PharmaCyte Biotech Inc (PMCB)

[HighHoeSilverado]

If u werent so hemi chevy might sho u sumthin.


If a product with an Active Pharmaceutical Ingredient (API) is already approved for a rare disease indication, but a different formulation is believed to have a greater efficacy for the same rare disease indication, can an orphan designation be requested? The public policy objective of the Orphan Drug Act is to further the testing and marketing of products for rare diseases in which no current therapy exists or where the product will significantly improve the existing therapy. If a product has received marketing approval in the United States for use in the proposed orphan indication, the only way the proposed product can be designated as an orphan drug is if the sponsor provides a reasonable hypothesis that their product is “clinically superior” to the approved product by means of greater effectiveness, greater safety, or that it provides a major contribution to patient care (MC–to-PC).  Further, if orphan drug designation is ultimately granted, in order for the product to receive orphan drug exclusive approval for this indication, the sponsor must actually demonstrate the superiority claim by showing their product is significantly more effective than the approved product in a clinical trial, is safer in a substantial portion of the target population, or can provide a major contribution to patient care.  Any claim for clinical superiority could require a head-to–head trial.