A sponsor submitting a request for orphan-drug designation for a drug intended to treat a specific rare disease or condition must ensure that the request is formatted and contains the information specified in paragraph (b) of this section. Sponsors may request orphan-drug designation for a drug that has not yet been approved, or for a new use of a drug that is already on the market. Additionally, a sponsor may seek orphan-drug designation for a drug that is otherwise identical to an already approved drug, provided they can present a credible hypothesis that their drug may offer clinical superiority over the existing one. Multiple sponsors can receive orphan-drug designation for the same drug and the same rare disease or condition, but each sponsor must submit a complete designation request as outlined in paragraph (b) of this section.

Read the original FDA guidance on Special Protocol Assessment requests.