How do I submit an orphan drug designation request?

Mail the CD along with a signed cover letter as described at Corresponding with the OOPD Drug Designation Programs. Alternatively, during this public health emergency associated with the COVID-19 pandemic, orphan designation requests may be submitted by email to [email protected].

When should I request orphan drug designation?

Timeline and Maintenance for Orphan Drug Designation ODD requests are submitted to the FDA’s Office of Orphan Products Development (OOPD) at any time during drug development prior to NDA or BLA submission for the desired orphan drug and condition.

What are the benefits of orphan drug designation?

Companies that receive orphan drug designation are entitled to several advantages, including the possibility of free of charge advice from the FDA and certain financial benefits, such as R&D tax credits (which is not part of the company’s current plans) and exemptions or reductions in regulatory submission fees.

Are orphan drugs exempt from FDA user fees?

Because the market for orphan drugs is often limited, Congress and FDA have traditionally accorded orphan drugs special consideration. For example, sponsors of applications for designated orphan drugs are statutorily exempt from paying the one-time application user fee.

What does orphan FDA mean?

Orphan Drug Act (ODA) as a drug intended to treat a condition affecting fewer than 200,000 persons in the United States, or which will not be profitable within 7 years following approval by the FDA. Incentives

What does FDA orphan drug status mean?

Orphan status is given to drugs and biologics defined as “those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.”.