21 U.S.C. § 360cc

Protection for drugs for rare diseases or conditions

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(a) Exclusive approval, certification, or licenseExcept as provided in subsection (b), if the Secretary—(1) approves an application filed pursuant to section 355 of this title, or(2) issues a license under section 262 of title 42for a drug designated under section 360bb of this title for a rare disease or condition, the Secretary may not approve another application under section 355 of this title or issue another license under section 262 of title 42 for the same drug for the same approved use or indication within such rare disease or condition for a person who is not the holder of such approved application or of such license until the expiration of seven years from the date of the approval of the approved application or the issuance of the license. Section 355(c)(2) 11 See References in Text note below. of this title does not apply to the refusal to approve an application under the preceding sentence.(b) ExceptionsDuring the 7-year period described in subsection (a) for an approved application under section 355 of this title or license under section 262 of title 42, the Secretary may approve an application or issue a license for a drug that is otherwise the same, as determined by the Secretary, as the already approved drug for the same approved use or indication for which such 7-year period applies to such already approved or licensed drug if—(1) the Secretary finds, after providing the holder of exclusive approval or licensure notice and opportunity for the submission of views, that during such period the holder of the exclusive approval or licensure cannot ensure the availability of sufficient quantities of the drug to meet the needs, relating to the approved use or indication, of persons with the disease or condition for which the drug was designated; or(2) the holder provides the Secretary in writing the consent of such holder for the approval of other applications or the issuance of other licenses before the expiration of such seven-year period.(c) Condition of clinical superiority(1) In general

If a sponsor of a drug that is designated under section 360bb of this title and is otherwise the same, as determined by the Secretary, as an already approved or licensed drug is seeking exclusive approval or exclusive licensure described in subsection (a) for the same use or indication for which the already approved or licensed drug was approved or licensed, the Secretary shall require such sponsor, as a condition of such exclusive approval or licensure, to demonstrate that such drug is clinically superior to any already approved or licensed drug that is the same drug.

(2) Definition

For purposes of paragraph (1), the term “clinically superior” with respect to a drug means that the drug provides a significant therapeutic advantage over and above an already approved or licensed drug in terms of greater efficacy, greater safety, or by providing a major contribution to patient care.

(3) Applicability

This subsection applies to any drug designated under section 360bb of this title for which an application was approved under section 355 of this title or licensed under section 262 of title 42 after August 18, 2017, regardless of the date on which such drug was designated under section 360bb of this title.

(d) Regulations

The Secretary may promulgate regulations for the implementation of subsection (c). Beginning on August 18, 2017, until such time as the Secretary promulgates regulations in accordance with this subsection, the Secretary may apply any definitions set forth in regulations that were promulgated prior to such date, to the extent such definitions are not inconsistent with the terms of this section, as amended by such Act.

(e) Demonstration of clinical superiority standardTo assist sponsors in demonstrating clinical superiority as described in subsection (c), the Secretary—(1) upon the designation of any drug under section 360bb of this title, shall notify the sponsor of such drug in writing of the basis for the designation, including, as applicable, any plausible hypothesis offered by the sponsor and relied upon by the Secretary that the drug is clinically superior to a previously approved drug; and(2) upon granting exclusive approval or licensure under subsection (a) on the basis of a demonstration of clinical superiority as described in subsection (c), shall publish a summary of the clinical superiority findings.(f) Approved use or indication defined

In this section, the term “approved use or indication” means the use or indication approved under section 355 of this title or licensed under section 262 of title 42 for a drug designated under section 360bb of this title for a rare disease or condition.

(June 25, 1938, ch. 675, § 527, as added Pub. L. 97–414, § 2(a), Jan. 4, 1983, 96 Stat. 2050; amended Pub. L. 98–417, title I, § 102(b)(6), Sept. 24, 1984, 98 Stat. 1593; Pub. L. 99–91, §§ 2, 3(a)(3), Aug. 15, 1985, 99 Stat. 387, 388; Pub. L. 103–80, § 3(v), Aug. 13, 1993, 107 Stat. 778; Pub. L. 105–115, title I, § 125(b)(2)(J), (K), Nov. 21, 1997, 111 Stat. 2326; Pub. L. 107–281, § 4, Nov. 6, 2002, 116 Stat. 1993; Pub. L. 115–52, title VI, § 607(a), Aug. 18, 2017, 131 Stat. 1049; Pub. L. 116–260, div. BB, title III, § 323, Dec. 27, 2020, 134 Stat. 2933; Pub. L. 119–75, div. J, title VI, § 6605(a), Feb. 3, 2026, 140 Stat. 701.)Editorial NotesReferences in Text

Section 355(c)(2) of this title, referred to in subsec. (a), was redesignated as section 355(c)(1)(B) of this title by Pub. L. 98–417, title I, § 102(a)(2), Sept. 24, 1984, 98 Stat. 1592.

This section, as amended by such Act, referred to in subsec. (d), means this section as amended by the FDA Reauthorization Act of 2017, Pub. L. 115–52.

Amendments

2026—Subsec. (a). Pub. L. 119–75, § 6605(a)(1), substituted “same approved use or indication within such rare disease or condition” for “same disease or condition” in concluding provisions.

Subsec. (b). Pub. L. 119–75, § 6605(a)(2)(A), substituted “same approved use or indication for which such 7-year period applies to such already approved or licensed drug” for “same rare disease or condition” in introductory provisions.

Subsec. (b)(1). Pub. L. 119–75, § 6605(a)(2)(B), inserted “, relating to the approved use or indication,” after “the needs”.

Subsec. (c)(1). Pub. L. 119–75, § 6605(a)(3), substituted “same use or indication for which the already approved or licensed drug was approved or licensed” for “same rare disease or condition as the already approved drug”.

Subsec. (f). Pub. L. 119–75, § 6605(a)(4), added subsec. (f).

2020—Subsec. (c)(3). Pub. L. 116–260 added par. (3).

2017—Subsec. (a). Pub. L. 115–52, § 607(a)(1), substituted “the same drug for the same disease or condition” for “such drug for such disease or condition” in concluding provisions.

Subsec. (b). Pub. L. 115–52, § 607(a)(2)(A), in introductory provisions, substituted “During the 7-year period described in subsection (a) for an approved application under section 355 of this title or license under section 262 of title 42, the Secretary may approve an application or issue a license for a drug that is otherwise the same, as determined by the Secretary, as the already approved drug for the same rare disease or condition if” for “If an application filed pursuant to section 355 of this title is approved for a drug designated under section 360bb of this title for a rare disease or condition or if a license is issued under section 262 of title 42 for such a drug, the Secretary may, during the seven-year period beginning on the date of the application approval or of the issuance of the license, approve another application under section 355 of this title or issue a license under section 262 of title 42, for such drug for such disease or condition for a person who is not the holder of such approved application or of such license if”.

Subsec. (b)(1). Pub. L. 115–52, § 607(a)(2)(B), substituted “of exclusive approval or licensure notice and opportunity for the submission of views, that during such period the holder of the exclusive approval or licensure cannot ensure” for “notice and opportunity for the submission of views, that in such period the holder of the approved application or of the license cannot assure”.

Subsec. (b)(2). Pub. L. 115–52, § 607(a)(2)(C), substituted “the holder provides” for “such holder provides”.

Subsecs. (c) to (e). Pub. L. 115–52, § 607(a)(3), added subsecs. (c) to (e).

2002—Subsec. (a). Pub. L. 107–281, in concluding provisions, struck out “, of such certification,” after “such approved application” and “, the issuance of the certification,” after “approval of the approved application”.

1997—Subsec. (a). Pub. L. 105–115, § 125(b)(2)(J), struck out “, issue another certification under section 357 of this title,” before “or issue another license” in closing provisions, inserted “or” at end of par. (1), redesignated par. (3) as (2), and struck out former par. (2) which read as follows: “issues a certification under section 357 of this title, or”.

Subsec. (b). Pub. L. 105–115, § 125(b)(2)(K), in introductory provisions, struck out “, if a certification is issued under section 357 of this title for such a drug,” after “rare disease or condition”, “, of the issuance of the certification under section 357 of this title,” after “application approval”, “, issue another certification under section 357 of this title,” after “application under section 355 of this title”, and “, of such certification,” after “approved application”.

Subsec. (b)(1). Pub. L. 105–115, § 125(b)(2)(K), struck out “, of the certification,” after “holder of the approved application”.

Subsec. (b)(2). Pub. L. 105–115, § 125(b)(2)(K), struck out “, issuance of other certifications,” after “approval of other applications”.

1993—Subsec. (b). Pub. L. 103–80 struck out extraneous comma before “or issue a license under section 262” in introductory provisions and substituted “the” for “The” at beginning of par. (1).

1985—Pub. L. 99–91, § 2(3), struck out “unpatented” before “drugs” in section catchline.

Subsec. (a). Pub. L. 99–91, §§ 2(1), 3(a)(3)(A)–(D), struck out “or” at end of par. (1), added par. (2), redesignated former par. (2) as (3), struck out “and for which a United States Letter of Patent may not be issued” after “rare disease or condition”, inserted in first sentence “, issue another certification under section 357 of this title,” after “section 355 of this title” the second time it appeared, inserted “, of such certification,” after “holder of such approved application”, and inserted “, the issuance of the certification,” after “approval of the approved application”.

Subsec. (b). Pub. L. 99–91, §§ 2(2), 3(a)(3)(E)–(K), struck out “and if a United States Letter of Patent may not be issued for the drug” after “such a drug”, substituted “, if a certification is issued under section 357 of this title for such a drug, or if a license” for “or a license”, inserted “, of the issuance of the certification under section 357 of this title,” after “application approval”, struck out “, if the drug is a biological product,” before “issue a license”, inserted “, issue another certification under section 357 of this title,” after “section 355 of this title”, inserted “, of such certification,” after “holder of such approved application”, inserted “, of such certification,” after “application” in par. (1), and inserted “, issuance of other certifications,” after “other applications” in par. (2).

1984—Subsecs. (a), (b). Pub. L. 98–417 substituted “section 355” for “section 355(b)” wherever appearing.

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.

Construction

Pub. L. 115–52, title VI, § 607(b), Aug. 18, 2017, 131 Stat. 1050, provided that: “Nothing in the amendments made by subsection (a) [amending this section] shall affect any determination under sections 526 and 527 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bb, 360cc) made prior to the date of enactment of the FDA Reauthorization Act of 2017 [Aug. 18, 2017].”

Application of Amendments by Pub. L. 119–75

Pub. L. 119–75, div. J, title VI, § 6605(b), Feb. 3, 2026, 140 Stat. 701, provided that: “The amendments made by subsection (a) [amending this section] shall apply with respect to any drug designated under section 526 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bb), regardless of the date on which the drug was so designated, and regardless of the date on which the drug was approved under section 505 of such Act (21 U.S.C. 355) or licensed under section 351 of the Public Health Service Act (42 U.S.C. 262).”

Notes of Decisions

Cited in 22 cases (6 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 15× “9 In 2017, while this case was in district court, the Congress amended the ODA to codify a clinical superiority requirement for exclusivity and supersede Depomed’s holding.”

In Re Modafinil Antitrust Litig., 837 F.3d 238 (3rd Cir. 2016). · cites it 2× “See 21 U.S.C. § 360cc(a). 14 District of New Jersey on March 28, 2003.”

Depomed, Inc. v. United States Dep't of Health & Human Servs., 66 F. Supp. 3d 217 (D.D.C. 2014). · cites it 8× “Elsewhere, the regulations define the term “same drug” — a term that the regulations use in place of the statutory term “such drug” that appears in 21 U.S.C. § 360cc — to mean, in relevant part, “a drug that contains the same active moiety as a previously approved drug and is…”

Spectrum Pharm., Inc v. Sylvia Burwell, 824 F.3d 1062 (D.C. Cir. 2016). · cites it 4× “21 U.S.C. § 360cc. An application will necessarily include only stated indications, not intended off-label uses.”

Catalyst Pharm., Inc. v. Xavier Becerra, 14 F.4th 1299 (11th Cir. 2021). · cites it 3× “LAGOA, Circuit Judge: This appeal asks us to determine whether the statutory phrase “same disease or condition” contained in the Orphan Drug Act, see 21 U.S.C. § 360cc, is ambiguous. It is not.”

Braeburn Inc. v. U.S. Food & Drug Admin., 389 F. Supp. 3d 1 (D.C. Cir. 2019). · cites it 4× “Orphan-designated drugs receive 7-year exclusivity periods under the conditions set forth in 21 U.S.C. § 360cc. Before Congress passed FDARA in 2017, § 360cc provided, with exceptions not relevant here, that if the FDA: [A]pproves an application filed pursuant to section 355 of…”

Jazz Pharm., Inc. v. Robert F. Kennedy Jr., 141 F.4th 254 (D.C. Cir. 2025). · cites it 13× “First, in subsection 360cc(a), the Congress replaced “such drug for such disease or condition” with “the same drug for the same disease or condition,” so the FDA “may not approve another application . . . for the same drug for the same disease or condition” for seven years from…”

Genentech, Inc. v. Bowen, 676 F. Supp. 301 (D.D.C. 1987). · cites it 8× “On December 12, 1985, the FDA designated Protropin as an orphan drug, thus granting Genentech marketing exclusivity, pursuant to 21 U.S.C. § 360cc, until December 12, 1992.”

Baker Norton Pharm., Inc. v. United States Food & Drug Admin., 132 F. Supp. 2d 30 (D.D.C. 2001). · cites it 3× “Once the drug is designated an orphan drug, it goes through the approval process for orphan drug exclusivity under 21 U.S.C. § 360cc. B. Baker Norton’s and BMS’s FDA applications Baker Norton and BMS are pharmaceutical manufacturers.”

Mut. Pharm. Co. v. Iyax Pharm., Inc., 459 F. Supp. 2d 925 (C.D. Cal. 2006). “See 21 U.S.C. § 360cc(a). As the House Report submitted in connection with the passage of the Orphan Drug Act explained, the purpose of this seven-year market exclusivity period was to allow the orphan drug's sponsor "to recoup the cost of development by capturing all revenues…”

Pharm. Rsch. & Mfrs. of Am. v. United States Dep't of Health & Human Servs., 138 F. Supp. 3d 31 (D.C. Cir. 2015). “For that reason, the Orphan Drug Act provides several incentives to those pharmaceutical manufacturers that develop orphan drugs, including: a seven-year market exclusivity period during which no drugs, other than the designated orphan drug, can be licensed or approved “for such…”

K-V Pharm. Co. v. United States Food & Drug Admin., 889 F. Supp. 2d 119 (D.D.C. 2012). · cites it 4× “§ 558 (c); section 360cc(a) of the FDCA, 21 U.S.C. § 360cc; and the Due Process Clause of the Fifth Amendment of the United States Constitution, and acted arbitrarily, capriciously, in an abuse of discretion, and in excess of its authority, in *128 violation of the APA, 5 U.”

Show all 22 citing cases →

— 21 U.S.C. § 360cc(a) — 18 cases

Eagle Pharm., Inc. v. Alex Azar, II, 952 F.3d 323 (D.C. Cir. 2020). “9 In 2017, while this case was in district court, the Congress amended the ODA to codify a clinical superiority requirement for exclusivity and supersede Depomed’s holding.”

In Re Modafinil Antitrust Litig., 837 F.3d 238 (3rd Cir. 2016). “See 21 U.S.C. § 360cc(a). 14 District of New Jersey on March 28, 2003.”

Depomed, Inc. v. United States Dep't of Health & Human Servs., 66 F. Supp. 3d 217 (D.D.C. 2014). “Elsewhere, the regulations define the term “same drug” — a term that the regulations use in place of the statutory term “such drug” that appears in 21 U.S.C. § 360cc — to mean, in relevant part, “a drug that contains the same active moiety as a previously approved drug and is…”

Catalyst Pharm., Inc. v. Xavier Becerra, 14 F.4th 1299 (11th Cir. 2021). “LAGOA, Circuit Judge: This appeal asks us to determine whether the statutory phrase “same disease or condition” contained in the Orphan Drug Act, see 21 U.S.C. § 360cc, is ambiguous. It is not.”

Genentech, Inc. v. Bowen, 676 F. Supp. 301 (D.D.C. 1987). “On December 12, 1985, the FDA designated Protropin as an orphan drug, thus granting Genentech marketing exclusivity, pursuant to 21 U.S.C. § 360cc, until December 12, 1992.”

— 21 U.S.C. § 360cc(a)(2) — 1 case

K-V Pharm. Co. v. United States Food & Drug Admin., 889 F. Supp. 2d 119 (D.D.C. 2012). “§ 558 (c); section 360cc(a) of the FDCA, 21 U.S.C. § 360cc; and the Due Process Clause of the Fifth Amendment of the United States Constitution, and acted arbitrarily, capriciously, in an abuse of discretion, and in excess of its authority, in *128 violation of the APA, 5 U.”

— 21 U.S.C. § 360cc(b) — 7 cases

Spectrum Pharm., Inc v. Sylvia Burwell, 824 F.3d 1062 (D.C. Cir. 2016). “21 U.S.C. § 360cc. An application will necessarily include only stated indications, not intended off-label uses.”

Jazz Pharm., Inc. v. Robert F. Kennedy Jr., 141 F.4th 254 (D.C. Cir. 2025). “First, in subsection 360cc(a), the Congress replaced “such drug for such disease or condition” with “the same drug for the same disease or condition,” so the FDA “may not approve another application . . . for the same drug for the same disease or condition” for seven years from…”

— 21 U.S.C. § 360cc(b)(1) — 2 cases

— 21 U.S.C. § 360cc(b)(l) — 2 cases

Spectrum Pharm., Inc v. Sylvia Burwell, 824 F.3d 1062 (D.C. Cir. 2016). “21 U.S.C. § 360cc. An application will necessarily include only stated indications, not intended off-label uses.”

— 21 U.S.C. § 360cc(c) — 2 cases

Neurelis Inc. v. Califf (D.D.C. 2025).

— 21 U.S.C. § 360cc(c)(1) — 5 cases

Braeburn Inc. v. U.S. Food & Drug Admin., 389 F. Supp. 3d 1 (D.C. Cir. 2019). “Orphan-designated drugs receive 7-year exclusivity periods under the conditions set forth in 21 U.S.C. § 360cc. Before Congress passed FDARA in 2017, § 360cc provided, with exceptions not relevant here, that if the FDA: [A]pproves an application filed pursuant to section 355 of…”