Catalyzing controversy: FDA emails challenge its claims that price plays no role in approvals
Data dump details FDA’s scramble to approve Ruzurgi as alternative to high-priced Firdapse
Over 200 pages of emails released as a result of litigation against FDA and HHS challenge FDA’s claims that it does not consider price when making approval decisions. The emails pull back the curtain on the agency’s scramble to prevent a drug company from turning an old, cheap medicine into a gold mine.
The docket raises questions about how far FDA can and should stretch its authority to address pricing issues, and whether FDA or Congress should change policies and laws that incentivize companies to obtain marketing approvals for unapproved drugs.
The litigation was filed by Catalyst Pharmaceuticals Inc. (NASDAQ:CPRX), which markets Firdapse amifampridine phosphate to treat adult patients with Lambert-Eaton myasthenic syndrome (LEMS), a rare autoimmune disease.
Firdapse has Orphan Drug status, which provides seven years of market exclusivity.
Catalyst claims FDA illegally approved Ruzurgi amifampridine from Jacobus Pharmaceutical Co. Inc. (Princeton, N.J.) for pediatric LEMS patients. The approval, Catalyst asserts, was intended to undermine its exclusivity -- and its ability to generate over $100 million annually in sales -- by giving physicians the option to prescribe Ruzurgi off label to adults.
In a court filing, the U.S. government asserted that FDA’s decisions were consistent with precedent, promote the interests of pediatric patients, and were not motivated by price.
The court will have to determine if FDA improperly considered the price of Firdapse when it approved Ruzurgi.
“This is a difficult case, but more so perhaps because we know about the competition and price issues.”
Whether legally justified or not, the emails leave no doubt that FDA officials at all levels, including those responsible for reviewing Ruzurgi, were acutely aware of pricing issues. They