Cyclo Therapeutics Announces Positive Outcome from Type C Meeting with the U.S. FDA Discussing Trappsol® Cyclo™ Clinical Program for the Treatment of Niemann-Pick Disease Type C1
Read the full statement from Cyclo Therapeutics here:
Here is a summary of the article:
FDA agrees to change the timing of the interim analysis for the Phase 3 study: The company will conduct a comparative interim analysis after 48 weeks of treatment, instead of 96 weeks, based on suggestive evidence of therapeutic benefit and significant unmet need for NPC patients.
TransportNPC™ study is a randomized, double-blind, placebo-controlled trial: The study aims to enroll at least 93 pediatric and adult patients with NPC and evaluate the safety, efficacy, and tolerability of Trappsol ® Cyclo™ administered intravenously and standard of care, compared to placebo and standard of care alone2.
Trappsol ® Cyclo™ has received multiple designations from the FDA and the EU: The product has been granted Orphan Drug, Fast Track, and Rare Pediatric Disease Designations in the U.S. and Orphan Drug Designation in the EU3. The Rare Pediatric Disease Designation is a requirement for obtaining a Priority Review Voucher in the U.S4.
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