Pop­u­lar FDA com­mish Scott Got­tlieb has hand­ed in his res­ig­na­tion

In a stun­ning turn of events, FDA com­mis­sion­er Scott Got­tlieb is re­sign­ing from his post and leav­ing of­fice in a mat­ter of weeks.

The Wash­ing­ton Post re­port­ed that Got­tlieb, who’s been com­mut­ing from Con­necti­cut, told ad­min­is­tra­tion of­fi­cials that he wants to spend more time with his fam­i­ly.

Got­tlieb ar­rived at the FDA short­ly af­ter the Trump ad­min­is­tra­tion took of­fice, vow­ing to up­hold the gold stan­dard in drug de­vel­op­ment while do­ing every­thing pos­si­ble to fa­cil­i­tate drug mak­ers’ work — at least for the le­git­i­mate play­ers.

He has been enor­mous­ly pop­u­lar in his short stint, with the vast ma­jor­i­ty of bio­phar­ma ex­ecs ac­claim­ing his work at the agency. He’s al­so been pop­u­lar with Pres­i­dent Don­ald Trump, find­ing a se­cure spot for him­self in an ad­min­is­tra­tion not­ed for rapid turnover.

So what hap­pened here?

Just weeks ago Got­tlieb in­sist­ed on Twit­ter that ru­mors of his pend­ing de­par­ture from the agency were flat wrong. He tweet­ed:

We’ve got a lot im­por­tant pol­i­cy we’ll ad­vance this year. I look for­ward to shar­ing my 2019 strate­gic roadmap soon.

The big ques­tion now is who will re­place him. Trump freaked out many ex­ecs by en­ter­tain­ing Pe­ter Thiel’s push for a lib­er­tar­i­an ap­proach, which would have erased many of the bar­ri­ers to in­tro­duc­ing new drugs — along with the cred­i­bil­i­ty that goes with an FDA sanc­tion. Trump, though, has many open po­si­tions in his ad­min­is­tra­tion, in­clud­ing Cab­i­net-lev­el posts. Stephen Os­troff has tak­en the helm on a strict­ly in­ter­im ba­sis be­fore, but he re­tired last fall, re­placed by Wal­mart ex­ec­u­tive Frank Yian­nas.

Got­tlieb’s let­ter of res­ig­na­tion in­cludes his list of ac­com­plish­ments for the past 23 months, from a record num­ber of new drug ap­provals to their con­tin­ued work on mas­ter­ing new tech­nolo­gies and tak­ing on e-cig­a­rette use by mi­nors.

I’m con­fi­dent that the FDA will con­tin­ue to ad­vance all these ef­forts, and many oth­er goals.

In­dus­try lead­ers quick­ly not­ed the sig­nif­i­cance of the move to­day.

“We’re go­ing to miss him. He was a ter­rif­ic leader of the FDA,” Al­ny­lam CEO and BIO chair­man John Maraganore told me.

“It’s a damn shame,” Tweet­ed long­time se­r­i­al en­tre­pre­neur Mike Gilman, echo­ing the sharp lev­el of dis­ap­point­ment about the news.

 


Im­age: Scott Got­tlieb, com­mis­sion­er of the Food and Drug Ad­min­is­tra­tion (FDA), pos­es for por­trait pho­tographs in front of the old FDA sign at the FDA in White Oak, MD on No­vem­ber 5, 2018 The Wash­ing­ton Post

A New Fron­tier: The In­ner Ear

What happens when a successful biotech venture capitalist is unexpectedly diagnosed with a chronic, life-disrupting vertigo disorder? Innovation in neurotology.

That venture capitalist was Jay Lichter, Ph.D., and after learning there was no FDA-approved drug treatment for his condition, Ménière’s disease, he decided to create a company to bring drug development to neurotology. Otonomy was founded in 2008 and is dedicated to finding new drug treatments for the hugely underserved community living with balance and hearing disorders. Helping patients like Jay has been the driving force behind Otonomy, a company heading into a transformative 2020 with three clinical trial readouts: Phase 3 in Ménière’s disease, Phase 2 in tinnitus, and Phase 1/2 in hearing loss. These catalysts, together with others in the field, highlight the emerging opportunity in neurotology.
Otonomy is leading the way in neurotology
Neurotology, or the treatment of inner ear neurological disorders, is a large and untapped market for drug developers: one in eight individuals in the U.S. have moderate-to-severe hearing loss, tinnitus or vertigo disorders such as Ménière’s disease.1 With no FDA-approved drug treatments available for these conditions, the burden on patients—including social anxiety, lower quality of life, reduced work productivity, and higher rates of depression—can be significant.2, 3, 4

Patrik Jonsson, the president of Lilly Bio-Medicines

Who knew? Der­mi­ra’s board kept watch as its stock price tracked Eli Lil­ly’s se­cret bid­ding on a $1.1B buy­out

In just 8 days, from December 6 to December 14, the stock jumped from $7.88 to $12.70 — just under the initial $13 bid. There was no hard news about the company that would explain a rise like that tracking closely to the bid offer, raising the obvious question of whether insider info has leaked out to traders.

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Ab­b­Vie do­nates $1M+ of the HIV drug that Chi­na is now rec­om­mend­ing for coro­n­avirus treat­ment

AbbVie is donating more than $1 million worth of an HIV drug to help combat the fast-spreading coronavirus outbreak in China, the company announced on Friday.

China’s National Health Commission has suggested Aluvia, a pill containing lopinavir and ritonavir, as one of two possible treatments for the symptoms of the virus currently known as 2019-nCoV in the absence of effective antiviral medications. The other part is nebulized alpha-interferon.

UP­DAT­ED: Ab­b­Vie and Al­ler­gan di­vesti­tures are in, and an old As­traZeneca drug comes home

When AbbVie announced their $63-billion Allergan acquisition last year, executives acknowledged the two companies would have to divest some drugs to satisfy regulators. The two main assets in discussion have now been sold off – and one of them is coming home.

AstraZeneca will acquire brazikumab, Allergan’s late-stage IL-23 candidate for Crohn’s disease and ulcerative colitis. The drug was originally developed by AstraZeneca’s defunct subsidiary MedImmune, in collaboration with Amgen. Allergan licensed it for $250 million upfront and $1.27 billion in milestones.

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As­traZeneca makes case for use of blood thin­ner Bril­in­ta in stroke pa­tients

AstraZeneca’s extravagant projections for its clot fighter Brilinta may have fizzled in the face of underwhelming trial data — but a new pivotal study is set to expand its use substantially.

On Monday, the British drugmaker said the drug, when taken in conjunction with aspirin, induced a statistically significant reduction in the risk of the primary composite endpoint of stroke and death, compared to aspirin alone, in 11,000 patients that have suffered minor acute ischaemic stroke or a high-risk transient ischemic attack (TIA).

Samantha Truex (file photo)

Bruce Booth and Saman­tha Truex's lat­est ven­ture aims just above Hu­mi­ra

In 2000, about a year after the first trial data on Humira came out, a Japanese team identified a new gene that appeared to prevent GI cancer in mice: gasdermin, they called it, after the particular proteins it expressed.

Over the next decade-and-a-half, researchers found five more genes in the same family – often identified as gasdermin A, B, C, D, E and F – and yet their purpose baffled scientists. Mutations in appeared to make mice bald (alopecia), but deleting it had no effect. Mutations in F and A were linked to deafness. Mutant E caused human cells to self-destruct.

“The exact biological function of these proteins remained unknown for more than 15 years,” three of the field’s top researchers wrote in a  Nature review in November.

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FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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Aymeric Le Chatelier, Ipsen

A $1B-plus drug stum­bles in­to an­oth­er big PhI­II set­back — this time flunk­ing fu­til­i­ty test — as FDA hold re­mains in ef­fect for Ipsen

David Meek

At the time Ipsen stepped up last year with more than a billion dollars in cash to buy Clementia and a late-stage program for a rare bone disease that afflicts children, then CEO David Meek was confident that he had put the French biotech on a short path to a mid-2020 launch.

Instead of prepping a launch, though, the company was hit with a hold on the FDA’s concerns that a therapy designed to prevent overgrowth of bone for cases of fibrodysplasia ossificans progressiva might actually stunt children’s growth. So they ordered a halt to any treatments for kids 14 and under. Meek left soon after to run a startup in Boston. And today the Paris-based biotech is grappling with the independent monitoring committee’s decision that their Phase III had failed a futility test.

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Roche's check­point play­er Tecen­triq flops in an­oth­er blad­der can­cer sub­set

Just weeks after Merck’s star checkpoint inhibitor Keytruda secured FDA approval for a subset of bladder cancer patients, Swiss competitor Roche’s Tecentriq has failed in a pivotal bladder cancer study.

The 809-patient trial — IMvigor010 — tested the PD-L1 drug in patients with muscle-invasive urothelial cancer (MIUC) who had undergone surgery, and were at high risk for recurrence.

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