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

Celyad’s view on developing and delivering a CAR T-cell therapy with multi-tumor specificity combined with cell manufacturing success
Overview
Transitioning potential therapeutic assets from academia into the commercial environment is an exercise that is largely underappreciated by stakeholders, except for drug developers themselves. The promise of preclinical or early clinical results drives enthusiasm, but the pragmatic delivery of a therapy outside of small, local testing is most often a major challenge for drug developers especially, including among other things, the manufacturing challenges that surround the production of just-in-time and personalized autologous cell therapy products.

The ads piqued interest as soon as they started appearing in 2016: at Grand Central Station, on the Red Line in Cambridge, and on a billboard off the New Jersey Turnpike. All showed a young person, generally with his or her arms crossed, and the question, “what is Vivitrol?”

Vivitrol’s maker, Alkermes, was in the midst of a marketing and lobbying campaign to promote the anti-opioid addiction drug — a campaign that would face significant backlash for tarnishing competitors despite little evidence for Vivitrol’s superiority.

The FDA in-house review highlights a disagreement of investigators’ use of a key endpoint by Horizon Pharma in the late-stage trial for the top drug in its pipeline, but largely agreed that the antibody was effective.

Horizon submitted a BLA for thyroid eye disease (TED) drug teprotumumab in March, less than two years after they bought the drug (and the rest of a division) from Narrow River for $145 million upfront. With breakthrough status, priority review, orphan designation and in-house sales projections of up to $750 million, the one-time Roche reject became the marquee pipeline asset for a company that’s developed some of the world’s most expensive drugs.

Seattle Genetics $SGEN is showing off more positive data around tucatinib, its pivotal-stage drug for HER2 positive breast cancer.

A month after hearing about solidly upbeat hazard ratios, we learned today that the estimated progression-free survival rate at one year was 33% in the tucatinib arm compared to 12% for patients taking trastuzumab and capecitabine alone.

Median PFS was 7.8 months (95% CI: 7.5, 9.6) in the tucatinib arm, compared to 5.6 months (95% CI: 4.2, 7.1) in the control arm.

It’s a case of some bad timing for Intec.

Just when a key trial testing the company’s Accordion drug delivery tech imploded in Parkinson’s disease, they handed Novartis data from a successful PK study of a custom Accordion pill engineered to deliver a Novartis compound to entice the Swiss drugmaker into signing a licensing agreement.

Novartis said thanks, but no thanks.

For the cash-strapped Israeli drug developer, the failure to clinch the deal marks a big blow. As of the third quarter, the company has $15.7 million in cash and equivalents, which HC Wainwright analysts estimate will keep the lights on into mid-2020.

When the FDA spurned Astellas’ pitch for atrial fibrillation drug vernakalant in 2008, regulators made it abundantly clear that it wasn’t the efficacy they had a problem with — two Phase III trials had shown the drug successfully restored 52% of patients’ heartbeat from irregular to normal — but the cardio safety issues for a drug that was to compete with well established, low-risk options. One licensing deal, one clinical hold and several studies later, the chances of approval aren’t looking any better.