Sanders escalates fight with Catalyst, asking FDA to allow unbranded copies of $375K Firdapse

After a seemingly vain attempt to appeal to Catalyst Pharmaceuticals’ conscience, Sen. Bernie Sanders, I-Vt., has turned to the FDA, asking the agency to allow cheaper, unapproved versions of Catalyst’ Firdapse to be distributed as they had been over the past three decades.

In a bid to circumvent Catalyst's monopoly on the pricey drug, Sanders called on the agency to forego punishing pharmacies and manufacturers that had been providing its equivalent for free, Reuters reported, citing a letter sent to FDA Commissioner Scott Gottlieb, M.D., on Tuesday.

"Catalyst may be the most recent company to exploit their monopoly after receiving FDA approval for an inexpensive old drug, but they were certainly not the first," Sanders said in the letter, as quoted by Reuters. The pharma has seven-year exclusivity on Firdapse thanks to its orphan drug status.

Recently approved by the FDA to treat the rare neuromuscular disorder of Lambert-Eaton myasthenic syndrome (LEMS), Firdapse bears a list price of $375,000. Its active ingredient, 3,4-DAP, was previously distributed by family-owned drugmaker Jacobus Pharmaceuticals at no cost for three decades under the FDA’s compassionate use program.

Sanders took issues with that huge cost jump, calling it "a blatant fleecing of American taxpayers" and a prime example of "corporate greed" in a letter sent to Catalyst earlier this month.

Catalyst Chairman and CEO Patrick McEnany posted his firm’s response last week, invoking some familiar arguments biopharma companies have often used to justify their prices. McEnany said the company spent "millions of dollars" on the drug’s R&D, and an FDA approval means Firdapse is now a safety- and efficacy-guaranteed option for patients and therefore shouldn’t be considered an old drug.

Sanders’ request is likely referring to drug compounding regulated under Section 503A and Section 503B of the Federal Food, Drug and Cosmetic Act. The 503A part covers licensed pharmacists and physicians, while the 503B chapter covers the so-called "outsourcing facilities," or simply put, manufacturers. Both sections have specific items regarding compounding drugs that are "essentially a copy of an approved drug."

Gottlieb’s FDA last January provided final guidance for the industry on how the agency intends to enforce those provisions. And that guidance could make Sanders’ request unachievable by law.

Specifically, the law forbids a pharmacist to compound an approved drug "regularly or in inordinate amounts." The FDA, in its own explanation (PDF), said it wouldn't take enforcement action if the compounder fills four or fewer prescriptions in a calendar month. As for "outsourcing facilities," to which 3,4-DAP’s original distributor Jacobus could belong, the law is quite clear; they’re forbidden to compound FDA-approved drugs unless there’s a shortage.

FDA had previously allowed compounding of marketed drugs, most notably in the case of KV Pharmaceutical’s Makena, a hormone given to pregnant women with a history of giving birth prematurely. Before that drug was approved, it had been compounded by pharmacies at $10 to $20 per dose. But KV set Makena's launch price at $1,500 a dose, and even on state discount programs, it still carried a retail price of $595.

In its statement back in 2011, the agency specifically said it allowed that compounding "[i]n order to support access to this important drug, at this time and under this unique situation."

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