This section highlights FDA-related milestones and regulatory updates for drugs developed by Cyclo Therapeutics (CYTH).
Over the past two years, Cyclo Therapeutics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
Trappsol and Trappsol®. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Trappsol Cyclo (AD) - FDA Regulatory Timeline and Events
Trappsol Cyclo (AD) is a drug developed by Cyclo Therapeutics for the following indication: Alzheimer's Disease (AD).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Trappsol Cyclo (AD)
- Announced Date:
- May 30, 2024
- Indication:
- Alzheimer's Disease (AD)
Announcement
Cyclo Therapeutics, Inc announced the last patient has been enrolled in the Company's pivotal Phase 3 study ("TransportNPC™") evaluating Trappsol® Cyclo™ for the treatment of systemic and neurological symptoms of Niemann-Pick Disease Type C1 (NPC1).
AI Summary
Cyclo Therapeutics, Inc. announced that the last patient has been enrolled in its pivotal Phase 3 study, TransportNPC™, which is designed to evaluate Trappsol® Cyclo™ for treating both the systemic and neurological symptoms of Niemann-Pick Disease Type C1 (NPC1). This study stands as the most comprehensive controlled trial for NPC1 to date, with a total of 104 patients enrolled globally. In addition to adult patients, the trial includes a substudy evaluating infants and toddlers, as early treatment could potentially prevent or lessen disease progression. The study will provide key data at a 48-week interim analysis in the first half of 2025, which could support future regulatory submissions. With robust support and guidance from health authorities, the enrollment milestone marks a significant step toward bringing a much-needed treatment option to the NPC1 patient community.
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Trappsol® Cyclo™ - FDA Regulatory Timeline and Events
Trappsol® Cyclo™ is a drug developed by Cyclo Therapeutics for the following indication: For the treatment of Niemann-Pick Disease Type C1 (NPC1).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Trappsol® Cyclo™
- Announced Date:
- June 18, 2025
- Indication:
- For the treatment of Niemann-Pick Disease Type C1 (NPC1)
Announcement
Rafael Holdings, Inc. that its subsidiary Cyclo Therapeutics' 96-week pivotal phase 3 TransportNPC study evaluating intravenous (IV) Trappsol® Cyclo™ for the potential treatment of Niemann-Pick Disease Type C1 (NPC1) will continue based on the independent Data Monitoring Committee (DMC) review of safety and efficacy data at the prespecified 48-week interim analysis.
AI Summary
Rafael Holdings, Inc., through its subsidiary Cyclo Therapeutics, announced that its 96‑week pivotal Phase 3 TransportNPC study will continue based on positive findings from an independent Data Monitoring Committee (DMC) review. The interim analysis conducted at 48 weeks showed that the investigational intravenous treatment, Trappsol® Cyclo™, is well-tolerated and has a favorable safety profile. This decision reinforces the potential of Trappsol® Cyclo™ as a promising treatment option for patients with Niemann-Pick Disease Type C1 (NPC1), a rare and progressive genetic disorder. By extending the study to the full 96 weeks, Cyclo Therapeutics is moving forward to evaluate both the systemic and neurological benefits of the therapy. This step underscores the company’s commitment to thorough clinical evaluation and the development of effective treatments that address the underlying cause of NPC1.
Read Announcement- Drug:
- Trappsol® Cyclo™
- Announced Date:
- February 7, 2025
- Estimated Event Date Range:
- January 1, 2025 - June 30, 2025
- Target Action Date:
- H1 2025
- Indication:
- For the treatment of Niemann-Pick Disease Type C1 (NPC1)
Announcement
Cyclo Therapeutics, Inc announced that Company on track for topline data from the 48-week interim analysis of 104 enrolled patients in TransportNPC™ in H1 2025
AI Summary
Cyclo Therapeutics, Inc. announced promising progress in its Pivotal Phase 3 TransportNPC™ study for Niemann-Pick Disease Type C1 (NPC1). The company stated that it is on track to release topline data from the 48-week interim analysis, which includes 104 enrolled patients, in the first half of 2025. This key milestone is expected to provide valuable insights into the safety, tolerability, and efficacy of Trappsol® Cyclo™ for treating this rare genetic condition, which causes harmful cholesterol buildup in vital organs.
The interim analysis focuses on evaluating the impact of Trappsol® Cyclo™ on disease progression by mobilizing cholesterol. These results may pave the way for marketing applications and offer hope for a much-needed therapeutic option for patients and families affected by NPC1.
Read Announcement- Drug:
- Trappsol® Cyclo™
- Announced Date:
- February 7, 2025
- Indication:
- For the treatment of Niemann-Pick Disease Type C1 (NPC1)
Announcement
Cyclo Therapeutics, Inc announced the presentation of data from its ongoing Pivotal Phase 3 global study (TransportNPC™) evaluating Trappsol® Cyclo™ for Niemann-Pick Disease Type C1 ("NPC1") at the 21st Annual WORLDSymposium™ in San Diego, CA.
AI Summary
Cyclo Therapeutics, Inc. recently presented encouraging data at the 21st Annual WORLDSymposium™ in San Diego, CA, from its ongoing Pivotal Phase 3 global study (TransportNPC™). The study is evaluating Trappsol® Cyclo™ as a treatment for Niemann-Pick Disease Type C1 (NPC1), a rare genetic disorder that leads to harmful cholesterol buildup and severe health complications. In a sub-study focusing on patients under age 3, early results showed that 87% of participants at 24 weeks and 86% at 48 weeks exhibited stabilization or improvement as measured by the Clinical Global Impression – Change Scale. These findings support the potential of Trappsol® Cyclo™ to slow disease progression and address significant unmet needs for patients with NPC1, with further topline data expected in the first half of 2025.
Read Announcement- Drug:
- Trappsol® Cyclo™
- Announced Date:
- September 5, 2024
- Indication:
- For the treatment of Niemann-Pick Disease Type C1 (NPC1)
Announcement
Cyclo Therapeutics, Inc announced the presentation of positive preliminary data from its ongoing pivotal Phase 3 study ("TransportNPC™") and substudy evaluating Trappsol® Cyclo™ for the treatment of Niemann-Pick Disease Type C1 (NPC1).
AI Summary
Cyclo Therapeutics, Inc. announced positive preliminary results from its ongoing pivotal Phase 3 study, TransportNPC™, and a related substudy evaluating Trappsol® Cyclo™ for Niemann-Pick Disease Type C1 (NPC1). This major global trial, which enrolled 104 patients, is one of the most comprehensive studies for NPC1 in terms of patient size, duration, and clinical outcomes. The data, presented at the SSIEM Annual Symposium 2024, shows that Trappsol® Cyclo™ is well tolerated with a safety profile matching that of previous stages. In addition, a substudy involving children aged newborn to three years is exploring the benefits of early treatment, which could lead to better management of both visceral and neurological symptoms. An interim analysis at 48 weeks is expected in the first half of 2025, potentially supporting future applications for marketing approval of the treatment.
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