Policy & Regulation
CTD Holdings Gains US FDA Approval of US Expanded Access Treatment Programme in Niemann-Pick Disease Type C
18 September 2019 - - US-based biotechnology company CTD Holdings, Inc. (OTCQB: CTDH) will provide Trappsol Cyclo, its proprietary hydroxypropyl beta cyclodextrin drug, to a pediatric patient diagnosed with Niemann-Pick Disease Type C, the company said.

The company received notice TODAY of the FDA approval of the individual IND application from the treating physician, Caroline Hastings, MD, pediatric hematologist/oncologist at the UCSF Benioff Children's Hospital in Oakland, CA.

Niemann-Pick Type C Disease is a rare and fatal genetic disease affecting one in 100,000 live births globally. NPC affects every cell in the body due to the defect in the NPC protein which is responsible for cholesterol processing in the cell.

Because of the NPC defect in this disease, cholesterol accumulates abnormally in every cell in the body, causing symptoms in the brain, liver, spleen, lung and other organs.

There are no approved drug therapies for NPC in the United States.

CTD is currently developing Trappsol Cyclo as a treatment for NPC in 2 main clinical trials, one based in the United States (a Phase I study) and one based in Europe and Israel (a Phase I/II study).

Both trials are nearing completion of enrollment, and design of the pivotal trial is underway.

Dr. Hastings serves as the co-principal investigator for the Phase I study in the US in addition to her role as Senior Clinical Advisor to the Phase I/II study.

She is also the first physician in the US to administer cyclodextrins (CTD's product) to NPC patients on an expanded access basis.

Dr. Hrynkow and Dr. Hastings have presented at several scientific and medical conferences on the outcomes of CTD's longstanding expanded access programs globally using Trappsol Cyclo by intravenous administration in NPC patients.

Data from the company's expanded access programmes formed the basis of the clinical trial applications for the current trials underway in the US, Sweden, Israel and the United Kingdom.

CTD Holdings, Inc. is a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of disease.

The company's Trappsol Cyclo, an orphan drug designated product in the United States and Europe, is used to treat Niemann-Pick Disease Type C, a rare and fatal genetic disease, on a compassionate use basis as well as in three ongoing formal clinical trials (Clinical Trials.gov NCT02939547, NCT02912793 and NCT03893071).