C.F.R. » Title 21 » CHAPTER I—FOOD AND DRUG ADMINISTRATION, DEPARTMENT OF HEALTH AND HUMAN SERVICES › SUBCHAPTER D—DRUGS FOR HUMAN USE › PART 316—ORPHAN DRUGS › Subpart C—Designation of an Orphan Drug

21 C.F.R. § 316.29

Revocation of orphan-drug designation

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(a) FDA may revoke orphan-drug designation for any drug if the agency finds that:

(1) The request for designation contained an untrue statement of material fact; or

(2) The request for designation omitted material information required by this part; or

(3) FDA subsequently finds that the drug in fact had not been eligible for orphan-drug designation at the time of submission of the request therefor.

(b) For an approved drug, revocation of orphan-drug designation also suspends or withdraws the sponsor's exclusive marketing rights for the drug but not the approval of the drug's marketing application.

(c) Where a drug has been designated as an orphan drug because the prevalence of a disease or condition (or, in the case of vaccines, diagnostic drugs, or preventive drugs, the target population) is under 200,000 in the United States at the time of designation, its designation will not be revoked on the ground that the prevalence of the disease or condition (or the target population) becomes more than 200,000 persons.

(d) If FDA revokes orphan-drug designation, FDA will publicize that the drug is no longer designated in accordance with § 316.28(e).

[57 FR 62085, Dec. 29, 1992, as amended at 78 FR 35134, June 12, 2013]
Notes of Decisions
Cited in 2 cases (1 in the last 5 years), 2020–2025 · leading case: Eagle Pharm., Inc. v. Alex Azar, II, 952 F.3d 323 (D.C. Cir. 2020).
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Eagle Pharm., Inc. v. Alex Azar, II, 952 F.3d 323 (D.C. Cir. 2020). “See 21 C.F.R. § 316.29 . The FDA’s ability to revoke designation or approval (and thus exclusivity) because of fraud or mistake does not run afoul of the language of § 360cc(a) in the same way that including an additional post-approval hurdle a manufacturer must clear before…”
Jazz Pharm., Inc. v. Robert F. Kennedy Jr., 141 F.4th 254 (D.C. Cir. 2025). “21 C.F.R. § 316.29 (c). 4 Xyrem had exclusivity periods from 2002 to 2009 to treat cataplexy associated with narcolepsy, from 2005 to 2012 to treat 8 In July 2020, the FDA approved Jazz’s Xywav, an oxybate- based drug that the FDA determined was clinically superior to Xyrem…”
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