An in­censed Cat­a­lyst Phar­ma sues the FDA, ac­cus­ing agency of bow­ing to po­lit­i­cal pres­sure and break­ing fed­er­al law

Af­ter hint­ing it was ex­plor­ing the le­gal­i­ty of the FDA’s ap­proval of a ri­val drug from fam­i­ly-run com­pa­ny Ja­cobus Phar­ma­ceu­ti­cals, Cat­a­lyst Phar­ma­ceu­ti­cals on Wednes­day filed a law­suit against the health reg­u­la­tor — ef­fec­tive­ly ac­cus­ing the agency of bow­ing to po­lit­i­cal pres­sure sur­round­ing sky­rock­et­ing drug prices.

Patrick McE­namy Twit­ter

Be­fore Cat­a­lyst’s Fir­dapse (which car­ries an av­er­age an­nu­al list price of $375,000) was sanc­tioned for use in Lam­bert-Eaton myas­thenic syn­drome (LEMS) 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.

Then, in an un­ex­pect­ed twist, weeks ago the FDA en­dorsed New Jer­sey-based Ja­cobus’ ver­sion in pe­di­atric pa­tients, on the ba­sis of adult da­ta — a move that could spark off-la­bel pre­scrip­tion in adults (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). Cat­a­lysts’ shares $CPRX sank on the an­nounce­ment and have not since re­vived. Adding fu­el to the fire, ear­li­er this week Ja­cobus re­vealed its drug, Ruzur­gi, will car­ry a list price that is less than half of Fir­dapse’s.

Cat­a­lyst main­tains that typ­i­cal­ly, cov­ered pa­tients pay less than $10 per month out-of-pock­et.

In LEMS pa­tients, the body’s own im­mune sys­tem launch­es an at­tack on the neu­ro­mus­cu­lar junc­tion — which con­nects nerves and mus­cles. The con­di­tion is as­so­ci­at­ed with oth­er au­toim­mune dis­eases, but tends to oc­cur in pa­tients with can­cer. Glob­al­ly it is es­ti­mat­ed to af­fect three per mil­lion in­di­vid­u­als, ac­cord­ing to the FDA, but its pe­di­atric preva­lence is not clear. Cat­a­lyst sug­gest­ed there are 3,000 LEMS US pa­tients, of which 300 are on Fir­dapse. Some es­ti­mates sug­gest there are few­er than three dozen pe­di­atric pa­tients in the Unit­ed States.

Un­der fed­er­al law, the agency is meant to treat all com­pa­nies in the same man­ner. Cat­a­lyst has as­sert­ed the agency un­der­mined the com­pa­ny’s or­phan drug ex­clu­siv­i­ty, and vi­o­lat­ed fed­er­al law by play­ing fa­vorites in con­text of a hy­per­vig­i­lant pric­ing en­vi­ron­ment.

The ap­proval let­ters for Cat­a­lyst and Ja­cobus sug­gest that while the for­mer sub­mit­ted a body of clin­i­cal tri­al ev­i­dence as part of the pack­age the FDA re­viewed, the lat­ter did not sub­mit da­ta from cer­tain tox­i­col­o­gy stud­ies. In­stead, the FDA has asked Ja­cobus to pro­vide these da­ta as a post-mar­ket­ing re­quire­ment.

“We felt that the FDA’s ap­proval of Ruzur­gi was ar­bi­trary, capri­cious and not in ac­cord with the Food, Drug & Cos­met­ic Act,” Cat­a­lyst chief Patrick McE­nany told End­points News. 

“We be­lieve the FDA was im­prop­er­ly in­flu­enced in mak­ing this de­ci­sion by po­lit­i­cal pres­sures with re­gard to high drug pric­ing and we think it sets a hor­ri­ble prece­dent for oth­er com­pa­nies work­ing to de­vel­op drugs to treat rare dis­eases — it could in fact dis­cour­age com­pa­nies from in­vest­ing mil­lions of dol­lars and spend­ing many years to do this,” he said.

Ear­li­er this year, Ver­mont Sen­a­tor Bernie Sanders — ahead of his an­nounce­ment to make a sec­ond at­tempt at the pres­i­den­cy — spot­light­ed Cat­a­lyst for the “im­moral ex­ploita­tion of pa­tients.”

McE­nany em­pha­sized that Cat­a­lyst has spent more than $100 mil­lion dol­lars in de­vel­op­ing the drug over the last sev­en years — and that his hope is the law­suit will cul­mi­nate in the with­draw­al of the Ruzur­gi ap­proval.

An FDA spokesper­son said the agency does not com­ment on pend­ing lit­i­ga­tion. End­points News has con­tact­ed Ja­cobus for com­ment.

Im­age: An­drew Harnik, AP Im­ages

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls



Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Vi­a­tris with­draws ac­cel­er­at­ed ap­proval for top­i­cal an­timi­cro­bial 24 years lat­er

After 24 years without confirming clinical benefit, the FDA announced Tuesday morning that Viatris (formed via Mylan and Pfizer’s Upjohn) has decided to withdraw a topical antimicrobial agent, Sulfamylon (mafenide acetate), after the company said conducting a confirmatory study was not feasible.

Sulfamylon first won FDA’s accelerated nod in 1998 as a topical burn treatment, with the FDA noting that last December, Mylan told the agency that it wasn’t running the trial.

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

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Lynn Baxter, Viiv Healthcare's head of North America

Vi­iV dri­ves new cor­po­rate coali­tion in­clud­ing Uber, Tin­der and Wal­mart, aimed at end­ing HIV

ViiV Healthcare is pulling together an eclectic coalition of consumer businesses in a new White House-endorsed effort to end HIV by the end of the decade.

The new US Business Action to End HIV includes pharma and health companies — Gilead Sciences, CVS Health and Walgreens — but extends to a wide range of consumer companies that includes Tinder, Uber and Walmart.

ViiV is the catalyst for the group, plunking down more than half a million dollars in seed money and taking on ringmaster duties for launch today on World AIDS Day, but co-creator Health Action Alliance will organize joint activities going forward. ViiV and the alliance want and expect more companies to not only join the effort, but also pitch in funding.

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Roche HQ in Basel, Switzerland. (Image credit: Kyle LaHucik/Endpoints News)

As com­peti­tors near FDA goal­post, Roche spells out its re­peat Alzheimer's set­back

Before Roche can turn all eyes on a new version of its more-than-once-failed Alzheimer’s drug gantenerumab, the Big Pharma had to flesh out data on the November topline failure at an annual conference buzzier than in years past thanks to hotly watched rivals in the field: Eisai and Biogen’s lecanemab, and Eli Lilly’s donanemab.

There was less than a 10% difference between Roche’s drug and placebo at slowing cognitive decline across two Phase III trials, which combined enrolled nearly 2,000 Alzheimer’s patients. In its presentation at the conference Wednesday, Roche said it saw less sweeping away of toxic proteins than it had anticipated. For years, researchers and investors have put their resources behind the idea that more amyloid removal would equate to reduced cognitive decline.

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Ei­sai’s ex­pand­ed Alzheimer’s da­ta leave open ques­tions about safe­ty and clin­i­cal ben­e­fit

Researchers still have key questions about Eisai’s investigational Alzheimer’s drug lecanemab following the publication of more Phase III data in the New England Journal of Medicine Tuesday night.

In the paper, which was released in conjunction with presentations at an Alzheimer’s conference, trial investigators write that a definition of clinical meaningfulness “has not been established.” And the relative lack of new information, following topline data unveiled in September, left experts asking for more — setting up a potential showdown to precisely define how big a difference the drug makes in patients’ lives.

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Illustration: Assistant Editor Kathy Wong for Endpoints News

Twit­ter dis­ar­ray con­tin­ues as phar­ma ad­ver­tis­ers ex­tend paus­es and look around for op­tions, but keep tweet­ing

Pharma advertisers on Twitter are done — at least for now. Ad spending among the previous top spenders flattened even further last week, according to the latest data from ad tracker Pathmatics, amid ongoing turmoil after billionaire boss Elon Musk’s takeover now one month ago.

Among 18 top advertisers tracked for Endpoints News, only two are spending: GSK and Bayer. GSK spending for the full week through Sunday was minimal at just under $1,900. Meanwhile, German drugmaker Bayer remains the industry outlier upping its spending to $499,000 last week from $480,000 the previous week. Bayer’s spending also marks a big increase from a month ago and before the Musk takeover, when it spent $16,000 per week.

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Tim Van Hauwermeiren, argenx CEO

Ar­genx pur­chas­es $100M+ FDA pri­or­i­ty re­view vouch­er from blue­bird bio

Argenx’s Vyvgart is due for a speedy review at the FDA, thanks to a $102 million priority review voucher (PRV).

The Netherland-based biotech picked up the PRV from bluebird bio, the companies announced on Wednesday. PRVs shorten a drug’s FDA review period from 10 months to 6 months, though they often sell on the open market for around $100 million each.

Argenx plans on using the express ticket on efgartigimod, its neonatal Fc receptor (FcRn) blocker marketed as Vyvgart for adults with generalized myasthenia gravis (gMG). While Vyvgart won its first approval last December for the chronic neuromuscular disease — which is characterized by difficulties with facial expression, speech, swallowing and breathing — CEO Tim Van Hauwermeiren said in a news release that he plans to “be active in fifteen disease targets by 2025.”