Internal FDA Emails Reveal How Price, Sanders’s Pressure Played a Role in Competitor’s Approval, Catalyst Claims

Regulatory NewsRegulatory News | 13 February 2020 |  By 

A series of partially redacted internal emails from the US Food and Drug Administration (FDA), released as part of a lawsuit related to competing treatments for the rare disorder Lambert-Eaton Myasthenic Syndrome (LEMS), attempt to show how price and Sen. Bernie Sanders’s (D-VT) pressure played a role in FDA’s approval of a competitor.

The case concerns Catalyst Pharmaceuticals, which sued FDA last June in the US District Court for the Southern District of Florida because FDA approved rival Jacobus Pharmaceutical’s Ruzurgi (amifampridine) for the treatment of LEMS. Catalyst claims that the orphan drug exclusivity on its competing but more expensive LEMS treatment Firdapse (amifampridine) should have barred Jacobus from winning approval for Ruzurgi.

In documents released as part of the suit late last month, Catalyst attempts to show how although FDA “has repeatedly admitted” that it is not allowed to consider drug prices as part of its analysis of drug approvals and exclusivities, the agency did actually consider the $200,000 annual price of Firdapse when approving Ruzurgi.

Ginny Boynton of Pennsylvania told Focus in a phone interview that she has LEMS and has taken both Ruzurgi and Firdapse, and that the price of Firdapse for her would have been closer to $500,000 annually. She said her insurance would currently cover most of that cost, but she does not know what will happen when she switches over to Medicare, and she previously took 3,4-diaminopyridine at no cost for 18 years.

On this issue of price, the suit says that in January 2019, Colleen LoCicero, associate director for regulatory affairs in FDA’s Office of Drug Evaluation I, emailed James Myers, division director of FDA’s division of regulatory policy in the Office of New Drug Policy, and multiple FDA decisionmakers with questions about Ruzurgi’s possible approval.

“The email chain is heavily redacted, but in one unredacted excerpt the Director of CDER’s Office of Medical Policy states: ‘This is a difficult case, but more so perhaps because we know about the competition and price issues,’” Catalyst says.

And within 24 hours of the Ruzurgi approval, an FDA press officer circulated an article from Stat News’ Pharmalot last May, which said the Ruzurgi approval was an “unexpected twist to a simmering controversy over a rare disease drug that earlier this year briefly became a poster child for high-priced medicines.”

The article also said, “Catalyst stock was down as much as 44%” and that if Ruzurgi is “on the market for children, it can be prescribed for adults.” FDA officials including LoCicero, Teresa Buracchio, Kelley Nduom, Jay Sitlani, Ellis Unger and others received the email about the article.

And Unger, according to the suit, responded: “Good Press! Thanks for sending. I like being crafty…Too bad the Catalyst lawyers will be on our doorstep soon.”

In February 2019, Sanders also announced that he would be investigating Firdapse’s price and an FDA staffer forwarded a news article discussing Sanders’ campaign to CDER management. The response from CDER Director Janet Woodcock was: “Do you know where we are with Jacobus?”

At the end of February, Sanders called on the agency to do what it did with the pre-term birth drug Makena, when FDA exercised discretion to allow a competing but unapproved compounded version of the drug. Catalyst says FDA specifically noted in December 2018 that this precedent could be a solution here.

Catalyst also maintains that the pricing controversy was factually misguided because although Ruzurgi “was ultimately priced at approximately $200,000 per year,” Catalyst established a “comprehensive patient financial assistance/insurance navigation program which allowed most patients to pay $10 or less per month for Firdapse out of pocket. Catalyst also committed to distribute the drug for no cost to patients who are unable to obtain insurance coverage, subject to applicable regulatory requirements.”

And Catalyst also claims that FDA funded a study “which apparently served to reduce Jacobus’ costs and thus allow it to offer a lower price.”

Moving forward, Catalyst is calling on the court to provide unredacted versions of FDA’s internal documents, including a “completely redacted memorandum on whether ‘the orphan drug exclusivit[y] the FDA recognized for Firdapse . . . block[s] the approval of Ruzurgi’” and a completely redacted email from LoCicero to a colleague “apparently describing what FDA’s Office of Chief Counsel (‘OCC’) is concluding about exclusivity and related issues.”

Editor's note: Article updated with comment from Ginny Boynton.


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