Eleventh Circuit finds FDA fell foul of Orphan Drug Act
The US Court of Appeals for the Eleventh Circuit has found that the US Food and Drug Administration (FDA) violated the exclusivity of a patent covering a drug for a rare autoimmune disorder, by approving the same treatment specifically for children.
The decision was delivered on Thursday, September 30.
At the centre of the dispute is the Orphan Drug Act 1983, which incentivises the development of treatments for rare diseases. One such incentive is to grant market exclusivity to the manufacturer of an FDA-approved orphan drug for a seven-year period.
Once granted, the FDA may not approve another application “for the same drug for the same disease or condition”, according to the statute.
Florida-based biopharmaceutical company Catalyst Pharmaceuticals is the developer of Firdapse (amifampridine phosphate), which the FDA designated as an orphan drug for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in 2009.
LEMS is a rare autoimmune disease that impacts the signals sent from nerves to muscles, resulting in muscle weakness.
In 2018, the FDA approved Catalyst’s new drug application for Firdapse for the treatment of LEMS in adults. In line with the Orphan Drug Act, the FDA granted exclusivity for seven years.
However, Jacobus Pharmaceuticals filed a new drug application for Ruzurgi (amifampridine) for the treatment of LEMS in children, which the FDA approved in 2019.
Catalyst filed a lawsuit at the US District Court for the Southern District of Florida.
The biopharmaceutical company said the FDA’s approval of Ruzurgi for the treatment of LEMS in children violated the exclusivity granted to Firdapse under the Orphan Drug Act, because Ruzurgi is the same drug as Firdapse and it treats the same disease.
In response to the complaint, the FDA said that approving Ruzurgi did not violate Catalyst’s exclusivity because the approval of Ruzurgi for children constituted a different indication or use from the approval given to Firdapse for adults.
Last year, the US District Court for the Southern District of Florida dismissed Catalyst’s lawsuit. The court found that the language of “same disease or condition” in the Orphan Drug Act is ambiguous, so the FDA’s interpretation of the legislation should be upheld.
Catalyst appealed against the ruling and, last week, the Eleventh Circuit reversed the district court’s decision.
Considering the language of the statute, the Eleventh Circuit held that Congress is not required to define each and every word within legislation. Words are to be understood with their ordinary and plain meaning.
Here, Catalyst had the exclusive right to market the drug for LEMS for seven years and the FDA was, therefore, prohibited from approving the sale of the same drug for the same disease when it was manufactured by Jacobus.
“The FDA’s approval of Ruzurgi was contrary to the unambiguous language of the Orphan Drug Act,” the Eleventh Circuit concluded, remanding the matter back to the district court with instructions that summary judgment be granted in favour of Catalyst.
In a statement released on Wednesday, Patrick McEnany, chairman and CEO of Catalyst, said: “We are extremely pleased with the Eleventh Circuit Court’s decision and are hopeful that it brings to a close this case that is not just important for Catalyst, but for all patients living with rare diseases who depend on medicines that would not be available if not for the efforts and investment of pharmaceutical companies willing to pursue these indications.”
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