FDA gives Mer­ck ‘re­al-time’ OK for Keytru­da, of­fer­ing a peek at the lat­est ex­ten­sion of the agen­cy's da­ta ex­press high­way

The FDA doesn’t al­ways wait for a mar­ket­ing ap­pli­ca­tion to come in be­fore it be­gins its re­view these days. And that suits Mer­ck just fine.

The fed­er­al agency has stamped a full ap­proval — fol­low­ing last year’s con­di­tion­al OK — on Mer­ck’s mega-block­buster Keytru­da for front­line use in a chemo com­bo for fight­ing non-squa­mous non-small cell lung can­cer. Reg­u­la­tors based their de­ci­sion on the da­ta set from KEYNOTE-189, put on dis­play last April, one in a se­ries of late-stage tri­als that Mer­ck has been rolling out to grab the lead on the PD-1/L1 mar­ket from a group of tough com­peti­tors at Bris­tol-My­ers Squibb.

The green light is any­thing but un­ex­pect­ed. But it is note­wor­thy for rea­sons that the en­tire in­dus­try should be pay­ing at­ten­tion to.

Roger Perl­mut­ter

What dis­tin­guish­es this par­tic­u­lar OK is the tim­ing. FDA com­mis­sion­er Scott Got­tlieb has been push­ing a new pi­lot pro­gram called re­al-time on­col­o­gy re­views, where reg­u­la­tors start their work ahead of a for­mal ap­pli­ca­tion. The first re­al-time OK went to No­var­tis a few weeks ago for two com­bi­na­tions us­ing Kisqali against breast can­cer. And top-tier drug de­vel­op­ers are find­ing that in­stead of just be­ing greet­ed with an open door, some­times the door is be­ing tak­en off the hinges en­tire­ly.

In this case they were deal­ing with a haz­ard ra­tio of 0.49, mark­ing a 51% drop in the risk of death for pa­tients tak­ing the com­bo. There’s still no me­di­an sur­vival da­ta for the com­bo avail­able, but the matchup of the Keytru­da/chemo ap­proach ver­sus chemo alone pro­vid­ed a clear set of ad­van­tages for Mer­ck.

  • The me­di­an pro­gres­sion-free sur­vival rate was 8.8 months for the com­bo, 4.9 months for chemo alone.
  • The over­all re­sponse rate was 48% com­pared to 19% in the con­trol.
  • Me­di­an re­sponse du­ra­tion hit 11.2 months for the Keytru­da arm and 7.8 months for con­trol.

These re­al-time re­views now on of­fer at the FDA look to ex­tend the da­ta ex­press high­way that’s been built for the agency’s break­through drug des­ig­na­tion and oth­er hur­ry-up cam­paigns that have trans­formed the speed and de­sign of on­col­o­gy stud­ies over the past few years. In this case the FDA wants to re­ly en­tire­ly on US stud­ies, ex­clud­ing any­thing out­side the bor­ders, while pre­fer­ring drugs that are de­liv­er­ing clear ben­e­fits and are be­ing test­ed on some ob­vi­ous end­points. And they don’t want any for­mu­la­tion changes to pon­der when mak­ing a snap call.

The FDA is tak­ing no chances with Keytru­da, a land­mark drug by any de­f­i­n­i­tion that is be­ing pushed through hun­dreds of com­bi­na­tion stud­ies and new ap­proach­es. Got­tlieb is like­ly to earn even more de­vo­tion from the in­dus­try’s lead­ers for fol­low­ing through on im­prov­ing the FDA’s re­spon­sive­ness. And Mer­ck is hap­py to have the fresh set of brag­ging rights to­day.

“Keytru­da is rapid­ly be­com­ing a foun­da­tion for the treat­ment of ap­pro­pri­ate pa­tients with metasta­t­ic non-small cell lung can­cer,” said Mer­ck R&D chief Roger Perl­mut­ter. “To­day’s ap­proval of the ex­pand­ed la­bel for Keytru­da based on da­ta from the KEYNOTE-189 tri­al is an im­por­tant mile­stone.”

 

UP­DAT­ED: Roche bags 'break­through' an­ti-fi­bro­sis drug in $1.4B biotech buy­out deal

Roche is snapping up a “breakthrough” anti-fibrotic drug in a $1.4 billion buyout.

The pharma giant announced Friday that it is acquiring Promedior, primarily to get its hands on PRM-151, a recombinant form of human pentraxin-2 (PTX-2) protein that has nailed down mid-stage clinical data on idiopathic pulmonary fibrosis and demonstrating its potential for a range of fibrotic conditions.

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Amarin emerges from an ex­pert pan­el re­view with a clear en­dorse­ment for Vas­cepa and high odds of suc­cess when the FDA weighs in for­mal­ly

Several FDA experts who gathered Thursday to consider the landmark approval of Vascepa to reduce cardio events in an at-risk population voiced their unease about various aspects of the efficacy and safety data, or ultimately the population it should be used to treat. But the overwhelming belief that the data pointed to the drug’s benefit and clearly outweighed risks carried the day for Amarin.

The panel voted unanimously (16 to 0) to support the company’s positive data presentation — backing an OK for expanding the label to include reducing cardio risk. The vote points Amarin $AMRN down a short path to a formal decision by the FDA, with the odds heavily in its favor. Chances are the rest of the questions about the future of this drug will be hashed out in the label’s small print.

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Federal Trade Commission commissioner Rohit Chopra testifies on Capitol Hill (AP Photo/Susan Walsh)

FTC clears Bris­tol-My­ers’ $74B deal to buy Cel­gene — but Dems sig­nal a po­ten­tial hard shift against Big Phar­ma M&A

Bristol-Myers Squibb’s record $74 billion takeover of Celgene is a done deal. And it will all be over — except for the lingering complaints from die-hard Celgene investors — on Wednesday.

Like much else that’s going on in Washington these days, the vote among the 5 FTC commissioners split along party lines, with the 3 Republicans voting to clear the way and the 2 Democrats steamed over what they see as a major M&A move that will lessen competition and innovation. And that split has big implications for the M&A side of the business if the Dems take the White House in 2020.

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No­var­tis spin­out’s first an­ti-ag­ing PhI­II is a flop, so now they’ll turn to Parkin­son’s chal­lenge as shares wilt

Novartis spinout resTORbio is grappling with the collapse of its lead clinical program this morning — an anti-aging R&D failure that will badly damage their rep in the field.

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BeiGene CEO John Oyler at an Endpoints event in Shanghai, October 2018 (Credit: Endpoints News/PharmCube)

UP­DAT­ED: In a first, FDA green-lights use of a Chi­nese built can­cer ther­a­py — and more are com­ing

Weeks after Amgen took a $2.7 billion stake in BeiGene, the Beijing-based biotech has secured its first-ever FDA approval for zanubrutinib, a BTK inhibitor, months ahead of schedule.

BeiGene’s drug, branded as Brukinsa, has secured accelerated approval for adult patients with mantle cell lymphoma (MCL) — a typically aggressive, rare, form of blood cancer — who have received at least one prior therapy.

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What does $62B buy you these days? A lot, says Take­da ex­ecs as the phar­ma play­er promis­es a block­buster R&D fu­ture

First comes the $62 billion buyout. Then comes the asset auction and reorganization to pay down debt. Now comes the detailed pledge of a bigger, brighter future in drug development.

That’s where Takeda finds itself on R&D day today, about 11 months after closing on their Shire acquisition. R&D chief Andy Plump is joining CEO Christophe Weber and other top members of the team to outline a new set of priorities in the greatly expanded pipeline at Takeda, which has jumped into the top ranks of the world’s pharma giants in the wake of the Shire deal.

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GSK's asth­ma bi­o­log­ic Nu­cala scores in rare blood dis­or­der study

GlaxoSmithKline’s asthma drug Nucala, which received a resounding FDA rejection for use in chronic obstructive pulmonary disease (COPD) last year, has shown promise in a rare blood disorder.

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Mer­ck buys a fledg­ling neu­rode­gen­er­a­tive biotech spawned by an old GSK dis­cov­ery al­liance. What’s up with that?

Avalon Ventures chief Jay Lichter has a well-known yen for drug development programs picked up in academia. And what he found in Haoxing Xu’s lab at the University of Michigan pricked his interest enough to launch one of his umbrella biotechs in San Diego.

Xu’s work laid the foundation for Avalon to launch Calporta, which has been working on finding small molecule agonists of TRPML1 (transient receptor potential cation channel, mucolipin subfamily, member 1) for lysosomal storage disorders. And that pathway, they believe, points to new approaches on major market neurodegenerative diseases like Parkinson’s, ALS and Alzheimer’s.

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No­var­tis scores its lat­est FDA OK — this time for a new sick­le cell dis­ease drug picked up in a $665M deal

Novartis’ decision to buy Oklahoma-based biotech Selexys 3 years ago for up to $665 million has paid off with an FDA approval today.

Blessed with the FDA’s breakthrough drug designation for a speedy review, the pharma giant has pinned down an approval for crizanlizumab, a new therapy designed to reduce the frequency of painful incidents of vaso-occlusive crises among sickle cell disease patients 16 or older.

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