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.
CTD’s Chairman and CEO, N. Scott Fine, said, “CTD was the first to provide cyclodextrins for use in NPC patients on an expanded access basis in the United States, this was in 2009. 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.”
Niemann-Pick Type C Disease (NPC) is a rare and fatal genetic disease affecting 1 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.
“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 Dr. Hastings.
CTD’s Chief Scientific Officer and Senior Vice President for Medical Affairs Sharon Hrynkow PhD added, “We have learned a great deal about Trappsol® Cyclo™ in NPC through expanded access programs over the years: the positive safety and tolerability profile when the drug is administered intravenously coupled with the neurologic and systemic benefits as observed in multiple patients and by multiple physicians show the value of these kinds of programs for patients and for CTD.”
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 programs formed the basis of the clinical trial applications for the current trials underway in the US, Sweden, Israel and the United Kingdom.
Source: Company Press Release