According to a story from globenewswire.com, the US Food and Drug Administration (FDA) recently accepted the New Drug Application (NDA) for arimoclomol. This application is for the use of the drug as a treatment for Niemann-Pick disease type C. The drug is being developed by the biopharmaceutical company Orphazyme A/S. The application was accepted under Priority Review protocols.
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 the disease, 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 Priority Review
Priority Review is permitted for applications in which the agency determines that the medication in question could offer significant improvements in treatment, diagnosis, safety, or prevention for a serious disease or medical condition. The period of review for the application is reduced from ten months to six months. The target action date for this application March 17, 2021.
Arimoclomol is designed to trigger the increased production of heat shock proteins. These proteins have the ability to improve the function of cellular lysosomes, repair misfolded proteins, and get rid of unusual protein aggregates, which are often implicated in neurodegenerative illnesses. The drug is capable of crossing the blood-brain barrier and is orally available. The drug has earned Breakthrough Therapy and Fast Track designations for Niemann-Pick disease type C from the FDA and is also being evaluated for Gaucher disease, sporadic inclusion body myositis, and amyotrophic lateral sclerosis.