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.”


Aerial view of Genentech's campus in South San Francisco [Credit: Getty]

Genen­tech sub­mits a big plan to ex­pand its South San Fran­cis­co foot­print

The sign is still there, a quaint reminder of whitewashed concrete not 5 miles from Genentech’s sprawling, chrome-and-glass campus: South Francisco The Industrial City. 

The city keeps the old sign, first erected in 1923, as a tourist site and a kind of civic memento to the days it packed meat, milled lumber and burned enough steel to earn the moniker “Smokestack of the Peninsula.” But the real indication of where you are and how much has changed both in San Francisco and in the global economy since a couple researchers and investors rented out an empty warehouse 40 years ago comes in a far smaller blue sign, resembling a Rotary Club post, off the highway: South San Francisco, The Birthplace of Biotech.

Here comes the oral GLP-1 drug for di­a­betes — but No­vo Nordisk is­n't dis­clos­ing Ry­bel­sus price just yet

Novo Nordisk’s priority review voucher on oral semaglutide has paid off. The FDA approval for the GLP-1 drug hit late Friday morning, around six months after the NDA filing.

Rybelsus will be the first GLP-1 pill to enter the type 2 diabetes market — a compelling offering that analysts have pegged as a blockbuster drug with sales estimates ranging from $2 billion to $5 billion.

Ozempic, the once-weekly injectable formulation of semaglutide, brought in around $552 million (DKK 3.75 billion) in the first half of 2019.

As Nas­daq en­rolls the fi­nal batch of 2019 IPOs, how have the num­bers com­pared to past years?

IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

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Oxitec biologist releases genetically modified mosquitoes in Piracicaba, Brazil in 2016 [credit: Getty Images]

In­trex­on unit push­es back against claims its GM mos­qui­toes are mak­ing dis­ease-friend­ly mu­tants

When the hysteria of Zika transmission sprang into the American zeitgeist a few years ago, UK-based Oxitec was already field-testing its male Aedes aegypti mosquito, crafted to possess a gene engineered to obliterate its progeny long before maturation.

But when a group of independent scientists evaluated the impact of the release of these genetically-modified mosquitoes in a trial conducted by Oxitec in Brazil between 2013 and 2015, they found that some of the offspring had managed to survive — prompting them to speculate what impact the survivors could have on disease transmission and/or insecticide resistance.

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Pur­due threat­ens to walk away from set­tle­ment, asks to pay em­ploy­ees mil­lions in bonus­es

There are two updates on the lawsuit against Purdue Pharma over its role in fueling the opioid epidemic, as the Sackler family threatens to walk away from their pledge to pay out $3 billion if a bankruptcy judge does not stop outstanding state lawsuits against them. At the same time, the company has asked permission to pay millions in bonuses to select employees.

Purdue filed for chapter 11 bankruptcy this week as part of its signed resolution to over 2,000 lawsuits. The deal would see the Sackler family that owns Purdue give $3 billion from their personal wealth and the company turned into a trust committed to curbing and reversing overdoses.

While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

Jason Kelly. Mike Blake/Reuters via Adobe

Eye­ing big ther­a­peu­tic push, Gink­go bags $290M to build a cell pro­gram­ming em­pire

Ginkgo Bioworks is on a roll. Days after publicizing a plan to nurture new startups via partnerships with accelerators Y Combinator and Petri, the Boston biotech says it has raised another $290 million for its cell programming platform to reach further and wider.

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UP­DAT­ED: Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.

David Grainger [file photo]

'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

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