The Sun Will Come Up Tomorrow (or Will It)? FDA Issues Notification About Orphan Drug Exclusivity in Light of Recent Court Decision

Recently, the Food and Drug Administration published a Notification to clarify its policy about orphan drug exclusivity.¹ The agency issued the announcement because the U.S. Court of Appeals for the 11^th Circuit found that FDA erred in approving a drug product, in violation of the Orphan Drug Act. Learn more about another case involving orphan drug exclusivity and a general overview of the regulatory framework here.

The bottom line is that, while FDA agreed to comply with the 11^th Circuit’s decision for the specific case, the agency said that it plans to continue its policy of applying “its regulations tying the scope of orphan-drug exclusivity to the uses or indication for which a drug is approved to matters beyond the scope of the order.”²

The Notification went into effect on January 24, 2023.

Summary

• Both Jacobus Pharmaceutical Company and Catalyst Pharmaceuticals 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 granted Catalyst’s drug orphan-drug exclusivity for the adult indication.
  • Shortly thereafter, FDA approved Jacobus’s drug for the treatment of LEMS in children.
  • FDA followed its longstanding rule, codified in its regulations (e.g., 21 C.F.R. § 316.31), that the orphan-drug exclusivity for Catalyst’s drug protected only the approved use or indication within the designated disease (i.e., adult patients).
  • Catalyst sued FDA, challenging approval of Jacobus’s application, arguing that the statute prohibited FDA from approving Jacobus’s new drug application. 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 found for FDA, holding that the agency reasonably interpreted the statute to tie orphan-drug exclusivity to the uses or indications for which the drug was approved.
• On appeal, the 11^th Circuit reversed, noting that the phrase, “same disease or condition” in the Orphan Drug Act, unambiguously foreclosed FDA’s interpretation. Therefore, orphan-drug exclusivity for Catalyst’s drug blocked FDA’s approval of Jacobus’s drug for all uses or indications within the orphan-designated disease (LEMS).
• In its Notification, FDA stated that it intends to continue to apply its existing regulations linking orphan-drug exclusivity to the uses or indications for which the orphan drug was approved.
  • The agency said 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.
  • FDA said that its regulations incentivize sponsors to continue to develop a drug for use in all persons affected by a rare disease or condition.

AGG Observations

• FDA is not deterred by the 11^th Circuit decision and concluded that it would stick by its interpretation of the Orphan Drug Act.
• Notwithstanding the Catalyst ruling, FDA intends to apply its regulations at 21 C.F.R. Part 316 more narrowly than the 11^th Circuit (i.e., the agency will limit the scope of orphan drug exclusivity to the specific uses or indications in the drug approval).
• While FDA has clarified its position, sponsors now have alternative precedent to cite for potential challenges to this interpretation.
• It looks like not much has changed in FDA’s mind, so further litigation should be expected; “tomorrow, tomorrow, I love ya, tomorrow, you’re always a day away.”

[1] Catalyst Pharms., Inc. vs. Becerra, 14 4^th 1299 (11^th Cir. 2021).

[2] 88 Fed. Reg. 4086 (Jan. 24, 2023).