In­ter­nal FDA emails re­veal how price, Sanders’ pres­sure played a role in com­peti­tor’s ap­proval, Cat­a­lyst claims

A se­ries of par­tial­ly redact­ed in­ter­nal emails from the 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 the 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 the 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.

For in­stance, 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’s 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 the FDA ex­er­cised dis­cre­tion to al­low a com­pet­ing but un­ap­proved com­pound­ed ver­sion of the drug. Cat­a­lyst says the 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 the 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 the 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.”

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