In a stun­ning set­back, FDA spurns Eli Lil­ly’s mar­ket­ing ap­pli­ca­tion for baric­i­tinib, de­mands more da­ta


The FDA has re­ject­ed Eli Lil­ly’s block­buster con­tender baric­i­tinib, say­ing that the phar­ma gi­ant will need to gath­er sig­nif­i­cant­ly more da­ta be­fore reg­u­la­tors will re­con­sid­er their de­ci­sion.

Ac­cord­ing to a state­ment from Lil­ly, the phar­ma gi­ant will not on­ly need to pro­vide more clin­i­cal da­ta on the ap­pro­pri­ate dos­es to be used for rheuma­toid arthri­tis, reg­u­la­tors are al­so de­mand­ing more safe­ty da­ta, which could sig­nif­i­cant­ly de­lay any re­sponse to the CRL. Lil­ly says it dis­agreed with the FDA’s con­clu­sion and the re­jec­tion.

The kick­back marks a stun­ning set­back for Eli Lil­ly $LLY CEO Dave Ricks, who owes in­vestors a string of promised ap­provals fol­low­ing a long drought in the clin­ic. The phar­ma gi­ant is known for its care­ful, if slow, ap­proach to late-stage de­vel­op­ment. Baric­i­tinib has been tagged as one of the top block­busters in late-stage de­vel­op­ment and an im­por­tant new drug for Lil­ly.

Hervé Hop­penot, In­cyte CEO

The re­jec­tion is al­so a set­back for its biotech part­ner, In­cyte $IN­CY, which li­censed out the drug to Lil­ly 8 years ago for $90 mil­lion up­front. An­a­lysts have pegged this as a po­ten­tial block­buster ca­pa­ble of earn­ing up to $2 bil­lion in peak an­nu­al sales, but that’s an­oth­er num­ber that is like­ly to get a se­ri­ous re­view.

Ever­cor­eISI’s Umer Raf­fat be­lieves this could in­di­cate that the FDA may ul­ti­mate­ly ap­prove a low dose of the drug, which could be­dev­il the de­vel­op­ers’ plans. He not­ed:

We’re wait­ing to hear back from com­pa­ny … but my sum­ma­ry thoughts are along the lines of:  there is a clear de­lay here, but I think they should get ap­proved ul­ti­mate­ly (keep in mind baric­i­tinib ap­proved in Eu­rope now).  How­ev­er, if FDA is re­al­ly fo­cused so much on “ap­pro­pri­ate dose”, I won­der if 2 mg is ul­ti­mate­ly the best dose … and if that’s the case, then it’s not so clear that 2 mg is su­pe­ri­or to Hu­mi­ra (re­call the head to head su­pe­ri­or­i­ty of baric­it­nib vs Hu­mi­ra was us­ing 4 mg dose … and plen­ty of da­ta from oth­er tri­als sug­gest­ing 2 mg has low­er ra­di­ograph­ic pro­gres­sion than 4 mg).

Lil­ly chose to re­lease the news on the morn­ing of Good Fri­day, a bank hol­i­day, de­lay­ing the beat­ing these two com­pa­nies faced. In­vestors, though, weren’t in a bet­ter mood Mon­day morn­ing. Lil­ly’s shares dropped 5.6% while In­cyte’s shares slid 13%.

Lil­ly’s John Lech­leit­er – now chair­man of the board – ini­tial­ly promised in­vestors at least two new drug ap­provals per year be­gin­ning in 2013, but had to de­lay on de­liv­er­ing un­til 2014. The com­pa­ny has al­so had three straight late-stage fail­ures for solanezum­ab in Alzheimer’s dis­ease. But new drugs have been kick­ing in, help­ing Lil­ly to start to grow its num­bers af­ter be­ing pum­meled by gener­ics for years.

I queried a com­pa­ny spokesper­son on whether Lil­ly now ex­pects to keep its promise on sev­er­al new drug ap­provals in 2017. She fol­lowed up by point­ing out that Lil­ly most re­cent­ly promised 20 new ap­provals be­tween 2014 and 2023, and that’s ev­i­dent­ly not re­strict­ed to FDA OKs. Baric­i­tinib al­so gets to count, as it was ap­proved in Eu­rope — as Olu­mi­ant — where it will be sub­ject­ed to a sin­gle-pay­er re­view and sig­nif­i­cant­ly low­er re­im­burse­ments.

That log­ic isn’t like­ly to help with dis­ap­point­ed an­a­lysts.

The re­jec­tion pro­vides Re­gen­eron and its part­ners at Sanofi a chance to over­come its re­cent re­jec­tion on sar­ilum­ab — for man­u­fac­tur­ing rea­sons — and push ahead with a ri­val ther­a­py that many an­a­lysts be­lieve is the odds-on fa­vorite. It al­so marks an­oth­er ad­van­tage for Ab­b­Vie, which has been cir­cling the wag­ons around Hu­mi­ra as it mounts a de­fense against new ther­a­pies bit­ing off pieces of its multi­bil­lion dol­lar fran­chise ther­a­py.

A new re­port from ICER took is­sue with the price for Hu­mi­ra, which it post­ed as a net of $40,415 a year. And ICER pro­vid­ed a thumbs up for baric­i­tinib and sar­ilum­ab. ICER clear­ly prefers sar­ilum­ab on ef­fi­ca­cy — though we don’t know the price — giv­ing on­ly a mar­gin­al stamp of ap­proval to baric­i­tinib.


“We are dis­ap­point­ed with this ac­tion. We re­main con­fi­dent in the ben­e­fit/risk of baric­i­tinib as a new treat­ment op­tion for adults with mod­er­ate-to-se­vere RA,” said Christi Shaw, pres­i­dent of Lil­ly Bio-Med­i­cines. “We will con­tin­ue to work with the FDA to de­ter­mine a path for­ward and ul­ti­mate­ly bring baric­i­tinib to pa­tients in the U.S.”

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Gold for adults, sil­ver for in­fants: Pfiz­er's Pre­vnar 2.0 head­ed to FDA months af­ter Mer­ck­'s green light

Pfizer was first to the finish line for the next-gen pneumococcal vaccine in adults, but Merck beat its rival with a jab for children in June.

Now, two months after Merck’s 15-valent Vaxneuvance won the FDA stamp of approval for kids, Pfizer is out with some late-stage data on its 20-valent shot for infants.

Known as Prevnar 20 for adults, Pfizer’s 20vPnC will head to the FDA by the end of this year for an approval request in infants, the Big Pharma said Friday morning. Discussions with the FDA will occur first and more late-stage pediatric trials are expected to read out soon, informing the regulatory pathway in other countries and regions.

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His­toric drug pric­ing re­forms pass; Pfiz­er ac­quires GBT; The long search for non-opi­oid pain drugs; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

The Endpoints Weekly has officially crossed the 60,000 mark on subscribers — thanks to all of your support. As the editorial team grows, we’ve been able to do a lot more, with many of those on display this week. Be sure to check out Lei Lei Wu’s deep dive on pain R&D. If you missed it, you may also rewatch her companion panel here.

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No­var­tis re­ports two pa­tient deaths af­ter treat­ment with Zol­gens­ma

Two children with spinal muscular atrophy have died after receiving Novartis’ Zolgensma, a gene therapy designed as a one-time treatment for the rare fatal disease.

The deaths, which resulted from acute liver failure, occurred in Russia and Kazakhstan, Novartis confirmed in a statement to Endpoints News. Having notified health authorities across all the markets where Zolgensma is available, it will update the drug label “to specify that fatal acute liver failure has been reported,” a spokesperson wrote.

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Senate Finance Committee Chair Ron Wyden (D-OR) (Francis Chung/E&E News/POLITICO via AP Images)

Sen­ate Fi­nance chair con­tin­ues his in­ves­ti­ga­tion in­to phar­ma tax­es with re­quests for Am­gen

Amgen is the latest pharma company to appear on the radar of Senate Finance Committee Chair Ron Wyden (D-OR), who is investigating the way pharma companies are using subsidiaries in low- or zero-tax countries to lower their tax bills.

Like its peers Merck, AbbVie and Bristol Myers Squibb, Wyden notes how Amgen uses its Puerto Rico operations to consistently pay tax rates that are substantially lower than the U.S. corporate tax rate of 21%, with an effective tax rate of 10.7% in 2020 and 12.1% in 2021.

FDA ap­proves sec­ond in­di­ca­tion for As­traZeneca and Dai­ichi's En­her­tu in less than a week

AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Enhertu scored its second approval in less than a week, this time for a subset of lung cancer patients.

Enhertu received accelerated approval on Thursday to treat adults with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have already received a prior systemic therapy.

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J&J to re­move talc prod­ucts from shelves world­wide, re­plac­ing with corn­starch-based port­fo­lio

After controversially spinning out its talc liabilities and filing for bankruptcy in an attempt to settle 38,000 lawsuits, Johnson & Johnson is now changing up the formula for its baby powder products.

J&J is beginning the transition to an all cornstarch-based baby powder portfolio, the pharma giant announced on Thursday — just months after a federal judge ruled in favor of its “Texas two-step” bankruptcy to settle allegations that its talc products contained asbestos and caused cancer. An appeals court has since agreed to revisit that case.

CSL is gathering its four business units under a unified brand identity strategy (Credit: CSL company site)

CSL brings Se­qirus, Vi­for un­der par­ent um­brel­la brand in iden­ti­ty re­vamp

CSL is gathering its brands under the family name umbrella, renaming its vaccine and newly acquired nephrology specialty businesses with the parent initials.

CSL Seqirus and CSL Vifor join CSL Plasma and CSL Behring as the four now uniformly branded business units of the global biopharma. The Seqirus vaccine division was formed in 2015 with the combination of bioCSL and its purchase of Novartis’ flu vaccine business. CSL picked up Vifor Pharma late last year in an $11.7 billion deal for the nephrology, iron deficiency and cardio-renal drug developer.

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Sanofi, GSK, Ha­le­on see stock prices dip and dive amid lit­i­ga­tion for re­called heart­burn drug

Zantac became one of the most well-known drugs on the market after being FDA-approved in 1983 — and now close to four decades later, lawsuits over safety concerns are rattling analysts and investors.

Sanofi, GSK and Haleon, GSK’s former consumer healthcare unit, have lost billions of dollars in market cap since Tuesday’s market close, according to Bloomberg. While Zantac is no longer on the market, the drop came after a suite of analysts, from Morgan Stanley and other firms, sounded the alarm on the potential impact of ongoing personal injury litigation.

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