Thanks, NC! Good info. I wasn't exactly clear on what was necessary to qualify for the orphan drug designation, so I looked up on the
FDA site and found this:
"
The regulations say that the sponsor is required to submit all relevant data about their drug, why doesn’t the sponsor have to submit animal toxicology data for orphan designation?
- In order to designate a product as an orphan drug, the scientific rationale portion of the designation application must include enough information to establish a medically plausible basis for expecting the drug to be effective in the rare disease. This is best supported by clinical trials of the drug in the rare disease or condition
- However, in absence of human data, the application for orphan drug designation may be satisfactorily supported with compelling preclinical data that uses the active moiety or principal molecular structure of the proposed orphan drug in a relevant animal model for the rare human disease. Animal toxicology data, which describes the safety of the drug in animals, does not provide efficacy data, so is not useful in supporting the scientific rationale section of the orphan drug designation.
"
So I guess the FDA is concerned only with plausible efficacy of the drug, not the toxicology. I suppose this may be why drugs which are designated as potential orphan drugs at a preclinical stage have a much higher chance of success; i.e., because they have already been shown to be effective at producing a desired effect. However, since the toxicology has not been reviewed yet, that may still be a relative unknown. We should find out the toxicology in the first phase of clinical trials, so we should know sooner rather than later. Of course, efficacy in a mouse does not automatically imply efficacy in a human, but I think things are looking promising because of their more stringent animal models.
Also, regarding the Fast Track process, I found this (
link):
"
Fast track designation must be requested by the drug company. The FDA reviews the request and will generally make a decision within 60 days based on whether the drug fills an unmet medical need treating a serious disease. The request can be initiated at any time during the drug development process, but typically, the fast track designation will occur during Phase 2 or 3 of the drug's clinical trials.
The FDA receives approximately 100-130 applications a year, and has stated that close to 80 percent of all filed applications will eventually be approved.
"
So maybe we can expect this to happen sometime after Phase I results are known.
