FDA OKs first cannabi­noid, gives green light to GW Phar­ma’s Epid­i­olex for rare seizures

GW Phar­ma $GW­PH fin­ished its layup at the FDA to­day with Epid­i­olex, grab­bing its first ever mar­ket­ing OK for one of its cannabi­noid drugs and set­ting up the biotech for a ma­jor shift to­ward com­mer­cial­iza­tion.

Justin Grover, GW Phar­ma CEO

Two months af­ter a unan­i­mous rec­om­men­da­tion by an out­side group of FDA ex­perts and some glow­ing rec­om­men­da­tions in the in­ter­nal re­view, GW will now set out to open up a new class of meds af­ter demon­strat­ing the drug’s abil­i­ty to pre­vent seizures in chil­dren suf­fer­ing from rare cas­es of Lennox-Gas­taut syn­drome and Dravet syn­drome. 

The ap­proval was record­ed on the FDA’s web­site and word spread quick­ly through Twit­ter.

Clar­i­vate has pegged 2022 sales at a block­buster sized $1.2 bil­lion, mak­ing this roll­out a huge­ly im­por­tant event for the UK-based com­pa­ny.

The FDA’s Scott Got­tlieb did the hon­ors in the state­ment to­day, cel­e­brat­ing GW for the work they did, and warn­ing any­one else against try­ing to mar­ket cannabis-re­lat­ed prod­ucts with du­bi­ous sup­port for their claims.

Scott Got­tlieb

“This ap­proval serves as a re­minder that ad­vanc­ing sound de­vel­op­ment pro­grams that prop­er­ly eval­u­ate ac­tive in­gre­di­ents con­tained in mar­i­jua­na can lead to im­por­tant med­ical ther­a­pies. And, the FDA is com­mit­ted to this kind of care­ful sci­en­tif­ic re­search and drug de­vel­op­ment,” said Got­tlieb. “Con­trolled clin­i­cal tri­als test­ing the safe­ty and ef­fi­ca­cy of a drug, along with care­ful re­view through the FDA’s drug ap­proval process, is the most ap­pro­pri­ate way to bring mar­i­jua­na-de­rived treat­ments to pa­tients. Be­cause of the ad­e­quate and well-con­trolled clin­i­cal stud­ies that sup­port­ed this ap­proval, pre­scribers can have con­fi­dence in the drug’s uni­form strength and con­sis­tent de­liv­ery that sup­port ap­pro­pri­ate dos­ing need­ed for treat­ing pa­tients with these com­plex and se­ri­ous epilep­sy syn­dromes. We’ll con­tin­ue to sup­port rig­or­ous sci­en­tif­ic re­search on the po­ten­tial med­ical us­es of mar­i­jua­na-de­rived prod­ucts and work with prod­uct de­vel­op­ers who are in­ter­est­ed in bring­ing pa­tients safe and ef­fec­tive, high qual­i­ty prod­ucts. But, at the same time, we are pre­pared to take ac­tion when we see the il­le­gal mar­ket­ing of CBD-con­tain­ing prod­ucts with se­ri­ous, un­proven med­ical claims. Mar­ket­ing un­ap­proved prod­ucts, with un­cer­tain dosages and for­mu­la­tions can keep pa­tients from ac­cess­ing ap­pro­pri­ate, rec­og­nized ther­a­pies to treat se­ri­ous and even fa­tal dis­eases.”

GW CEO Justin Gov­er told the Fi­nan­cial Times over the week­end that it had tak­en “an aw­ful long time to get to where we are . . . but we’ve re­al­ly built a huge lead. We’re the world-lead­ers in cannabi­noid sci­ence, and I think we ex­pect to stay there for quite some time.”

From GW’s per­spec­tive this is just the be­gin­ning, with plans to pur­sue more re­search work in a va­ri­ety of ail­ments.

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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A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

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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.

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.

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. 

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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.