The U.S. District Court for the District of Columbia has ruled in favor of the Food and Drug Administration (FDA) in a dispute between Jazz Pharmaceuticals and Avadel Pharmaceuticals concerning orphan drug exclusivity for sodium oxybate products used to treat narcolepsy. The court upheld the FDA's decision to approve Avadel's Lumryz, a once-nightly formulation of sodium oxybate, despite Jazz's Xywav, a twice-nightly formulation, already holding orphan drug exclusivity. This decision hinged on the FDA's determination that Lumryz is clinically superior due to its convenience and reduced sleep disruption.
Clinical Superiority and the Orphan Drug Act
The core of the legal battle revolved around the interpretation of the Orphan Drug Act, which grants market exclusivity to drugs designated for rare diseases. Jazz Pharmaceuticals argued that the FDA's approval of Lumryz violated Xywav's existing exclusivity. However, the FDA contended, and the court agreed, that the Orphan Drug Act allows for the approval of a subsequent drug with the same active moiety if it demonstrates clinical superiority, defined as greater efficacy, greater safety, or a major contribution to patient care. In this case, the FDA determined that Lumryz's once-nightly dosing, which avoids disrupting the sleep cycle, constituted a major contribution to patient care compared to Xywav's twice-nightly regimen.
Jazz's Arguments and the Court's Rejection
Jazz Pharmaceuticals challenged the FDA's decision on multiple grounds, asserting that the agency lacked the authority to approve Lumryz during Xywav's exclusivity period. They also claimed that the FDA deviated from a supposed longstanding policy requiring a clinically superior drug to have a comparable safety profile. Jazz highlighted that Lumryz contains more sodium, posing a potential health risk to narcolepsy patients. Furthermore, Jazz argued that the FDA's clinical superiority determination was arbitrary and capricious due to procedural inconsistencies and conflicts with scientific literature.
The court dismissed all of Jazz's arguments. It found no evidence of a "comparable safety" policy within the Orphan Drug Act, agency regulations, or precedent. The court also deferred to the FDA's expertise in assessing the clinical benefits and risks, stating that the agency provided a rational explanation for its decision, considering the trade-offs between sodium intake and sleep disruption. The court acknowledged that the FDA's Sleep Team experts' consultation was within the agency's discretion to modify its procedural rules when justice requires.
Implications for Orphan Drug Exclusivity
This ruling affirms the FDA's authority to determine clinical superiority when evaluating orphan drug applications and to approve subsequent drugs with the same active moiety during an existing drug's exclusivity period if clinical superiority is demonstrated. The decision underscores the importance of clinical benefits, such as improved dosing regimens and reduced side effects, in assessing drug superiority. While Jazz Pharmaceuticals may appeal the decision, the current ruling supports the FDA's stance on orphan drug exclusivity and its commitment to promoting innovation in treatments for rare diseases.
