Cyclo Therapeutics Announces Submission of New Drug Application to FDA for Trappsol® Cyclo™ (remibetadex) for the Treatment of Niemann-Pick Disease Type C

October 29, 2026

Dear NPC Organizational Leaders,

As we approach the conclusion of Niemann-Pick Disease Awareness Month, we are overjoyed to announce the submission of our New Drug Application (NDA) to the FDA for Trappsol® Cyclo™ (remibetadex, previously identified as intravenous hydroxypropyl-β-cyclodextrin). This NDA seeks approval for the treatment of the neurovisceral manifestations of NPC in adults and pediatric patients from birth. The full details on the NDA submission were announced in our press release that was issued earlier today.

We also expect to submit applications for marketing approval in other regions around the world and will share more details on those plans as we are able. It goes without saying, of course, that pending FDA’s decision on the filing of our NDA and the Agency’s substantive review of the data in the NDA, remibetadex remains an investigational product and has not been approved by the FDA or any other regulatory agency for any indication.

This milestone was only made possible by the commitment and perseverance of the entire NPC community – the patients and families, healthcare providers and advocacy leaders like you all over the world. With 94 patients studied across 27 sites in 13 countries, the TransportNPC™ study is the most comprehensive, placebo-controlled pivotal study – in terms of patient size, duration, and clinical outcomes measured – of an investigational therapy for NPC in history.

We first began evaluating this investigational therapy in patients to address symptoms of NPC back in 2009, and our commitment has not wavered. We wish to acknowledge just a few of the pioneers who believed in the promise of intravenous cyclodextrin and who paved the way for this milestone today, including Dr. Benny Liu and Dr. Caroline Hastings. We are also tremendously grateful to Chris and Hugh Hempel who are the epitome of the advocacy, resilience and hope that make up the backbone of the NPC community and their heroic girls Addi and Cassi who remain our inspiration today.

While October is Niemann-Pick Disease Awareness Month, there is not a day that goes by that our hearts and minds are not focused upon the unmet medical need that exists within the NPC community. Please accept our heartfelt gratitude for your support and partnership.

Kind regards,

The Team at Cyclo Therapeutics, a Rafael Holdings Company