Catalyst Pharmaceuticals received a positive decision from the 11th Circuit Court of Appeals over a suit against the U.S. FDA’s approval of a competitor’s amifampridine product.
Coral Gables, Fla.-based Catalyst Pharmaceuticals received a positive decision from the 11th Circuit Court of Appeals over a suit against the U.S. Food and Drug Administration’s (FDA) approval of a competitor’s amifampridine product.
In November 2018, the FDA approved Catalyst’s Firdapse (amifampridine), the first-ever approved drug for Lambert-Eaton myasthenic syndrome (EMS). Then the agency approved Jacobus Pharmaceutical’s Ruzurgi (amifampridine) for LEMS, only for patients between the ages of six to 17.
The FDA said in its announcement that the use of Ruzurgi in the pediatric population was supported by data from clinical trials of the drug in adult LEMS patients, which suggested that physicians treating adults with LEMS could choose to prescribe Ruzurgi over Catalyst’s Firdapse.
LEMS is an autoimmune disease affecting the connection between nerves and muscles. It results in weakness and fatigue. It affects about one in 100,000 people in the U.S. and approximately 3 million globally. If it affects respiratory muscle, it can be life-threatening.
The original case came with a political angle as well. Catalyst had set the list price of Firdapse at $375,000. Senator Bernie Sanders (I-Vt), an outspoken critic of high drug prices, accused Catalyst of price gouging. He specifically cited Jacobus Pharmaceutical, which gave away treatments to 200 LEMS patients each year. He also encouraged the FDA to approve Ruzurgi, which had only been provided to LEMS patients under the FDA’s compassionate use program.
In June 2019, Catalyst filed a lawsuit against the FDA over the agency’s decision to approve Ruzurgi. The FDA denied allegations in the lawsuit, arguing that although they both contain the same active ingredient, LEMS in adults is not the same as LEMS in children between the ages of six and 17.
In September 2020, a district judge dismissed the Catalyst lawsuit. District Court Judge Beth Bloom argued that Catalyst’s interpretation of the relevant laws didn’t support its case. Bloom wrote that “Catalyst’s view of [applicable law] is not necessarily wrong, but it is not the only reasonable way to interpret the plain language of the statute.” Bloom admitted that the wording of the law was ambiguous but sided with the FDA’s interpretation.
Now, the 11th Circuit Court has reversed the District Court’s decision and remanded it to the District Court with instructions to enter summary judgement for Catalyst. They essentially agreed with Catalyst’s argument that the FDA’s approval of Ruzurgi violated Catalyst’s rights to Orphan Drug Exclusivity.
“We are extremely pleased with the 11th 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,” said Patrick J. McEnany, Catalyst’s Chairman and Chief Executive Officer. “The purpose of the Orphan Drug Act is to encourage pharmaceutical companies to make the R&D investments necessary to bring FDA-approved therapies to patient populations living with very rare diseases, and the biggest incentive the Orphan Drug Act provides is the ability to have an exclusive market within that rare disease for their drug for seven years once they obtain approval.”
He went on to say, “The Orphan Drug Act has proven successful for the more than 350 rare diseases that now have an FDA-approved treatment, but there still remains about 6,500 rare disease that do not have an approved therapy — and the incentives to develop drugs to treat these rare diseases need to remain intact. This decision increases the hope for the patients living with these diseases that one day soon an approved drug for their rare disease might be a reality. This is a goal that we feel we share with the FDA despite this dispute, and we look forward to working with the FDA in the future on these issues.”
