In 1983, the United States Congress passed legislation, known as the Orphan Drug Act, to incentivize companies to develop drugs for diseases with prevalence rates of less than 200,000 patients in the United States. Having such low prevalence rates, these diseases were defined in the Act as “rare diseases” or “orphan diseases”.

Prior to the passing of this Act, there were few, if any, companies developing treatments for rare diseases. Because of such low prevalence rates, companies considered these drugs too costly to produce.

In order to provide the necessary ongoing support to private industry for researching and producing orphan drugs, the FDA, through the Orphan Drug Act, offers:

• A tax credit of 50% off the cost of conducting human trials for orphan drugs
• Research grants for clinical trials of new therapies to treat orphan diseases
• An exclusive marketing monopoly to the sponsoring company for 7 years post-approval
• Waiver of Prescription Drug User Fee Act (PDUFA) filing fees, which are currently about $1,000,000 per application

Since passing the Orphan Drug Act in 1983, the FDA has provided more than 1,000 designations and has approved over 300 new products. It’s important to note that from 1973 to 1983 (before the Orphan Drug Act legislation) only 10 treatments for rare diseases were developed.

So how does the Orphan Drug Act apply to Nutra Pharma? Quite simply, we believe that one of our leading drug candidates, RPI-78M, may be eligible for Orphan Drug Status as a therapy for treating Adrenomyeloneuropathy (AMN). With an estimated 30,000 patients affected by AMN globally, having Orphan Drug Status would provide us with additional financial support during the development stage and then after we gain drug approval.

Additional Resources

• Orphan Drug Act
• Celebrating the Successes of the Orphan Drug Act (Article on FDA.gov)
• List of US Orphan Designations and Approvals
• List of European Orphan Drugs

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