CTD Holdings, Inc. secured a rare pediatric disease designation for Trappsol Cyclo from the U.S. Food and Drug Administration.
The medication is used in the treatment of a rare and fatal genetic disease, Niemann-Pick type C, that affects the brain, lung, liver, spleen and other organs.
The rare pediatric disease designation is granted to drugs that have the potential to treat orphan diseases affecting fewer than 200,000 patients in the U.S. and with more than 50% of those affected age 18 years or younger.
The medication is undergoing a phase 1 trial in the U.S. and a phase 1/2 trial in Europe and Israel. The company recently filed an application to conduct a clinical trial in Italy.
The treatment earlier secured orphan drug designations in the U.S. and the European Union.