Court Supports Catalyst Pharma’s Appeal for Orphan Drug Exclusivity for Firdapse for LEMS

One year after a federal judge dismissed a lawsuit charging the U.S. Food and Drug Administration violated its rights to Orphan Drug exclusivity for Firdapse for pediatric patients with Lambert-Eaton myasthenic syndrome, Catalyst Pharmaceuticals won a reversal of the decision by the 11th Circuit Court of Appeals.

Photo: Patrick McEnany, chairman and CEO of Catalyst Pharmaceuticals

The Appeals Court adopted Catalyst’s core argument that the FDA’s approval of Ruzurgi violated Catalyst’s rights to Orphan Drug exclusivity and remanded the matter back to the District Court with instructions to enter summary judgment for Catalyst.

“We are extremely pleased with the 11th Circuit Court’s decision and are hopeful that it brings to a close this case,” said Patrick McEnany, chairman and CEO of Catalyst Pharmaceuticals. 

In June 2019, Catalyst filed a lawsuit against the FDA demanding it vacate its approval of Jacobus’ drug Ruzurgi for the treatment of Lambert-Eaton Myasthenic syndrome, or LEMS, in pediatric patients. Catalyst’s drug Firdapse was approved in late 2018 to treat adults with LEMS. LEMS is a rare autoimmune condition in which the body attacks the junction between nerves and muscles.

The lawsuit was part of an ongoing controversy over the price of a drug (amifampridine) that had been available for free from Jacobus under an FDA compassionate use program. That ended when the FDA approved Catalyst’s Firdapse in late November 2018. Catalyst then proceeded to price its drug at an annual list price of $375,000 along with a patient assistance program. The price sparked public outrage.

Only a few months after the Catalyst approval, the FDA approved Jacobus’ LEMS drug for use in pediatric patients based on data from studies of adult LEMS patients. Jacobus announced that it was pricing its LEMS pill at just under half the cost of a similar dosage of Catalyst’s pill.

Catalyst had argued the approval of Jacobus’ 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.

The Appeals Court said the district court erred in its determination that the phrase “same disease or condition” is ambiguous is not supported by the statutory text. In reversing the district court’s ruling, Judge Barbara Lagoa of the 11th Circuit Court of Appeals wrote:

“The FDA’s approval of Ruzurgi was contrary to the unambiguous language of the Orphan Drug Act. Catalyst Pharmaceuticals, Inc., held the exclusive right to market, Firdapse, an orphan drug, for a period of seven years in order to treat the rare autoimmune disease, LEMS. Because it is undisputed that none of the statutory exceptions to Catalyst’s market exclusivity apply, the FDA was prohibited from approving for sale the same drug manufactured by Jacobus Pharmaceutical Company, Inc., to treat the same autoimmune disease during the period of Catalyst’s market exclusivity. As a result, the FDA’s agency’s action was arbitrary, capricious, and not in accordance with law, and its approval of Ruzurgi must be set aside.”

Catalyst is preparing to transition LEMS patients from Ruzurgi to Firdapse through its Catalyst Pathways patient services program.

Author: Rare Daily Staff