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.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

FDA chief Stephen Hahn on Capitol Hill earlier this week (Getty Images)

As FDA buck­les un­der the strain of a pan­dem­ic work­load, Trump again ac­cus­es the agency of a po­lit­i­cal hit job

Peter Marks appeared before a virtual SVB Leerink audience yesterday and said that his staff at FDA’s CBER is on the verge of working around the clock. Manufacturing inspections, policy work and sponsor communications have all been pushed down the to-do list so that they can be responsive to Covid-related interactions. And the agency’s objective right now? “To save as many lives as we can,” Marks said, likening the mortality on the current outbreak as equivalent to “a nuclear bomb on a small city.”

Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

Play-by-play of Gilead­'s $21B Im­munomedics buy­out de­tails a fren­zied push — and mints a new biotech bil­lion­aire

Immunomedics had not really been looking for a buyout when the year began. Excited by its BLA for Trodelvy, submitted to the FDA in late 2019, executive chairman Behzad Aghazadeh started off looking for potential licensing deals and zeroed in on four potential partners, including Gilead, following January’s JP Morgan Healthcare Conference in San Francisco. Such talks advanced throughout the year, with discussions advancing to the second round in mid-August.

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President Donald Trump reacts after signing an executive order following his remarks on his healthcare policies yesterday in Charlotte, North Carolina (Getty Images)

Op-ed: Will phar­ma re­al­ly pay for Trump’s lat­est law­less promise to 33 mil­lion Medicare ben­e­fi­cia­ries? Not like­ly

Sitting atop the executive branch, President Donald Trump is the ultimate authority at the FDA. He can fast track any vaccine to approval himself. If it came to that, of course.

What he can’t do is unilaterally order the legislative branch to loosen the Treasury’s coffers for $6.6 billion. Nor can he command pharmaceutical companies to pay for $200 vouchers sent to 33 million Medicare beneficiaries for prescription drugs before the election.

President Donald Trump and FDA Commissioner Stephen Hahn (AP Images)

FDA is­sues fi­nal rule al­low­ing im­por­ta­tion of drugs from Cana­da — but al­so keeps the pow­er to re­voke it

Just over a month away from the presidential election, the FDA has issued a final regulation fulfilling President Trump’s promise to let states import certain prescription drugs from Canada.

On Thursday, Trump told a crowd in North Carolina that the new rule goes into effect “today.” But the published regulation states that it won’t take effect for 60 days. And even then, it could be a while before cheaper drugs make it across the border.

The win­dow is wide open as four more biotechs join the go-go IPO class of 2020

It’s another day of hauling cash in the biopharma world as four more IPOs priced Friday and a fifth filed its initial paperwork.

The biggest offering comes from PMV Pharma, an oncology biotech focusing on p53 mutations, which raised $211.8 million after pricing shares at $18 apiece. Prelude Therapeutics, developing PRMT5 inhibitors for rare cancers, was next with a $158 million raise, pricing shares at $19 each. Graybug Vision raised $90 million after pricing at $16 per share for its wet AMD candidates, and breast cancer biotech Greenwich Lifesciences brought up the rear with a small, $7 million raise after pricing shares at $5.75.

J&J of­fers PhI/IIa da­ta show­ing its sin­gle-dose vac­cine can stir up suf­fi­cient im­mune re­sponse

Days after J&J dosed the first participants of its Phase III ENSEMBLE trial, the pharma giant has detailed the early-stage data that gave them confidence in a single-dose regimen.

Testing two dose levels either as a single dose or in a two-dose schedule spaced by 56 days in, the scientists from Janssen, the J&J subsidiary developing its vaccine, reported that the low dose induced a similar immune response as the high dose. The interim Phase I/IIa results were posted in a preprint on medRxiv.

New York governor Andrew Cuomo (AP Images)

An­drew Cuo­mo says New York will un­der­take its own vac­cine re­view process, and wouldn’t rec­om­mend trust­ing the fed­er­al gov­ern­ment

The concerns keep mounting over President Donald Trump’s politicization of the FDA and other federal agencies guiding the development of a safe and effective vaccine. And today, the telegenic New York governor Andrew Cuomo appeared to introduce even more politics into the matter — latest in an ongoing series of incidents that have cast the proudly independent FDA in starkly political terms.

During his daily press conference Cuomo said that the state will review any coronavirus vaccines approved by the federal government, citing a lack of trust in the Trump administration. The announcement comes one day after Trump accused the FDA of making an “extremely political” move in proposing stricter vaccine guidance.

President Trump walks past HHS secretary Alex Azar (Getty Images)

Azar falls in line un­der Trump again. Ex­perts say he's re­in­forc­ing a dark sig­nal sent to the FDA

In the latest incident where Alex Azar has steadfastly taken the side of President Donald Trump over that of the FDA, the HHS secretary was noncommittal this morning when asked if he supports the attempt by his subordinates at the FDA to strengthen guidelines for a vaccine EUA.

Appearing on NBC’s Today Show, the HHS secretary muddied the waters, stating that the guidance that matters is the one that is “actually already out there.”