Rare Daily Staff
CTD Holdings said it has received rare pediatric disease designation from the FDA for Trappsol Cyclo, its experimental drug to treat Niemann-Pick Disease Type C, a rare, fatal and progressive genetic disease.
NPC is a lysosomal storage disorder caused by a lack of ability to process cholesterol and other fatty substances inside of cells. As these substances buildup within cell throughout the body, it can impair the proper functioning or organs including the brain.
Trappsol Cyclo is CTD’s proprietary formulation of hydroxypropyl beta cyclodextrin, which has been found to increase lifespan and reduce symptoms in NPC animal models by stabilizing cholesterol metabolism, the primary defect in NPC.
The FDA grants Rare Pediatric Disease designation to therapeutics intended to treat serious or life-threatening rare diseases that primarily affect individuals under the age of 18. If CTD is successful in winning marketing approval for Trappsol Cyclo, it will receive a rare pediatric disease priority review voucher.
The voucher is potentially lucrative because it is transferrable and can be used to accelerate the approval of any drug. That could give the holder faster access to multibillion markets. In the past, such vouchers have sold for as much as $350 million. Most recently BioMarin sold a priority review voucher for $125 million.
“The incentives provided by the Rare Pediatric Disease designation are significant and include the potential to obtain a valuable Rare Pediatric Disease Priority Review Voucher upon approval,” said N. Scott Fine, CTD chairman and CEO. “We are eager to leverage these benefits and to work closely with the FDA and the NPC patient community as we pursue our goal of developing Trappsol Cyclo to treat this devastating rare disease.”
CTD said in named patient programs globally, the drug has been shown to be safe and well-tolerated in compassionate use settings. CTD currently supports two clinical trials to evaluate the intravenous administration of Trappsol Cyclo in NPC patients, a phase 1 in the United States, and a phase 1/2 in Europe and Israel.
December 6, 2017
Photo: N. Scott Fine, CEO of CTD Holdings