In­cyte nabs 2nd ever ap­proval ahead of sched­ule as FDA in­sists it can still re­view on time

Al­most a full year af­ter In­cyte broke off a trou­bled part­ner­ship with Eli Lil­ly to fo­cus on its own in­ter­nal en­gine, one of the drugs from that en­gine will en­ter the mar­ket.

Pemi­ga­tinib, now brand­ed as Pe­mazyre, ob­tained FDA ap­proval as a sec­ond-line treat­ment for a rare form of can­cer known as cholan­gio­car­ci­no­ma, or bile duct can­cer. For In­cyte, a biotech that rose to promi­nence on the strength of a sin­gle drug — Jakafi — and a slew of Big Phar­ma part­ner­ships, the ap­proval marks the first of what they hope will be a se­ries of in­ter­nal suc­cess­es and the first sign of a new phase for the com­pa­ny, al­beit one that is un­like­ly to make a huge com­mer­cial splash.

The ap­proval came a month be­fore the May 30 PDU­FA date and on the same day that a Seat­tle Ge­net­ics drug was cleared four months ahead of its date. FDA of­fi­cials, hav­ing been hound­ed for over a month with ques­tions about how the Covid-19 cri­sis would af­fect the re­view process for non-Covid drugs, were clear about what the reg­u­la­to­ry an­nounce­ments should sig­nal.

“This ap­proval demon­strates that while we con­tin­ue to fo­cus our ef­forts on ad­dress­ing the COVID-19 pan­dem­ic, the FDA re­mains com­mit­ted to the im­por­tant work of re­view­ing treat­ments for pa­tients with can­cer and oth­er se­ri­ous con­di­tions,” Richard Paz­dur, CDER’s top on­col­o­gy of­fi­cial, said in a state­ment.

For all its con­fi­dent state­ments and now mul­ti­ple ear­ly ap­provals, though, the agency ac­knowl­edged last week that with staff in­creas­ing­ly fo­cused on re­view­ing Covid-19 work, “it is pos­si­ble that we will not be able to sus­tain our cur­rent lev­el of per­for­mance in­def­i­nite­ly.”

The In­cyte drug had re­ceived both break­through ther­a­py sta­tus and pri­or­i­ty re­view. Af­ter ES­MO last year, Eval­u­ate pegged peak sales for the drug at $127 mil­lion across in­di­ca­tions.

Pem­ga­tinib is an in­hibitor of FGFR, or fi­brob­last growth fac­tor, the same ki­nase tar­get­ed by J&J’s blad­der can­cer drug erdafi­tinib. In Phase II tri­al da­ta re­leased last year, In­cyte showed the drug led to a pro­gres­sion-free sur­vival of 6.9 months and over­all sur­vival of 21.1 months in pa­tients with an FGFR2 mu­ta­tion. Two oth­er small­er co­horts with dif­fer­ent forms of FGFR lived for 6.7 months and 4.0 months, in line with the dis­ease’s stan­dard prog­no­sis. The fi­nal du­ra­tion of re­sponse re­sult was 9.1 months.

In­cyte hopes to soon show ef­fec­tive­ness in blad­der can­cer, test­ing the drug in sev­er­al Phase II tri­als for that in­di­ca­tion, along with tri­als for pa­tients with any tu­mors that have FGFR mu­ta­tions, ir­re­spec­tive of type. Op­er­at­ing in re­verse, J&J is now test­ing their drug against bile duct can­cer, while In­cyte push­es to show ef­fi­ca­cy as a first-line treat­ment. Agios al­so has an ex­per­i­men­tal bile duct treat­ment that tar­gets the en­zyme IDH1.

If the FDA keeps up its rapid pace, In­cyte could soon see an­oth­er big can­cer ap­proval. No­var­tis’ cap­ma­tinib, which the Swiss gi­ant li­censed from In­cyte back in 2009, has al­so been grant­ed break­through sta­tus and pri­or­i­ty re­view for MET-14 breast can­cer, with a PDU­FA date in Au­gust. That deal pro­vid­ed for over $500 mil­lion in mile­stones if No­var­tis suc­cess­ful­ly de­vel­oped the drug, plus 12-14% of sales.

The ap­proval could help shore up in­vestors’ testy faith in In­cyte’s abil­i­ty to ex­pand be­yond Jakafi. The com­pa­ny’s stock has swung wild­ly over the past year, par­tic­u­lar­ly af­ter the with­draw­al from the strug­gling Lil­ly drug baric­i­tinib and, in Jan­u­ary, a large Phase III fail­ure for a graft-ver­sus-host-dis­ease drug. It was up 4.54% from Thurs­day’s mar­ket close, to a flat $100.

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.”