trending Market Intelligence /marketintelligence/en/news-insights/trending/DxrslKutR7qgZEc4Kf8RBQ2 content esgSubNav
In This List

CTD Holdings medicine secures rare pediatric disease designation from US FDA

Blog

Insight Weekly: Recession risk persists; Banks pull back from crypto; 2022 laggard stocks rally

Blog

Highlighting the Top Regional Aftermarket Research Brokers by Sector Coverage

Blog

Insight Weekly: Inflation eases; bank M&A slows; top companies boost market share

Blog

Insight Weekly: PE firms shift strategies; bank earnings kick off; bankruptcies plummet


CTD Holdings medicine secures rare pediatric disease designation from US FDA

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.