International Niemann–Pick Disease Alliance

CTD Holdings Receives FDA Fast Track Designation for Development of Trappsol® Cyclo™ to Treat Niemann-Pick Disease Type C


ALACHUA, FL — (Marketwired) – 1/17/17 — CTD Holdings, Inc. (OTCQB: CTDH), a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of disease, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Trappsol® Cyclo™ for the treatment of systemic Niemann-Pick Disease Type C (NPC), a rare and fatal genetic disease. Trappsol® Cyclo™ is the Company’s proprietary formulation of hydroxypropyl beta cyclodextrin in development for the treatment of NPC; it has previously been designated an Orphan Drug by the FDA and the European Medicines Agency.

The FDA’s Fast Track process is designed to facilitate the development and expedite the review of drugs that are intended to treat serious conditions and that demonstrate the potential to address an unmet medical need. Its purpose is to get important new drugs to patients earlier. Fast Track designation allows for more frequent interactions with the FDA during all aspects of a drug’s development to ensure the collection of appropriate data needed to support approval, and may result in eligibility for priority review and accelerated approval.

“We thank the FDA for granting Fast Track designation to Trappsol® Cyclo™ as a treatment for Niemann-Pick Disease Type C and look forward to working with the Agency as it reviews our clinical findings and expected marketing application,” said N. Scott Fine, Chairman and CEO of CTD. “We believe this is a very positive step for our development program that will support our efforts to make Trappsol® Cyclo™ available to NPC patients and their families as quickly as possible. Based on the data generated to date from this program, we believe Trappsol® Cyclo™ has significant potential in the treatment of NPC.”

NPC is a systemic disease that affects the brain, liver and other organs through abnormal accumulation of cholesterol in cells. It impacts primarily children, but is increasingly diagnosed in older patients who may live with this disability for many years.

About the Company:

CTD Holdings, Inc. is a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of disease. The company’s Trappsol® Cyclo™, an orphan-drug-designated product candidate in the United States and Europe, is in development to treat Niemann-Pick Disease Type C, a rare and fatal genetic disease. Additional indications for the active ingredient in Trappsol® Cyclo™ are also in development. For additional information, visit the company’s website:

Safe-Harbor Statement:

This press release contains “forward-looking statements” about the company’s current expectations about future results, performance, prospects and opportunities. Statements that are not historical facts, such as “anticipates,” “believes” and “expects” or similar expressions, are forward-looking statements. These statements are subject to a number of risks, uncertainties and other factors that could cause actual results in future periods to differ materially from what is expressed in, or implied by, these statements. The factors which may influence the company’s future performance include the company’s ability to obtain additional capital to expand operations as planned, success in achieving regulatory approval for clinical protocols, enrollment of adequate numbers of patients in clinical trials, unforeseen difficulties in showing efficacy of the company’s biopharmaceutical products, success in attracting additional customers and profitable contracts, and regulatory risks associated with producing pharmaceutical grade and food products. These and other risk factors are described from time to time in the company’s filings with the Securities and Exchange Commission, including, but not limited to, the company’s reports on Forms 10-K and 10-Q. Unless required by law, the company assumes no obligation to update or revise any forward-looking statements as a result of new information or future events.

Contact: Hans Vitzthum LifeSci Advisors, LLC 219-915-2568

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