Monday, November 25, 2019 10:49:41 AM
By nature, rare disease trials have two problems. One, not many patients and two, not enough time. NPC1 takes years to progress. It is different for every kid. Some kids have very bad mutations and die in a few years after birth. Others have less aggressive mutations and progression may take decades. I read an article about a women that was diagnosed with NPC1 at 60 years old.
So...an NPC1 trial only looks at a few patients( MNK and Orphazyme had 50-something patients, CTDH has less than that). The trials are for 52 weeks. In the FDA's opinion, 52 weeks is very small window into the disease progression. The idea behind conditional approval is to approve the drug on 52 week data if it seems positive. However, the trial participates would be monitored for the years to follow. If it appears after 3-5 years the drug actually does not work, approval is withdrawn.
I have heard many people lobby for this approach. My understanding is, as of now, it does not exist. I am not an expert. If you know otherwise please, direct me to that information. That would be very helpful!
I did hear a company argue that if a treatment is a re-purposed drug, with safety data already established, Phase one should be skipped. They argue in favor "conditional approval" with a short trial and few patients, as safety, theoretically, is not an issue.
Recent CYTH News
- Cyclo Therapeutics Presents Encouraging Preliminary Safety Data from Ongoing Pivotal Phase 3 Study and Substudy for the Treatment of Niemann-Pick Disease Type C1 (NPC1) • Business Wire • 09/05/2024 01:05:00 PM
- Kuehn Law Encourages CYTH, ARCH, CEIX, and VGR Investors to Contact Law Firm • PR Newswire (US) • 08/26/2024 01:37:00 PM
- Form 425 - Prospectuses and communications, business combinations • Edgar (US Regulatory) • 08/22/2024 01:37:04 PM
- Form 8-K - Current report • Edgar (US Regulatory) • 08/22/2024 01:36:40 PM
- Rafael Holdings and Cyclo Therapeutics Enter Into a Definitive Merger Agreement • Business Wire • 08/22/2024 10:50:00 AM
- Rafael Holdings and Cyclo Therapeutics Enter into a Definitive Merger Agreement • GlobeNewswire Inc. • 08/22/2024 10:50:00 AM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 08/20/2024 05:50:50 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 08/20/2024 05:49:54 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 08/20/2024 05:49:29 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 08/20/2024 05:49:08 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 08/20/2024 05:48:45 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 08/20/2024 05:48:24 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 08/20/2024 05:47:47 PM
- Cyclo Therapeutics Reports Second Quarter 2024 Financial Results and Provides Business Update • Business Wire • 08/15/2024 01:00:00 PM
- Form 10-Q - Quarterly report [Sections 13 or 15(d)] • Edgar (US Regulatory) • 08/14/2024 09:28:23 PM
- Cyclo Therapeutics Announces Notice of Decision to Grant European Patent Application for Treatment of Alzheimer’s Disease from European Patent Office • Business Wire • 07/29/2024 01:05:00 PM
- Form 8-K - Current report • Edgar (US Regulatory) • 07/17/2024 06:35:02 PM
- Cyclo Therapeutics to Present at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2024 • Business Wire • 06/25/2024 01:25:00 PM
- Rafael Holdings Reports Third Quarter Fiscal 2024 Financial Results • GlobeNewswire Inc. • 06/14/2024 08:01:00 PM
- Cyclo Therapeutics Achieves Landmark Milestone with Completion of Enrollment of Last Patient in Phase 3 Pivotal TransportNPC™ Trial of Niemann-Pick Type C1 • Business Wire • 05/30/2024 01:00:00 PM
- Cyclo Therapeutics Reports First Quarter 2024 Financial Results and Provides Business Update • Business Wire • 05/16/2024 12:05:00 PM
- Cyclo Therapeutics Reports Full Year 2023 Financial Results and Provides Business Update • Business Wire • 03/18/2024 01:05:00 PM
- Cyclo Therapeutics Announces Issuance of U.S. Patent Covering the Use of Trappsol® Cyclo™ for the Treatment of Alzheimer’s Disease • Business Wire • 03/14/2024 01:34:00 PM
- Rafael Holdings Reports Second Quarter Fiscal 2024 Financial Results • GlobeNewswire Inc. • 03/13/2024 09:04:24 PM
- Form 8-K/A - Current report: [Amend] • Edgar (US Regulatory) • 03/04/2024 09:33:34 PM
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