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.

Janet Woodcock (AP Images)

Janet Wood­cock to be act­ing FDA com­mis­sion­er while Biden team fi­nal­izes nom­i­nee — re­ports

Janet Woodcock is set to be the most powerful person at the FDA in less than a week.

The veteran regulator and longtime director of the Center for Drug Evaluation and Research has been tapped as acting commissioner of the FDA, according to reports by BioCentury’s Steve Usdin and Pink Sheet’s Sarah Karlin-Smith.

The appointment was requested by the incoming Biden team, Karlin-Smith added, as they sort out the nomination of a permanent successor to Stephen Hahn — whose one-year tenure has been defined by Covid-19.

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Janet Woodcock (AP Images)

Janet Wood­cock is in the run­ning for FDA com­mis­sion­er — what does that mean for the agen­cy's fu­ture?

Just a day after reports emerged that Janet Woodcock will serve as interim chief of the FDA, word has gotten out that she is also in the running for the permanent job.

The decision, as the initial wave of reactions suggest, could have dramatic implications for where the agency is headed in the next four years — if not beyond.

Woodcock, the longtime CDER director, is being vetted alongside former FDA principal deputy commissioner Joshua Sharfstein, Bloomberg reported. Already tapped as acting head of the agency, she’s set to take over from Stephen Hahn right after Biden’s inauguration next week.

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Steve Harr (L) and Hans Bishop

Paint­ing by the num­bers, Sana founders carve up a gi­ant uni­corn-sized IPO — for a biotech that has­n't quite made it to the clin­ic

Sana Biotechnology is one of those startups that was sketched in on the chalkboard day one in the shape of a unicorn.

A giant unicorn.

And from the numbers the cell therapy 2.0 play spelled out in their S-1 $SANA, it’s clear that the company founders — led by a pair of major VCs aligned with some high-profile industry figures — are hunting a big chunk of that value for themselves.

The raise they penciled in — $150 million — isn’t likely what they actually have in mind, and it doesn’t do justice to the size of their ambitions.

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Eli Lil­ly re-ups di­ver­si­ty pledge, pitch­ing in $30M to ven­ture fund for mi­nor­i­ty-owned health­care firms

The fight against racial injustice spurred by a series of high-profile shootings of Black men by police earlier this year put Big Pharma and healthcare — industries targeted for their lack of diversity — in the hot seat. Eli Lilly made an early pledge to change its ways and put more back into the community, and now it’s continuing to make good on that commitment.

Lilly will infuse $30 million into the Unseen Capital Health Fund, a venture fund looking to invest in early-stage minority-owned healthcare companies that have been historically “unseen” by the investment community, the pharma said Friday.

CEO Brett Monia (Ionis)

Can Brett Mo­nia push Io­n­is be­yond Spin­raza?

For 30 years, Brett Monia struggled as one of Ionis’ top scientists to get their antisense technology to work. Now, as CEO, he’s trying to use it to turn Ionis into one of the industry’s biggest biotechs.

Monia, one of the handful of young scientists who in 1989 followed Stanley Crooke across the country from SmithKline (now GSK) in Philadelphia to found Ionis in Northern California, replaced Crooke as CEO last January. By then, they had proven antisense, an RNA-based method for manipulating gene expression, could work dramatically well in at least some instances, transforming spinal muscular atrophy with the Biogen-partnered blockbuster Spinraza.

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David Kessler in April 2009 (Eric Risberg/AP Images)

Covid-19 roundup: Hack­ers start re­leas­ing 'ma­nip­u­lat­ed' Covid-19 vac­cine docs; Ex-FDA com­mish David Kessler to re­place Mon­cef Slaoui as Op­er­a­tion Warp Speed chief — re­port

There’s a new twist on the EMA Covid-19 hacking story.

Friday the European agency put out the 5th in a series of statements about the hackers who broke into their system, noting that some of the information on vaccines that was gleaned in the attack is showing up online — altered to raise questions about the Covid-19 vaccines now in use.

This included internal/confidential email correspondence dating from November, relating to evaluation processes for COVID-19 vaccines. Some of the correspondence has been manipulated by the perpetrators prior to publication in a way which could undermine trust in vaccines.

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UP­DAT­ED: Am­gen tops cost watch­dog's price gougers list based on 'un­sup­port­ed' in­creas­es for En­brel with­out mean­ing­ful da­ta

In a top 10 ranking of the most egregious price gougers from 2019, Amgen’s Enbrel topped US cost watchdog ICER’s naughty list with “unsupported” markups that added as much as $403 million to the nation’s drug spend during that time.

Price increases for some of pharma’s most popular drugs have long been a focus of consumer ire, but the industry has argued those increases are routine and meant to cover the cost of R&D innovation. Without meaningful guidance at the state or federal level, ICER looked to connect how much a drug had progressed in the clinic compared with its increase in both wholesale and net price in 2019.

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Albert Bourla, Pfizer CEO (AP Images)

#JPM21: Al­bert Bourla pre­pares Pfiz­er to set­tle in to next phase post-Up­john, but that does­n't mean he's rul­ing out deals

One of the iconic brands in biopharma, Pfizer took a big gamble on the strength of its in-house science when it decided to offload its flagging Upjohn generics business last year. Now, a more agile Pfizer is looking to cement its identity for the future, but one thing will stay the same: M&A is still very much a part of the game plan.

With its Upjohn generics business off the books, Pfizer is looking to double down on its branded medicines with the goal of hitting 6% annual growth each year — previously unheard-of at old Pfizer — while continuing to develop its blockbuster pipeline, CEO Albert Bourla said at a JP Morgan fireside chat Tuesday.

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Stefan Oelrich (Bayer)

What can a 157-year-old phar­ma gi­ant bring to the ta­ble of cell and gene ther­a­pies? Quite a bit, Bay­er says

By now, Bayer has sketched out in bold strokes some grand plans for cell and gene therapy, cemented by big-dollar acquisitions of platform companies.

But just how do you stitch together a new unit bursting with the newest ideas within a storied pharma?

Stefan Oelrich, the head of Bayer’s pharma division, briefly lifted the curtain and spotlighted three key factors as he took the stage on a virtual media day, flanked by Emile Nuwaysir and Sheila Mikhail, the chiefs of BlueRock and AskBio, who each introduced their work in a way you’d expect from a biotech CEO.