FDA Approves a Program for Expanded Treatment Access in Niemann-Pick Disease Type C

According to a story from ir.stockpr.com, the biotechnology company CTD Holdings, Inc., recently announced that the US Food and Drug Administration (FDA) has issued an approval for an individual Investigational New Drug (IND) application from a physician for the company’s drug Trappsol Cyclo. The drug will be used to treat a child patient with Niemann-Pick disease Type C, a rare disease. The physician in the case is Dr. Caroline Hastings, who is affiliated with the University of California San Francisco’s Benioff Children’s Hospital.

About Niemann-Pick Disease Type C (NPC)

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 this 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 this rare disease, click here.

Providing Treatment Using Expanded Access

CTD is focused on the development of cyclodextrin-based therapeutics for diseases with high unmet need. This is the second time that the company has provided its medications for Niemann-Pick disease type C under expanded access protocols; the first time was a decade ago, in 2009. The company’s experience with expanded access programs has given it greater insight into the potential capabilities of its drugs.

Trappsol Cyclo is currently being evaluated in two different clinical trials in Europe and the US as a treatment for Niemann-Pick disease type C. In expanded access situations, the therapy has shown positive safety and efficacy.

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