An incensed Catalyst Pharma sues the FDA, accusing agency of bowing to political pressure and breaking federal law
After hinting it was exploring the legality of the FDA’s approval of a rival drug from family-run company Jacobus Pharmaceuticals, Catalyst Pharmaceuticals on Wednesday filed a lawsuit against the health regulator — effectively accusing the agency of bowing to political pressure surrounding skyrocketing drug prices.
Before Catalyst’s Firdapse (which carries an average annual list price of $375,000) was sanctioned for use in Lambert-Eaton myasthenic syndrome (LEMS) by the FDA, hundreds of patients had been able to access a similar drug from compounding pharmacies for a fraction of the cost, or Jacobus’ for free, as part of an FDA-ratified compassionate use program. But the approval of the Catalyst drug — accompanied by market exclusivity spanning seven years — effectively precluded Jacobus and compounding pharmacies from selling their versions.
Then, in an unexpected twist, weeks ago the FDA endorsed New Jersey-based Jacobus’ version in pediatric patients, on the basis of adult data — a move that could spark off-label prescription in adults (As far as the FDA is concerned, doctors can prescribe drugs for off-label use when they judge that it is medically appropriate for their patient). Catalysts’ shares $CPRX sank on the announcement and have not since revived. Adding fuel to the fire, earlier this week Jacobus revealed its drug, Ruzurgi, will carry a list price that is less than half of Firdapse’s.
Catalyst maintains that typically, covered patients pay less than $10 per month out-of-pocket.
In LEMS patients, the body’s own immune system launches an attack on the neuromuscular junction — which connects nerves and muscles. The condition is associated with other autoimmune diseases, but tends to occur in patients with cancer. Globally it is estimated to affect three per million individuals, according to the FDA, but its pediatric prevalence is not clear. Catalyst suggested there are 3,000 LEMS US patients, of which 300 are on Firdapse. Some estimates suggest there are fewer than three dozen pediatric patients in the United States.
Under federal law, the agency is meant to treat all companies in the same manner. Catalyst has asserted the agency undermined the company’s orphan drug exclusivity, and violated federal law by playing favorites in context of a hypervigilant pricing environment.
The approval letters for Catalyst and Jacobus suggest that while the former submitted a body of clinical trial evidence as part of the package the FDA reviewed, the latter did not submit data from certain toxicology studies. Instead, the FDA has asked Jacobus to provide these data as a post-marketing requirement.
“We felt that the FDA’s approval of Ruzurgi was arbitrary, capricious and not in accord with the Food, Drug & Cosmetic Act,” Catalyst chief Patrick McEnany told Endpoints News.
“We believe the FDA was improperly influenced in making this decision by political pressures with regard to high drug pricing and we think it sets a horrible precedent for other companies working to develop drugs to treat rare diseases — it could in fact discourage companies from investing millions of dollars and spending many years to do this,” he said.
Earlier this year, Vermont Senator Bernie Sanders — ahead of his announcement to make a second attempt at the presidency — spotlighted Catalyst for the “immoral exploitation of patients.”
McEnany emphasized that Catalyst has spent more than $100 million dollars in developing the drug over the last seven years — and that his hope is the lawsuit will culminate in the withdrawal of the Ruzurgi approval.
An FDA spokesperson said the agency does not comment on pending litigation. Endpoints News has contacted Jacobus for comment.
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