Catalyst plays the R&D card to defend Firdapse’s $375K sticker against Bernie Sanders' ire

Caught in the crosshairs of Sen. Bernie Sanders, I-Vt., Catalyst Pharma has come up with a rationale for the $375,000 price tag on its rare disease therapy Firdapse—and it includes some well-worn arguments often invoked by biopharma companies.

Sanders, who’s been after drugmakers several times for their high drug prices, questioned the justification for Firdapse’s price in a scathing letter earlier this month, calling it “corporate greed.”

Catalyst Chairman and CEO Patrick McEnany posted his firm’s reply on Thursday. The main idea? Catalyst has shelled out, and will continue to invest, “millions of dollars” into the drug’s R&D. And getting an FDA approval means that all patients—not just a few—with rare autoimmune disease Lambert-Eaton myasthenic syndrome (LEMS) will have access to it.

The chief executive argues that Firdapse’s price is in line with those of other drugs for ultraorphan diseases of similar severity and is meant to “properly compensate” for the costs incurred with developing, producing and marketing the therapy.

To be fair, $375,000 a year for an orphan drug is not uncommon these days. But Sanders takes issue with that sticker, because the active ingredient of the drug, 3,4-diaminopyridine, has been around for decades. Firdapse is a tweaked version of that same active ingredient rather than a novel development.

Plus, 3,4 DAP had been given to LEMS patients for free under the FDA’s compassionate use pathway for about three decades, by family-owned drugmaker Jacobus Pharmaceuticals. Catalyst only got hold of the North American rights in 2012—along with market exclusivity tied to its orphan drug status—and worked it through the FDA finish line last November. And it immediately stirred up controversy by pricing the drug at its current tag.

Drugmakers’ practice of buying rights to a drug and then charging high prices has drawn repeated criticism, notably in the case of Martin Shkreli’s Turing Pharma for jacking up Daraprim’s price by 5,000%. Daraprim was already approved when Turing bought it. But Sanders, who is now seeking the Democratic presidential nomination for 2020, was himself instrumental in a case more analogous to Catalyst's. Back in 2017, he and Rep. Elijah Cummings, D-Md., fired at Marathon Pharmaceuticals, which had put an $89,000 tag on Duchenne muscular dystrophy drug Emflaza, a decades-old product that had been sold overseas at $1,000 per year.

RELATED: Catalyst holds firm on $300,000-plus net for Firdapse despite Bernie Sanders’ demands to justify the price

Industry watchers have taken aim at the FDA for what some consider a loophole in the orphan program that awards exclusivity to drugs for rare diseases, apparently regardless of the difficulty of developing a particular drug. “I will ask FDA Commissioner Gottlieb to act immediately to help patients who are suffering,” Sanders said in a statement, as quoted by Reuters.

But the agency was simply following the Orphan Drug Act drafted by Congress. And Catalyst also said the designation “was Congress’ way of incentivizing pharmaceutical companies” to develop treatments for rare disorders.

Sanders, in his original inquiry on Feb. 4, said Catalyst knew Firdapse was clinically effective, given that 3,4 DAP had been used for decades in Europe and, on a more limited basis, the U.S. McEnany, without providing any specific numbers, argued that Catalyst has taken on financial risks by spending “millions of dollars” to gather evidence from clinical studies to win FDA approval. The company continues to spend millions a year in postmarketing studies to confirm the drug’s safety and efficacy, and it’s evaluating Firdapse for other rare neuromuscular diseases, the company said.

To hear McEnany tell it, the final FDA approval somehow makes Firdapse not an “old drug” as Sanders put it, even though its active ingredient is decades old, because it's a different formulation. It’s also a game changer for patients, he argues.

Before Firdapse's FDA nod, only about 200 LEMS patients in the U.S. were receiving the drug under the compassionate use program. Catalyst estimates that 3,000 patients have LEMS and could benefit from Firdapse. Plus, McEnany contends, obtaining a drug through the compassionate use program is cumbersome for physicians and patients. “The approval of Firdapse by the FDA means that ALL LEMS patients in the United States now have access to this much-needed medication,” he said in the letter.

Indeed, compassionate use doesn’t cover all patients, but one could reasonably argue that neither does a $300,000-plus price tag. To that end, Catalyst said it has developed “an array of financial assistance programs that are available to reduce patient copays and deductibles to a nominal affordable amount.” According to the company, a copay assistance program could keep patient out-of-pocket costs to $10 or less per month.

But payers are still on the hook for their share of the cost—and McEnany said earlier this month that Catalyst expects to net $300,000 on Firdapse's $375,000 list price after discounts and rebates.