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.”

 

Andre Kalil, AP Images

A 9/11-era Om­a­ha fa­cil­i­ty, an old Ebo­la drug, and the ubiq­ui­tous Dr. Fau­ci: In­side the first US nov­el coro­n­avirus tri­al

The first 11 coronavirus patients who arrived in Omaha last week, airlifted across the globe after two weeks quarantined on a cruise ship, showed only minor symptoms or none at all. And then one of them — or one of the couple of Americans who arrived later — got worse. He developed pneumonia, a life-threatening complication for coronavirus patients.

In a biocontainment room at the University of Nebraska Medical Center on Friday, doctors infused him with an experimental Gilead drug once developed for Ebola, called remdesivir. Or they gave him a placebo. For the first time in the US, neither he nor the doctors knew.

The first US novel coronavirus trial was underway and with it, a mad dash for an answer. Sponsored by the NIH, the study marked a critical point in the epidemic. Since the start of the outbreak, the agency had helped lead a global effort to contain the virus. Now, as it spread worldwide and the CDC issued warnings the US could see a major internal outbreak, they were looking at home.

“We don’t have too much time,” Andre Kalil, the trial’s lead investigator, told Endpoints News. “Everything’s moving really fast.”

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Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Olivier Brandicourt (AP Images)

Ex-Sanofi chief Olivi­er Brandi­court, cur­rent Black­stone ad­vi­sor, jumps on Al­ny­lam board

Former Sanofi chief Olivier Brandicourt, who departed his post with an unexpected early retirement last year, has made his move — as most C-suite executives inevitably do — to become a director on the board of a biopharma company.

RNAi player Alnylam is Brandicourt’s destination. Meanwhile, the Cambridge, Massachusetts-based drugmaker — which pioneered the first approval in the field — also disclosed the retirement of Alnylam co-founder Dr. Paul Schimmel from its board.

Jim Wilson's gene ther­a­py start­up Pas­sage Bio bucks mar­ket sen­ti­ments, rais­ing up­sized $216M IPO

A coronavirus fear-induced bloodbath on the Nasdaq has not stopped Passage Bio from making a public debut — and an exuberant one.

By pricing an upsized offering at $18, the top of the range, the gene therapy biotech bagged $216 million from its IPO, 72% more than it’s originally penciled in.

The proceeds likely reflected confidence in Jim Wilson, who gathered all the tools he’s built over decades of gene therapy research to assemble the startup and teamed up with Frazier and OrbiMed to hone its focus on rare, monogenic disorders of the central nervous system. Just before the IPO, Deerfield partner Bruce Goldsmith took over from OrbiMed’s Stephen Squinto as CEO.

Dan O'Day (AP Images)

UP­DAT­ED: A name emerges out of the Gilead M&A ru­mor mill, and it’s a can­cer biotech

After months of questions and speculation about when and if Gilead will make a major acquisition, a name has emerged.

The California-based drugmaker has approached Forty Seven Inc, a cancer biotech, with a takeover offer, Bloomberg News reports. With Forty Seven’s market cap at $2.3 billion, an acquisition would likely be Gilead’s largest since they acquired Kite Pharma for $11.9 billion in 2017.

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Biogen head of R&D Al Sandrock, Sangamo CEO Sandy Macrae

UP­DAT­ED: Bio­gen makes an­oth­er bold Alzheimer’s bet, drop­ping $350M up­front to part­ner with genome-edit­ing fo­cused Sang­amo

While the fate of Biogen’s resurrected Alzheimer’s drug aducanumab remains uncertain, the Cambridge, MA-based drugmaker is joining forces with genome editing company Sangamo Therapeutics to develop therapies for neurological conditions.

Sangamo is set to receive a meaty $350 million upfront in cash and stock and is eligible to receive up to $2.37 billion in milestone payments, in addition to royalties. In return, Biogen gets the rights to two Sangamo preclinical compounds: ST-501 (for use in tauopathies including Alzheimer’s disease) and ST-502 (for synucleinopathies including Parkinson’s disease).

“The partnership represents a lower-cost way to expand its work in neurologic disease,” Credit Suisse’s Evan Seigerman said in a note, referring to Biogen.

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Take­da swoops in to buy lit­tle biotech part­ner and its celi­ac drug poised to 'change stan­dard of care'

Having spent three years carefully grooming PvP Biologics and its drug for celiac disease, Takeda is happy enough with the proof-of-concept data to buy it all.

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Spark los­es an­oth­er top ex­ec in the wake of $4.3B takeover by Roche — re­port

Days after bidding farewell to co-founder Kathy High, Spark Therapeutics — now operating under Roche — has one more opening on its C-suite.

Kathy Reape

Kathy Reape, who joined the Philadelphia-based biotech in 2016 as head of clinical R&D and became chief medical officer in 2018, is reportedly set to leave.

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'The head­lines are the head­lines, but': Bio­Marin talks up po­ten­tial sav­ings as he­mo­phil­ia gene ther­a­py launch looms

BioMarin execs are still staying tight-lipped about their pricing plans for what is poised to be the world’s first hemophilia gene therapy. But as the company enters the final regulatory stretch and approaches a potential launch this summer, they are also dropping more hints to get investors ready.

First thing to know: They really, really don’t expect an advisory committee to be convened for valrox, which is under priority review, to pop up before its PDUFA date on August 21.

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