Here is a link about the process of ODD.
http://www.fda.gov/ForIndustry/DevelopingProd...240819.htm
Also, there has been some comments that have been directed to the lack of human clinical data in regards to our first designation. Although human data is a big portion of ODD status, it is not the only means or limitation that is imposed.
he 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.
Although toxicology data alone is not sufficient, other data such as the MANF white paper, and other breakdown that the company has provided in regards to the molecule and its interaction in regards to cell death would be sufficient. Don't be fooled by posters who are saying that ODD is a long shot because of the toxicology data. We have other data that will be sufficient.
http://www.fda.gov/ForIndustry/DevelopingProd...240819.htm
Also, there has been some comments that have been directed to the lack of human clinical data in regards to our first designation. Although human data is a big portion of ODD status, it is not the only means or limitation that is imposed.
he 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.
Although toxicology data alone is not sufficient, other data such as the MANF white paper, and other breakdown that the company has provided in regards to the molecule and its interaction in regards to cell death would be sufficient. Don't be fooled by posters who are saying that ODD is a long shot because of the toxicology data. We have other data that will be sufficient.
