FDA Won’t Change Approach to Orphan Exclusivity after Court Ruling

The U.S. Food and Drug Administration published a notification in the Federal Register that said it will comply with the federal court’s ruling in a case over its application of the Orphan Drug Act, but will continue to tie the scope of orphan drug exclusivity to the indication or uses for which a drug is approved.

Photo: FDA Commissioner Robert Califf

The agency has not recognized orphan exclusivity for any product since November 2021 and has left uncertain the status of orphan exclusivity for these products following a ruling at the end of September from the U.S. Court of Appeals for the Eleventh Circuit.

Catalyst won FDA approval for its drug Firdapse in late 2018 to treat adults with LEMS and set the annual list price of the drug at $375,000. The FDA then approved Jacobus Pharmaceutical’s Ruzurgi for pediatric patients age 6 to 16. Both Firdapse and Ruzurgi are amifampridine and Catalyst charged that in approving Jacobus’ drug, it violated its right to orphan drug exclusivity, which prohibits the FDA from approving the same drug for the same indication for a period of seven years.

Jacobus priced its LEMS pill at just under half the cost of a similar dosage of Catalyst’s pill. Catalyst charged that the FDA’s approval of Ruzurgi was an illegal regulatory workaround of its seven years of exclusivity for Firdapse. While a drug approved for pediatric use can be prescribed off-label for adults, a drug approved for adult use cannot be prescribed for pediatric patients under the Orphan Drug Act as well as other provisions.

Catalyst sued the FDA saying the agency illegally approved Ruzurgi, but a U.S. district court dismissed the suit in October 2020 saying that that the phrase in the statute “same disease or condition” was ambiguous. The U.S. Court of Appeals for the Eleventh Circuit, however, reversed the decision, finding that the statute was clear when it said “same disease or condition.” It found Catalyst orphan drug exclusivity prevented Jacobus from winning approval for all indications and uses for LEMS.

The FDA said it issued the statement in the Federal Register to address the “uncertainty” created by the ruling in the Catalyst case. While it is honoring the court’s ruling in the Catalyst case, it is not applying the decision more broadly. Instead, it will continue to apply orphan drug exclusivity more narrowly than the court’s interpretation.

“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,” the agency said in its notice. “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.”

Author: Rare Daily Staff