Looking to Prevail in Court, Catalyst Takes on the FDA in Battle Over LEMS Treatment

It’s a daunting task to take on the federal government in a court battle, but that’s just what Catalyst Pharmaceutical is doing as the Florida-based company is challenging a recent approval from the regulatory agency over a rival medication.

Last year, Catalyst won approval for Firdapse (amifampridine), the first-ever approved treatment for Lambert-Eaton Myasthenic Syndrome (LEMS). Then, in May, Jacobus Pharmaceutical snagged regulatory approval of Ruzurgi, a treatment for pediatric patients with LEMS. Catalyst filed a lawsuit against the U.S. Food and Drug Administration (FDA) challenging that decision. The complaint, which was filed in the United States District Court for the Southern District of Florida, alleges that the approval of Ruzurgi for pediatric patients “violated multiple provisions of FDA regulations regarding labeling.”

Lawsuits against the FDA are fairly common. However, winning the lawsuit will be a tough road for Catalyst, as other pharma companies that challenged the regulatory agency learned. For example, in 2012, two months after KV Pharmaceuticals filed a lawsuit against the FDA for failing to stop compounding companies from making and selling cheaper versions of its premature birth medication, Makena, the court threw the case out. The judge in that case ruled the FDA exercised its discretion in whether or not enforcement should be pursued against compounders and also found the FDA “clearly conveyed the circumstances under which such actions may be taken and when compounding was permitted without violating the law.” In short, the judge said KV failed to bring appropriate claims to the courts.

More recently, Pharmaceutical Manufacturing Research Services (PMRS) filed a lawsuit against the FDA over the agency’s decision to limit the use of the company’s opioid drug RoxyBond to short-term use. The company said the labeling decision would cost it a significant amount of revenue loss. The case was dismissed in January after the courts determined the company didn’t have the standing to challenge the FDA’s approval.

Also last year, a former FDA official charged his former agency with relaxing reporting requirements for clinical trials. Peter Lurie, an ex-associate commissioner for public health strategy and analysis at the FDA, filed a lawsuit against the FDA, the National Institutes of Health and the Department of Health and Human Services.

Last month, Georgia-based Hi-Tech Pharmaceuticals also filed a lawsuit against the regulatory agency over concerns regarding 2-Aminoisopheptane, or DMHA. In April, the FDA raised concern about the dietary supplement. The agency said it is not aware of information that demonstrates the ingredient was lawfully marketed as a dietary ingredient. As a result, the FDA said it believes DHMA as a dietary supplement should be subjected to more rigorous standards. In its filing, Hi-Tech claimed the FDA “bypassed standard rule-making procedures in favor of a campaign of intimidation to try to force companies to destroy their products containing… DMHA.” Hi-Tech said in its complaint that DMHA is a natural product that has been included in its dietary supplements for years. The company said the FDA is attempting to eradicate DMHA from the market, which “exceeds its authority and violates the Administrative Procedures Act.”

Since the approval of Firdapse, Catalyst has had its share of PR nightmares. Most of the issues have had to do with the price of the medication, which caused Sen. Bernie Sanders to publicly castigate the company. Still, Catalyst is seeking to protect its investments in Firdapse R&D with the lawsuit. When the FDA approved Ruzugri last month for pediatric patients, the agency said the use of Ruzugri in the youth population was supported by evidence from well-controlled studies of the drug in adult LEMS patients. That announcement suggested that doctors treating adults with LEMS could opt to prescribe Ruzugri over Catalyst’s Firdapse, which is the cornerstone of Catalyst’s argument.

Additionally, the complaint said the FDA’s decision violated Catalyst’s statutory rights to Orphan Drug Exclusivity and to New Chemical Entity Exclusivity under the Federal Food, Drug, and Cosmetic Act (FDCA). The lawsuit seeks an order vacating the FDA's approval of Ruzurgi.

Patrick J. McEnanty, chairman and chief executive officer of Catalyst Pharmaceuticals, said new chemical entities are required to go through the full regulatory approval process, which requires a demonstration of safety and efficacy.

“We believe the FDA has misapplied its regulations, contradicting decades of precedent and has undercut Catalyst’s orphan drug exclusivity. We are compelled to bring this action, to preserve the specialized regulatory framework provided by the Orphan Drug legislation, and the prospect of future rare disease drug development for all rare disease patients in need of an approved treatment,” McEnanty said in a brief statement.