New Drug Application for Niemann-Pick Disease Type C Treatment Submitted Under Rolling Review

According to a story from, the biopharmaceutical company Orphazyme A/S has recently announced that it has submitted its New Drug Application (NDA) for its experimental treatment arimoclomol to the US Food and Drug Administration (FDA) under rolling review. Arimoclomol is in development as a treatment for Niemann-Pick disease type C, a rare illness. The experimental treatment has previously earned Orphan Drug designation, Rare Pediatric Disease designation, Breakthrough Therapy designation, and Fast Track designation from the FDA.

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, making them 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 Rolling Review

As the application has been submitted under rolling review, this will allow the company to send the FDA sections of the NDA as they are completed. This allows for an accelerated review process as under normal circumstances Orphazyme would be expected to wait until the entire application is complete before submitting.

About Arimoclomol

Arimoclomol is an investigational therapy that activates the increased production of heat shock proteins. This mechanism of action has the potential to confer benefits to a variety of rare disease states; such benefits include improving the function of cell lysosomes, intervening in misfolded proteins, and clearing protein aggregations. It is an orally active treatment that is capable of passing through the blood-brain barrier. Aside from Niemann-Pick disease type C, the drug is also being investigated as a treatment for Gaucher disease, amyotrophic lateral sclerosis, and sporadic inclusion body myositis.

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