Rule2023-01179
Clarification of Orphan-Drug Exclusivity Following Catalyst Pharms., Inc. v. Becerra; Notification
Primary source
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Published
January 24, 2023
Effective
January 24, 2023
Issuing agencies
Health and Human Services DepartmentFood and Drug Administration
Abstract
The Food and Drug Administration (FDA or Agency) is publishing this notification in light of the recent decision by the U.S. Court of Appeals for the Eleventh Circuit in Catalyst Pharms., Inc. v. Becerra. The Catalyst decision addressed the orphan-drug exclusivity provision of the Federal Food, Drug, and Cosmetic Act (FD&C Act), as amended by the Orphan Drug Act and subsequent amendments, and concluded that FDA's approval of Jacobus Pharmaceutical Company's (Jacobus's) drug (the drug at issue in the litigation) must be set aside. Consistent with the court's decision, FDA has set aside its approval of Jacobus's drug. This notification announces that, at this time, while complying with the court's order in Catalyst, FDA intends to continue to apply its regulations tying the scope of orphan-drug exclusivity to the uses or indications for which a drug is approved to matters beyond the scope of that order.
Full Text
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<title>Federal Register, Volume 88 Issue 15 (Tuesday, January 24, 2023)</title>
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[Federal Register Volume 88, Number 15 (Tuesday, January 24, 2023)]
[Rules and Regulations]
[Pages 4086-4087]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2023-01179]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
21 CFR Part 316
[Docket No. FDA-2011-N-0583]
Clarification of Orphan-Drug Exclusivity Following Catalyst
Pharms., Inc. v. Becerra; Notification
AGENCY: Food and Drug Administration, Department of Health and Human
Services (HHS).
ACTION: Notification.
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SUMMARY: The Food and Drug Administration (FDA or Agency) is publishing
this notification in light of the recent decision by the U.S. Court of
Appeals for the Eleventh Circuit in Catalyst Pharms., Inc. v. Becerra.
The Catalyst decision addressed the orphan-drug exclusivity provision
of the Federal Food, Drug, and Cosmetic Act (FD&C Act), as amended by
the Orphan Drug Act and subsequent amendments, and concluded that FDA's
approval of Jacobus Pharmaceutical Company's (Jacobus's) drug (the drug
at issue in the litigation) must be set aside. Consistent with the
court's decision, FDA has set aside its approval of Jacobus's drug.
This notification announces that, at this time, while complying with
the court's order in Catalyst, FDA intends to continue to apply its
regulations tying the scope of orphan-drug exclusivity to the uses or
indications for which a drug is approved to matters beyond the scope of
that order.
DATES: The policy set out in this document is effective January 24,
2023.
FOR FURTHER INFORMATION CONTACT: Aaron Friedman, Office of Orphan
Products Development, Food and Drug Administration, 10903 New Hampshire
Ave., Silver Spring, MD 20993, 301-796-2989.
SUPPLEMENTARY INFORMATION:
I. Background
On September 30, 2021, the U.S. Court of Appeals for the Eleventh
Circuit issued a decision in Catalyst Pharms., Inc. v. Becerra
(Catalyst), 14 F.4th 1299 (11th Cir. 2021).
At the time of the litigation, Jacobus and Catalyst Pharmaceuticals
(Catalyst) each had orphan-drug designation for the drug amifampridine
for the treatment of Lambert-Eaton myasthenic syndrome (LEMS). In
November 2018, FDA approved Catalyst's drug for the treatment of LEMS
in adults. FDA recognized Catalyst's drug as eligible for orphan-drug
exclusivity for its only approved indication--the treatment of LEMS in
adults.
In May 2019, FDA approved Jacobus's drug for the treatment of LEMS
in children. In approving Jacobus's drug, FDA followed its longstanding
rule, codified in its regulations, that the orphan-drug exclusivity for
Catalyst's drug protected only the approved use or indication within
the designated disease. See 21 CFR 316.3(b)(12), 316.31(a)-(b). The
regulation in 21 CFR 316.31(b) states, in part, that: ``Orphan-drug
exclusive approval protects only the approved indication or use of a
designated drug.'' \1\
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\1\ Emphasis added. Other regulatory provisions also reflect the
understanding that orphan-drug exclusivity is tied to the use or
indication for which the drug was approved. See Sec. 316.3(b)(12)
(stating that ``no approval will be given to a subsequent sponsor of
the same drug for the same use or indication for 7 years'' (emphasis
added)); see also id. Sec. 316.31(a) (explaining that FDA may
approve an orphan drug for ``select indication(s) or use(s) within
the rare disease or condition for which the drug was designated''
and that ``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'' (emphases
added)).
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In June 2019, Catalyst filed suit against FDA, challenging FDA's
approval of Jacobus's application under the Administrative Procedure
Act, 5 U.S.C. 701-706. Among other things, Catalyst argued that the
phrase ``same disease or condition'' in the Orphan Drug Act, 21 U.S.C.
360cc(a), unambiguously prohibited FDA from approving Jacobus's drug
application. Specifically, Catalyst argued that the Orphan Drug Act
required orphan-drug exclusivity to extend to all uses or indications
within the orphan-designated disease or condition--even uses or
indications for which Catalyst had not received approval, such as the
treatment of LEMS in children.
The district court rejected Catalyst's argument that the Orphan
Drug Act required orphan-drug exclusivity to apply to all uses or
indications within the orphan-designated disease or condition. The
district court concluded that, given the context and the overall
statutory scheme, the statute was ambiguous on the disputed issue, and
that FDA had reasonably interpreted the statute to tie orphan-drug
exclusivity to the uses or indications for which the drug was approved.
On appeal, the U.S. Court of Appeals for the Eleventh Circuit
reversed. The circuit court concluded that the phrase ``same disease or
condition'' in the Orphan Drug Act, 21 U.S.C. 360cc(a), unambiguously
foreclosed FDA's interpretation of the provision. Accordingly, the
circuit court held that orphan-drug exclusivity for Catalyst's
[[Page 4087]]
drug blocked FDA's approval of Jacobus's drug for all uses or
indications within the orphan-designated disease (LEMS)--even though
Catalyst's drug was approved at that time only for use in the treatment
of LEMS in adults. The court concluded that FDA's approval of Jacobus's
drug for the treatment of LEMS in children must be set aside.
Consistent with the court's decision, the Agency set aside the approval
of Jacobus's drug.
II. Orphan-Drug Exclusivity
The Agency is issuing this statement to address the uncertainty
created by the circuit court's decision in Catalyst. The court ordered
FDA to set aside its approval of Jacobus's drug, and FDA has set aside
that approval. This notification announces that, at this time, in
matters beyond the scope of that court order, FDA intends to continue
to apply its existing regulations tying orphan-drug exclusivity to the
uses or indications for which the orphan drug was approved. The Agency
believes that this approach is appropriate for several reasons. FDA
continues to believe that the statutory text does not unambiguously
require that orphan-drug exclusivity extend to the entire disease or
condition for which a drug received orphan-drug designation if the drug
is only approved for some uses within that disease or condition.
Further, FDA believes that its statutory interpretation embodied in its
regulations best advances the Orphan Drug Act's purposes, appropriately
balancing the need to incentivize the development of drugs for rare
diseases and conditions with the need to provide patient access to
orphan drugs. The regulations accomplish this by tying the scope of
orphan-drug exclusivity to only the approved use or indication of the
drug, which permits other sponsors to obtain approval of the drug for
uses or indications within the same orphan-designated disease or
condition that have not yet been approved (i.e., that are ``new'').
Under the regulations, a drug approved for a new use or indication
within the same orphan-designated disease or condition may also be
eligible for orphan-drug exclusivity for such use or indication. These
regulations incentivize sponsors to continue to develop a drug for use
in all persons affected by a rare disease or condition. Thus, FDA
believes that continued adherence to its validly promulgated
regulations will best serve the public health by facilitating patient
access to orphan drugs, especially for difficult-to-study patients such
as young children.
III. Conclusion
For the above reasons, at this time, the Agency intends to continue
to apply its longstanding regulations tying the scope of orphan-drug
exclusivity to the uses or indications for which the orphan drug was
approved.
Dated: January 18, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023-01179 Filed 1-23-23; 8:45 am]
BILLING CODE 4164-01-P
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</html>Indexed from Federal Register on January 24, 2023.
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