Weeks ago, the FDA en­dorsed a Lam­bert-Eaton myas­thenic syn­drome (LEMS) drug from fam­i­ly-run New Jer­sey-based com­pa­ny called Ja­cobus Phar­ma­ceu­ti­cals in pe­di­atric pa­tients, on the ba­sis of adult da­ta, to the sur­prise of Cat­a­lyst Phar­ma­ceu­ti­cals which on­ly last year got its pricey — yet sim­i­lar — treat­ment for the rare au­toim­mune dis­or­der across the fin­ish line in adults. It has now been re­vealed that Ja­cobus’ ver­sion car­ries a price that is less than half of Cat­a­lyst’s Fir­dapse — a move that could fu­el off-la­bel pre­scrip­tion in adults.

Be­fore Cat­a­lyst’s Fir­dapse was sanc­tioned for use by the FDA, hun­dreds of pa­tients had been able to ac­cess a sim­i­lar drug from com­pound­ing phar­ma­cies for a frac­tion of the cost, or Ja­cobus’ for free, as part of an FDA-rat­i­fied com­pas­sion­ate use pro­gram. But the ap­proval of the Cat­a­lyst drug, ac­com­pa­nied by mar­ket ex­clu­siv­i­ty span­ning sev­en years — ef­fec­tive­ly pre­clud­ed Ja­cobus and com­pound­ing phar­ma­cies from sell­ing their ver­sions.

Dos­ing for any of these treat­ments is based on the pa­tient’s weight and dis­ease sever­i­ty. Cat­a­lyst’s Fir­dapse, which is ap­proved with a max­i­mum dose of 80 mg, car­ries an av­er­age list price of $375,000 a year. The com­pa­ny does not dis­close a per pill num­ber, a Cat­a­lyst spokesper­son told End­points News. 

The list price for Ruzur­gi is $80 for each 10 mg tablet, and Ja­cobus’ treat­ment is ap­proved up to a max­i­mum dose of 100 mg, Lau­ra Ja­cobus, who runs the pri­vate­ly-held com­pa­ny, told End­points News. “As an ex­am­ple, the whole­sale cost for a 60 mg dos­ing reg­i­men would be $175,200.00 an­nu­al­ly.  The cost to sup­port a pa­tient re­quir­ing a dai­ly dose of 100 mg would be $292,000.00 an­nu­al­ly.”

Ver­mont Sen­a­tor Bernie Sanders — ahead of his an­nounce­ment to make a sec­ond at­tempt at the pres­i­den­cy — spot­light­ed Cat­a­lyst for the “im­moral ex­ploita­tion of pa­tients”. Oth­er crit­ics of Cat­a­lyst’s pric­ing strat­e­gy sug­gest­ed that de­pend­ing on how Ja­cobus prices its drug, Ruzur­gi, in­sur­ers could be per­suad­ed to fa­vor it over Cat­a­lyst’s prod­uct de­spite be­ing of­fi­cial­ly ap­proved for pe­di­atric use on­ly. As far as the FDA is con­cerned, doc­tors can pre­scribe drugs for off-la­bel use when they judge that it is med­ical­ly ap­pro­pri­ate for their pa­tient.

Fir­dapse land­ed on the US mar­ket this Jan­u­ary, and Cat­a­lyst has not­ed it has en­coun­tered min­i­mal push­back from pay­ers, and in­di­cat­ed that typ­i­cal­ly, cov­ered pa­tients pay less than $10 per month out-of-pock­et.

In a note pub­lished last month, Sun­Trust Robin­son Humphrey’s Ed­ward Nash sug­gest­ed that de­spite sug­ges­tions to the con­trary, “(W)e have not seen any prece­dent where pay­ers cov­er an off-la­bel drug for use in un­ap­proved pa­tient pop­u­la­tion”.

Mean­while, HC Wain­wright’s An­drew Fein sug­gest­ed that Ja­cobus, de­spite the rep­u­ta­tion of a “mod­ern-day Robin Hood” is not equipped with the in­fra­struc­ture nor the ex­pe­ri­ence to sup­port a com­mer­cial push of Ruzur­gi, even with the ap­proval in hand.

“Hand­i­capped by le­gal rea­sons, we do not be­lieve that Ja­cobus can open­ly pro­mote off-la­bel use in LEMS adults…It is un­clear if and how Ja­cobus would be able to ex­pand com­mer­cial pen­e­tra­tion oth­er than through pro­mot­ing a pro­lif­er­a­tion of off-la­bel use at aca­d­e­m­ic cen­ters that were en­rolled in the com­pas­sion­ate pro­gram (which mounts to ap­prox­i­mate­ly 200 pa­tients),” he wrote in a May note.

In LEMS pa­tients, the body’s own im­mune sys­tem launch­es an at­tack on the neu­ro­mus­cu­lar junc­tion — which con­nects nerves and mus­cles. The con­di­tion can as­so­ci­at­ed with oth­er au­toim­mune dis­eases, but tends to oc­curs in pa­tients with can­cer. Its preva­lence in pe­di­atric pa­tients is not known, but glob­al­ly it is es­ti­mat­ed to af­fect three per mil­lion in­di­vid­u­als, ac­cord­ing to the FDA. In a re­cent in­vestor pre­sen­ta­tion Cat­a­lyst sug­gest­ed there are 3,000 LEMS US pa­tients, of which 300 are on Fir­dapse.

A STAT re­port pub­lished on Mon­day sug­gest­ed that some adult LEMS pa­tients have in­sin­u­at­ed that Fir­dapse is not ef­fec­tive enough and more tablets be­yond the ap­proved 80 mg dose must be tak­en for re­lief — these ex­tra pills sad­dle them with out-of-pock­et bills that could climb to thou­sands of dol­lars month­ly. The Cat­a­lyst spokesper­son did not pro­vide com­ment on this as­ser­tion.

Im­age: Shut­ter­stock

Novartis is looking to milk one of its most prized possessions a little while longer without any generic competition, mostly because of a newly approved use that the Big Pharma won earlier this year for its megablockbuster heart drug Entresto.

In addition to battling (and winning) litigation last month to block a handful of Indian generics from entering the market early alongside Entresto, the company last week petitioned the FDA to try to stop a total of 18 Entresto generics from launching before 2024 at the earliest.