimoclomol was administered alongside the patient’s current standard of care and was compared alongside a placebo group. Based on the primary endpoint, the initial data revealed a reduction in progression of 74 percent. 

This data is still only the initial top line, but the remaining information from the study will be available by the end of the year. An open label extension trial is also ongoing, and is set to continue for a duration of 24 months. Data from it should appear by mid 2019.

Overall, patients should be intrigued by the data results so far, as arimoclomol has the potential to be a brand new treatment option for Niemann-Pick disease type C.


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