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 D—Orphan-drug Exclusive Approval

21 C.F.R. § 316.31

Scope of orphan-drug exclusive approval

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(a) FDA may approve a sponsor's marketing application for a designated orphan drug for use in the rare disease or condition for which the drug was designated, or for select indication(s) or use(s) within the rare disease or condition for which the drug was designated. Unless FDA previously approved the same drug for the same use or indication, FDA will not approve another sponsor's marketing application for the same drug for the same use or indication before the expiration of 7 years from the date of such approval as stated in the approval letter from FDA, except that such a marketing application can be approved sooner if, and at such time as, any of the following occurs:

(1) Withdrawal of exclusive approval or revocation of orphan-drug designation by FDA under any provision of this part; or

(2) Withdrawal for any reason of the marketing application for the drug in question; or

(3) Consent by the holder of exclusive approval to permit another marketing application to gain approval; or

(4) Failure of the holder of exclusive approval to assure a sufficient quantity of the drug under section 527 of the act and § 316.36.

(b) Orphan-drug exclusive approval protects only the approved indication or use of a designated drug. If such approval is limited to only particular indication(s) or uses(s) within the rare disease or condition for which the drug was designated, FDA may later approve the drug for additional indication(s) or uses(s) within the rare disease or condition not protected by the exclusive approval. If the sponsor who obtains approval for these new indication(s) or uses(s) has orphan-drug designation for the drug for the rare disease or condition, FDA will recognize a new orphan-drug exclusive approval for these new (not previously approved) indication(s) or use(s) from the date of approval of the drug for such new indication(s) or use(s).

(c) If a sponsor's marketing application for a drug product is determined not to be approvable because approval is barred under section 527 of the Federal Food, Drug, and Cosmetic Act until the expiration of the period of exclusive marketing of another drug, FDA will so notify the sponsor in writing.

[57 FR 62085, Dec. 29, 1992, as amended at 78 FR 35134, June 12, 2013]
Notes of Decisions
Cited in 8 cases (4 in the last 5 years), 2014–2026 · 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). “The FDA has interpreted “such drug” to mean “same drug,” 21 C.F.R. § 316.31 (a), and has determined that a drug is the “same” as a previously approved drug if it shares the same “active moiety”—the same active ingredient—and “is intended for the same use,” 21 C.”
Braeburn Inc. v. U.S. Food & Drug Admin., 389 F. Supp. 3d 1 (D.C. Cir. 2019). “21 C.F.R. § 316.31 (a). "Same drug," is defined to exclude any subsequent drug "shown to be clinically superior to the first drug.”
Depomed, Inc. v. United States Dep't of Health & Human Servs., 66 F. Supp. 3d 217 (D.D.C. 2014). “] 21 C.F.R. § 316.31 (a) (2012) (emphasis added).”
Jazz Pharm., Inc. v. Robert F. Kennedy Jr., 141 F.4th 254 (D.C. Cir. 2025). · cites it 2× “” See 21 C.F.R. § 316.31 (a); Orphan Drug Regulations, 57 Fed.”
Jazz Pharm., Inc. v. Becerra (D.D.C. 2024). · cites it 3× “]” 21 C.F.R. § 316.31 (a) 4 (1992) (emphasis added).”
Neurelis Inc. v. Califf (D.D.C. 2025). · cites it 2× “21 C.F.R. § 316.31 (a) (emphasis added). The FDA determined that Valtoco and Libervant were the “same drug,” insofar as they both rely on the same active moiety, 2 diazepam.”
Braeburn Inc. v. United States Food & Drug Admin. (D.D.C. 2019). “21 C.F.R. § 316.31 (a). “Same drug,” is defined to exclude any subsequent drug “shown to be clinically superior to the first drug.”
Ucb, Inc., Ucb Biopharma Srl, Hananja Ehf & Univ. of Iceland v. Cipla Ltd. & Cipla USA Inc. (D. Del. 2026). “The FDA granted Nayzilam orphan drug exclusivity under 21 C.F.R. § 316.31 , which expires on May 17, 2026.”
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