U.S. Code » Title 21 » Chapter CHAPTER 9— FEDERAL FOOD, DRUG, AND COSMETIC ACT › Subchapter SUBCHAPTER V— DRUGS AND DEVICES › Part Part B— Drugs for Rare Diseases or Conditions

21 U.S.C. § 360bb

Designation of drugs for rare diseases or conditions

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(a) Request by sponsor; preconditions; “rare disease or condition” defined(1) The manufacturer or the sponsor of a drug may request the Secretary to designate the drug as a drug for a rare disease or condition. A request for designation of a drug shall be made before the submission of an application under section 355(b) of this title for the drug, or the submission of an application for licensing of the drug under section 262 of title 42. If the Secretary finds that a drug for which a request is submitted under this subsection is being or will be investigated for a rare disease or condition and—(A) if an application for such drug is approved under section 355 of this title, or(B) if a license for such drug is issued under section 262 of title 42,the approval, certification, or license would be for use for such disease or condition, the Secretary shall designate the drug as a drug for such disease or condition. A request for a designation of a drug under this subsection shall contain the consent of the applicant to notice being given by the Secretary under subsection (b) 11 See References in Text note below. respecting the designation of the drug.(2) For purposes of paragraph (1), the term “rare disease or condition” means any disease or condition which (A) affects less than 200,000 persons in the United States, or (B) affects more than 200,000 in the United States and for which there is no reasonable expectation that the cost of developing and making available in the United States a drug for such disease or condition will be recovered from sales in the United States of such drug. Determinations under the preceding sentence with respect to any drug shall be made on the basis of the facts and circumstances as of the date the request for designation of the drug under this subsection is made.(b) Notification of discontinuance of drug or application as conditionA designation of a drug under subsection (a) shall be subject to the condition that—(1) if an application was approved for the drug under section 355(b) of this title or a license was issued for the drug under section 262 of title 42, the manufacturer of the drug will notify the Secretary of any discontinuance of the production of the drug at least one year before discontinuance, and(2) if an application has not been approved for the drug under section 355(b) of this title or a license has not been issued for the drug under section 262 of title 42 and if preclinical investigations or investigations under section 355(i) of this title are being conducted with the drug, the manufacturer or sponsor of the drug will notify the Secretary of any decision to discontinue active pursuit of approval of an application under section 355(b) of this title or approval of a license under section 262 of title 42.(c) Notice to public

Notice respecting the designation of a drug under subsection (a) shall be made available to the public.

(d) Regulations

The Secretary shall by regulation promulgate procedures for the implementation of subsection (a).

(June 25, 1938, ch. 675, § 526, as added Pub. L. 97–414, § 2(a), Jan. 4, 1983, 96 Stat. 2050; amended Pub. L. 98–551, § 4(a), Oct. 30, 1984, 98 Stat. 2817; Pub. L. 99–91, § 3(a)(2), Aug. 15, 1985, 99 Stat. 387; Pub. L. 100–290, § 2, Apr. 18, 1988, 102 Stat. 90; Pub. L. 105–115, title I, § 125(b)(2)(H), (I), Nov. 21, 1997, 111 Stat. 2326.)Editorial NotesReferences in Text

Subsection (b), referred to in subsec. (a)(1), was redesignated as subsec. (c) of this section by Pub. L. 100–290, § 2(b), Apr. 18, 1988, 102 Stat. 90.

Amendments

1997—Subsec. (a)(1). Pub. L. 105–115, § 125(b)(2)(H), struck out “the submission of an application for certification of the drug under section 357 of this title,” before “or the submission of an application for licensing of the drug” in introductory provisions, inserted “or” at end of subpar. (A), redesignated subpar. (C) as (B), and struck out former subpar. (B) which read as follows: “if a certification for such drug is issued under section 357 of this title, or”.

Subsec. (b)(1). Pub. L. 105–115, § 125(b)(2)(I)(i), struck out “, a certificate was issued for the drug under section 357 of this title,” before “or a license was issued”.

Subsec. (b)(2). Pub. L. 105–115, § 125(b)(2)(I)(ii), struck out “, a certificate has not been issued for the drug under section 357 of this title,” before “or a license has not been issued” and “, approval of an application for certification under section 357 of this title,” before “or approval of a license”.

1988—Subsec. (a)(1). Pub. L. 100–290, § 2(a), inserted after first sentence “A request for designation of a drug shall be made before the submission of an application under section 355(b) of this title for the drug, the submission of an application for certification of the drug under section 357 of this title, or the submission of an application for licensing of the drug under section 262 of title 42.”

Subsecs. (b) to (d). Pub. L. 100–290, § 2(b), added subsec. (b) and redesignated former subsecs. (b) and (c) as (c) and (d), respectively.

1985—Subsec. (a)(1). Pub. L. 99–91 struck out “or” at end of subpar. (A), struck out subpar. (B) and substituted subpars. (B) and (C), and inserted “, certification,” after “approval”.

1984—Subsec. (a)(2). Pub. L. 98–551 substituted “which (A) affects less than 200,000 persons in the United States, or (B) affects more than 200,000 in the United States and for which” for “which occurs so infrequently in the United States that”.

Statutory Notes and Related SubsidiariesEffective Date of 1985 Amendment

Amendment by Pub. L. 99–91 effective Aug. 15, 1985, see section 8(b) of Pub. L. 99–91, set out as a note under section 360aa of this title.

Notes of Decisions
Cited in 19 cases (4 in the last 5 years), 1987–2025 · 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). · cites it 7× “21 U.S.C. § 360bb. 2 Designation as an “orphan drug” provides benefits designed to promote orphan drug development such as tax credits, assistance with investigations and the approval process and monetary grants to defray the costs of developing orphan drugs.”
Pharm. Rsch. & Mfrs. of Am. v. United States Dep't of Health & Human Servs., 43 F. Supp. 3d 28 (D.D.C. 2014). · cites it 4× “§ 216 , as amended, (3) Section 526 of the FFDCA, 21 U.S.C. § 360bb, as amended, (4) Section 701(a) of the FFDCA, 21 U.”
Depomed, Inc. v. United States Dep't of Health & Human Servs., 66 F. Supp. 3d 217 (D.D.C. 2014). · cites it 6× “In other words, the plain language of the statute sets forth two procedural prerequisites for marketing exclusivity: first, the FDA must have “designated” the drug as an orphan drug, upon request- from the drug’s sponsor, pursuant to 21 U.S.C. § 360bb and its accompanying…”
Pharm. Rsch. & Mfrs. of Am. v. United States Dep't of Health & Human Servs., 138 F. Supp. 3d 31 (D.C. Cir. 2015). · cites it 3× “designated under 21 U.S.C. § 360bb. Compare id. (applying to a “drug or biological .”
Genentech, Inc. v. Bowen, 676 F. Supp. 301 (D.D.C. 1987). · cites it 6× “21 U.S.C. § 360bb. Orphan drug designation enables the manufacturer or sponsor to take advantage of the Act’s tax benefits, to request pre-ap *304 proval clinical testing recommendations, and to request financial assistance from the FDA in conducting the necessary clinical…”
Catalyst Pharm., Inc. v. Xavier Becerra, 14 F.4th 1299 (11th Cir. 2021). “Designation Pursuant to 21 U.S.C. § 360bb(a)(1), a drug manufacturer may request the FDA to designate a drug as an orphan drug—one that “is being or will be investigated for a rare disease or condition.”
Mut. Pharm. Co. v. Iyax Pharm., Inc., 459 F. Supp. 2d 925 (C.D. Cal. 2006). “See 21 U.S.C. § 360bb(a)(2)(de-fining a "rare disease or condition” as one "affect[ing] less than 200,000 persons in the United States”); see also David Duffield Roh-de, The Orphan Drug Act: An Engine of Innovation? At What Cost?, 55 Food & Drug L.”
Baker Norton Pharm., Inc. v. United States Food & Drug Admin., 132 F. Supp. 2d 30 (D.D.C. 2001). · cites it 2× “21 U.S.C. § 360bb(a)(2). Because the drug is designated as an orphan drug before it is approved, more than one applicant may receive orphan designation for what later may be deemed the same “drug” for treatment of the same disease or condition.”
Braeburn Inc. v. U.S. Food & Drug Admin., 389 F. Supp. 3d 1 (D.C. Cir. 2019). “" 21 U.S.C. § 360bb(a)(1). If the FDA finds that the drug under development will treat a rare disease or condition, the FDA shall designate the drug accordingly.”
In Re Transkaryotic Therapies, Inc. Sec. Litig., 319 F. Supp. 2d 152 (D. Mass. 2004). “The Food and Drug Administration (“FDA”) designated each product as “a drug for a rare disease or condition” pursuant to 21 U.S.C. § 360bb, meaning that if one received FDA approval for marketing the drug in the United States, the agency could not approve the rival product for…”
Bristol-Myers Squibb Co. v. Ivax Corp., 77 F. Supp. 2d 606 (D.N.J. 2000). “Orphan drug designation is a form of non-patent marketing exclusivity granted by the FDA pursuant to 21 U.S.C. § 360bb to encourage the development of drugs to treat rare diseases or conditions.”
Jazz Pharm., Inc. v. Robert F. Kennedy Jr., 141 F.4th 254 (D.C. Cir. 2025). · cites it 2× “2 21 U.S.C. § 360bb(a)(1). That designation provides benefits such as assistance with investigations and the approval process, monetary grants to defray drug development costs and tax credits.”
Show all 19 citing cases →
— 21 U.S.C. § 360bb(2) — 1 case
Berlex Labs., Inc. v. Food & Drug Admin., 942 F. Supp. 19 (D.D.C. 1996).
— 21 U.S.C. § 360bb(a) — 2 cases
Pharm. Rsch. & Mfrs. of Am. v. United States Dep't of Health & Human Servs., 43 F. Supp. 3d 28 (D.D.C. 2014). “§ 216 , as amended, (3) Section 526 of the FFDCA, 21 U.S.C. § 360bb, as amended, (4) Section 701(a) of the FFDCA, 21 U.”
Genentech, Inc. v. Bowen, 676 F. Supp. 301 (D.D.C. 1987). “21 U.S.C. § 360bb. Orphan drug designation enables the manufacturer or sponsor to take advantage of the Act’s tax benefits, to request pre-ap *304 proval clinical testing recommendations, and to request financial assistance from the FDA in conducting the necessary clinical…”
— 21 U.S.C. § 360bb(a)(1) — 8 cases
Eagle Pharm., Inc. v. Alex Azar, II, 952 F.3d 323 (D.C. Cir. 2020). “21 U.S.C. § 360bb. 2 Designation as an “orphan drug” provides benefits designed to promote orphan drug development such as tax credits, assistance with investigations and the approval process and monetary grants to defray the costs of developing orphan drugs.”
Catalyst Pharm., Inc. v. Xavier Becerra, 14 F.4th 1299 (11th Cir. 2021). “Designation Pursuant to 21 U.S.C. § 360bb(a)(1), a drug manufacturer may request the FDA to designate a drug as an orphan drug—one that “is being or will be investigated for a rare disease or condition.”
Braeburn Inc. v. U.S. Food & Drug Admin., 389 F. Supp. 3d 1 (D.C. Cir. 2019). “" 21 U.S.C. § 360bb(a)(1). If the FDA finds that the drug under development will treat a rare disease or condition, the FDA shall designate the drug accordingly.”
Jazz Pharm., Inc. v. Robert F. Kennedy Jr., 141 F.4th 254 (D.C. Cir. 2025). “2 21 U.S.C. § 360bb(a)(1). That designation provides benefits such as assistance with investigations and the approval process, monetary grants to defray drug development costs and tax credits.”
Braeburn Inc. v. United States Food & Drug Admin. (D.D.C. 2019).
— 21 U.S.C. § 360bb(a)(2) — 9 cases
Eagle Pharm., Inc. v. Alex Azar, II, 952 F.3d 323 (D.C. Cir. 2020). “21 U.S.C. § 360bb. 2 Designation as an “orphan drug” provides benefits designed to promote orphan drug development such as tax credits, assistance with investigations and the approval process and monetary grants to defray the costs of developing orphan drugs.”
Pharm. Rsch. & Mfrs. of Am. v. United States Dep't of Health & Human Servs., 43 F. Supp. 3d 28 (D.D.C. 2014). “§ 216 , as amended, (3) Section 526 of the FFDCA, 21 U.S.C. § 360bb, as amended, (4) Section 701(a) of the FFDCA, 21 U.”
Depomed, Inc. v. United States Dep't of Health & Human Servs., 66 F. Supp. 3d 217 (D.D.C. 2014). “In other words, the plain language of the statute sets forth two procedural prerequisites for marketing exclusivity: first, the FDA must have “designated” the drug as an orphan drug, upon request- from the drug’s sponsor, pursuant to 21 U.S.C. § 360bb and its accompanying…”
Mut. Pharm. Co. v. Iyax Pharm., Inc., 459 F. Supp. 2d 925 (C.D. Cal. 2006). “See 21 U.S.C. § 360bb(a)(2)(de-fining a "rare disease or condition” as one "affect[ing] less than 200,000 persons in the United States”); see also David Duffield Roh-de, The Orphan Drug Act: An Engine of Innovation? At What Cost?, 55 Food & Drug L.”
Pharm. Rsch. & Mfrs. of Am. v. United States Dep't of Health & Human Servs., 138 F. Supp. 3d 31 (D.C. Cir. 2015). “designated under 21 U.S.C. § 360bb. Compare id. (applying to a “drug or biological .”
— 21 U.S.C. § 360bb(a)(l) — 1 case
Pharm. Rsch. & Mfrs. of Am. v. United States Dep't of Health & Human Servs., 138 F. Supp. 3d 31 (D.C. Cir. 2015). “designated under 21 U.S.C. § 360bb. Compare id. (applying to a “drug or biological .”
— 21 U.S.C. § 360bb(a)(l)(C) — 1 case
Baker Norton Pharm., Inc. v. United States Food & Drug Admin., 132 F. Supp. 2d 30 (D.D.C. 2001). “21 U.S.C. § 360bb(a)(2). Because the drug is designated as an orphan drug before it is approved, more than one applicant may receive orphan designation for what later may be deemed the same “drug” for treatment of the same disease or condition.”
— 21 U.S.C. § 360bb(b) — 1 case
Genentech, Inc. v. Bowen, 676 F. Supp. 301 (D.D.C. 1987). “21 U.S.C. § 360bb. Orphan drug designation enables the manufacturer or sponsor to take advantage of the Act’s tax benefits, to request pre-ap *304 proval clinical testing recommendations, and to request financial assistance from the FDA in conducting the necessary clinical…”
— 21 U.S.C. § 360bb(d) — 3 cases
Eagle Pharm., Inc. v. Alex Azar, II, 952 F.3d 323 (D.C. Cir. 2020). “21 U.S.C. § 360bb. 2 Designation as an “orphan drug” provides benefits designed to promote orphan drug development such as tax credits, assistance with investigations and the approval process and monetary grants to defray the costs of developing orphan drugs.”
Pharm. Rsch. & Mfrs. of Am. v. United States Dep't of Health & Human Servs., 43 F. Supp. 3d 28 (D.D.C. 2014). “§ 216 , as amended, (3) Section 526 of the FFDCA, 21 U.S.C. § 360bb, as amended, (4) Section 701(a) of the FFDCA, 21 U.”
Depomed, Inc. v. United States Dep't of Health & Human Servs., 66 F. Supp. 3d 217 (D.D.C. 2014). “In other words, the plain language of the statute sets forth two procedural prerequisites for marketing exclusivity: first, the FDA must have “designated” the drug as an orphan drug, upon request- from the drug’s sponsor, pursuant to 21 U.S.C. § 360bb and its accompanying…”
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