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# FDA Backs Status Quo in Orphan Drug Exclusivity
- URL: https://www.fdaweb.com/fda-backs-status-quo-in-orphan-drug-exclusivity/
- Published: 2023-01-23T12:00:00.000Z
- Updated: 2026-09-14T18:12:47.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5153593

Despite an appeals court decision suggesting FDA was wrong to base previous orphan drug exclusivity decisions on the uses or indications for which a drug is approved instead of all uses or indications within an orphan-designated disease or condition, a just-posted agency notice says it has considered the court decision as only applying to that case; all other exclusivity decisions will be based on previous and long-standing rulings related to a drug’s approved uses/indications.

Orphan drug exclusivity (ODE) has been in limbo since the 9/2021 11th Circuit Court of Appeals [decision](https://media.ca11.uscourts.gov/opinions/pub/files/202013922.pdf?ref=fdaweb.com) in *Catalyst Pharms v. Becerra* struck down the agency’s indication-specific interpretation of ODE ([see earlier story](https://www.fdaweb.com/appeals-court-reverses-catalyst-firdapse-loss-to-fda/)). In the case, Catalyst argued that because FDA approved its NDA and granted ODE for amifampridine for treating Lambert-Eaton myasthenic syndrome (LEMS) in adult patients, it was then wrong for the agency to grant ODE for Jocubus’ amifampridine and its use for treating LEMS in children.

FDA’s [new notice](https://public-inspection.federalregister.gov/2023-01179.pdf?ref=fdaweb.com) indicates that it has set aside its approval of Jacobus’s drug. It says it “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,” the notice continues, “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.”

Meanwhile, FDA had been working with Congress and hoping for a legislative fix to articulate the agency’s long-standing practice of attaching ODE to uses or indications for which the orphan drug was approved. However, a proposed provision was absent in last month’s omnibus spending bill ([see story](https://www.fdaweb.com/fda-reforms-included-in-year-end-omnibus-bill/)) and that essentially forced FDA’s hand to issue a clarifying notice.