RARE Daily

FDA Approves CTD Holdings Expanded Access Treatment Program in NPC

September 17, 2019

CTD Holdings said that the company will provide Trappsol Cyclo, its proprietary hydroxypropyl beta cyclodextrin drug, to a pediatric patient diagnosed with Niemann-Pick Disease Type C, a rare and fatal lysosomal storage disorder.

Niemann-Pick Type C Disease (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.

The company received notice today of the FDA approval of the individual Investigational New Drug application from the treating physician, Caroline Hastings, a pediatric hematologist/oncologist at the UCSF Benioff Children’s Hospital in Oakland, California.

“For patients who are not eligible for ongoing clinical trials, expanded access programs such as this one are a critical means for them to receive experimental therapies,” said Hastings.

In 2009, CTD became the first company to provide cyclodextrins for use in NPC patients on an expanded access basis in the United States.

CTD is currently developing Trappsol Cyclo as a treatment for NPC in two 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. Hastings serves as the co-principal investigator for the phase I study in the United States and is also a senior clinical advisor to the phase 1/2 study.

“We are pleased to once again offer our product for intravenous administration to this pediatric patient on an expanded access basis, even as we advance our formal clinical trials for registration of the drug for the NPC indication,” said N. Scott Fine, chairman and CEO of CTD.

Photo: Caroline Hastings, a pediatric hematologist/oncologist at the UCSF Benioff Children’s Hospital

Author: Rare Daily Staff

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