A small biotech launched a legal battle Wednesday with the Food and Drug Administration, claiming the regulator's recent approval of a new drug is illegal and should be thrown out.
The lawsuit is the latest chapter in a bizarre competition between Catalyst Pharmaceuticals and Jacobus Pharmaceutical, two small biotechs that both have FDA-approved drugs for a rare disease called Lambert-Eaton Myasthenic Syndrome, or LEMS. Now, Catalyst is suing the agency over last month's approval of Jacobus' drug.
For years, Jacobus offered Ruzurgi (amifampridine) to LEMS patients for free as an unapproved drug through a compassionate use program.
But in 2012, Catalyst licensed the North American rights from BioMarin Pharmaceutical for a very similar version of therapy, called Firdapse (amifampridine phosphate). That set off a race between Catalyst and Jacobus, a family-run private pharma, to conduct clinical testing and secure FDA approval.
Catalyst won that race last year, as Firdapse was approved last November to treat adults with LEMS. Along with the approval came orphan drug exclusivity, a long-standing regulatory incentive of seven years of market exclusivity to encourage development of rare disease treatments.
And Catalyst wound up leveraging that exclusivity to do what many patient advocates and public health voices feared — setting the drug's annual list price at $375,000 with its commercial launch in January. No longer could Jacobus offer its drug for free through compassionate use with an FDA-approved option now on the market.
But then, in a twist that is now the center of Catalyst's lawsuit, the FDA approved Jacobus' drug, Ruzurgi, on May 6, just a few months after OK'ing Catalyst's therapy.
Firdapse was approved only for adults with LEMS, while Jacobus' was cleared for use in children with the disease — a distinction that could be seen as a regulatory workaround to Catalyst's seven-year exclusivity period.
This legitimacy of this labeling is the key issue for the newly-filed lawsuit. In its filing, Catalyst brings three main arguments that should have prevented Ruzurgi's approval.
First, the biotech says the FDA's label wades into the grounds of misleading or false information, claiming this amounts to illegal off-label marketing for Ruzurgi's use in adults. Ruzurgi's label has several sections that outline data showing the drug is safe and effective in adult patients.
"So, for example, no drug approved only for adults can suggest or imply in its labeling that the drug is safe and effective for use in children, or vice versa," the suit states.
The filing also argues there is no clinical difference between adult LEMS and pediatric LEMS — that they are the same disease. Therefore, the FDA violated Catalyst's orphan drug exclusivity period, according to the filing.
Finally, Catalyst's lawyers say that even if a pediatric indication was OK for Jacobus to pursue, the company's supporting clinical data was insufficient.
Despite its pediatric approval, Ruzurgi's OK was primarily based on a clinical study in adult LEMS patients, ranging from 23 to 83 years old, the suit says.
The pediatric LEMS market is a tiny portion of an already rare disease. Jacobus has estimated there are less than 15 pediatric cases of LEMS in the U.S. Roughly 3,000 Americans have LEMS, which is typically diagnosed later in life.
Jacobus also submitted safety data for 15 pediatric patients that were treated in its expanded access programs, showing similar adverse reactions to the adult patients.
Among post-approval requirements, the FDA outlined three activities for Catalyst and 10 for Jacobus, including a juvenile animal toxicology study among other animal studies.
The pediatric approval also raised concern for a Wall Street analyst covering Catalyst.
"What we find more disconcerting is that seizures have been observed in patients without a history of seizures and yet we believe approval was granted without substantive published pediatric safety studies," Charles Duncan, an analyst at Cantor Fitzgerald, wrote last month in a note to investors.
The suit argues that the FDA took these actions based on Firdapse's pricing and subsequent outrage over the cost.
For instance, Sen. Bernie Sanders sent a letter in February to Catalyst, requesting justification for the $375,000 price tag. And after Jacobus' drug was approved, Sanders described it in a May 8 statement as "a victory for patients with LEMS and American taxpayers, and a blow to the greed of Big Pharma."
Catalyst defends the pricing, saying it reflects the company's financial commitment of about $100 million in developing the drug.
The FDA declined to comment on pending litigation, and Jacobus executives did not respond to an interview request by time of publication.