How do you re­place a rock star like Scott Got­tlieb at the FDA? Maybe you can't

Any­one look­ing for a con­ve­nient weath­er vane to de­ter­mine the re­ac­tion to Scott Got­tlieb’s abrupt de­par­ture from the FDA need go no fur­ther than the $XBI. The S&P Biotech ETF took a 4% hit. And it was down an­oth­er 1.8% ahead of the bell on Thurs­day.


The mar­ket and the in­dus­try loved Got­tlieb, as we un­der­scored on sev­er­al oc­ca­sions with in­dus­try sur­veys high­light­ing the in­tense en­thu­si­asm for some­one who ad­vo­cat­ed for col­lab­o­rat­ing with the drug in­dus­try. Bio­phar­ma had a part­ner in Got­tlieb, and they all knew it.

Va­p­ing and the opi­oid epi­dem­ic — while big pub­lic pol­i­cy is­sues — meant ab­solute­ly noth­ing to the in­dus­try. An agency com­mit­ted to push­ing ap­provals while em­brac­ing in­no­va­tion at a time some land­mark ther­a­pies are be­ing ap­proved at a more rapid pace: That was pure gold. And it wasn’t just lip ser­vice. In this brave new world J&J could slip around the gold stan­dard on de­pres­sion drug da­ta and score an OK this week for es­ke­t­a­mine, a drug they plan to make a block­buster. A few years ago, that like­ly wouldn’t have hap­pened.

The one thing that could set­tle in­vestors and ex­ecs back down would be a new head of the FDA that could walk the line be­tween main­tain­ing the some­what weak­ened gold stan­dard on piv­otal da­ta and a free-for-all that would clear­ly aban­don a com­mit­ment to ap­prov­ing safe and ef­fec­tive drugs that met the risk/ben­e­fit analy­sis. 

Got­tlieb emerged ear­ly as a fa­vorite for the FDA job. He had ex­pe­ri­ence at the agency, he fit in­to the Trump ad­min­is­tra­tion’s orb with vows to speed things up, and an amaz­ing num­ber of in­dus­try in­sid­ers knew him and liked him.

He quick­ly be­came a rock star in bio­phar­ma, tweet­ing his way through every day about pol­i­cy.

So how do you re­place him and main­tain the same lev­el of en­thu­si­asm in bio­phar­ma? The usu­al sus­pects don’t cut it. 

We’re not even sure right now who will be the in­ter­im chief. Stephen Os­troff has tak­en the helm on a strict­ly in­ter­im ba­sis be­fore, but he re­tired last fall. Amy Aber­nethy was named prin­ci­pal deputy com­mis­sion­er just 2 months ago, com­ing to the FDA from a se­nior sci­en­tif­ic po­si­tion at Flat­iron af­ter a stint at Duke. She’s been tapped as the like­ly in­ter­im chief, and will def­i­nite­ly make the ru­mor mill as pos­si­bly the next head of the FDA. But she’s still large­ly an un­known in the in­dus­try, so don’t ex­pect much en­thu­si­asm right off the bat.

On the oth­er hand, if she gets the in­ter­im job, she’ll have a chance to shine. And Pres­i­dent Trump doesn’t seem pressed to get big jobs filled fast in Wash­ing­ton DC, so the in­ter­im role could last awhile. 

Na­tion­al Can­cer In­sti­tutes di­rec­tor Ned Sharp­less has emerged as one of the most fre­quent­ly cit­ed can­di­dates for the FDA job, with an ear­ly push from the Wall Street Jour­nal, which al­so quick­ly named Brett Giroir, as­sis­tant sec­re­tary at HHS, as an­oth­er pos­si­ble suc­ces­sor.

Jim O’Neill ev­i­dent­ly had a shot at the job a cou­ple of years ago, but the in­dus­try was shocked by the fact that Pe­ter Thiel’s can­di­date was even in the run­ning. He’s been as­so­ci­at­ed with some loony en­deav­ors, like seast­eading and the hunt for im­mor­tal­i­ty, and the in­dus­try sees him as a dis­as­ter wait­ing to hap­pen. In this en­vi­ron­ment, that makes O’Neill a cred­i­ble threat again, though it seems far fetched.

But he’s still go­ing to make every­one’s list. Fun­ny how that works.

Richard Paz­dur

How about Richard Paz­dur, the on­col­o­gy czar at the FDA whose com­mit­ment to rapid drug re­views has played a ma­te­r­i­al role in rev­o­lu­tion­iz­ing on­col­o­gy R&D over the last 5 years? It’s been sug­gest­ed. But when it comes to agency in­sid­ers, though, no one out­shines Janet Wood­cock at CDER. She not on­ly knows where the bod­ies are buried at the FDA, she of­ten helped put them there. Maybe she could cap a decades-long run as FDA chief? 

Janet Wood­cock

Stranger things have hap­pened.

Two years ago I called Got­tlieb the Re­pub­li­can shad­ow com­mis­sion­er, out front of­fer­ing po­si­tions on re­form­ing the FDA. We don’t seem to have one of those now, by virtue of the fact that no one ex­pect­ed Got­tlieb to stand down so soon. And with a na­tion­al elec­tion loom­ing next year with a wild­ly con­tro­ver­sial pres­i­dent, any­one who does get the job may ul­ti­mate­ly prove an in­ter­im chief in any case.

Bio­phar­ma hates reg­u­la­to­ry un­cer­tain­ty of any kind. This year we’ve had a gov­ern­ment shut­down fol­lowed by an abrupt res­ig­na­tion by Got­tlieb. Un­cer­tain­ty at the FDA ap­pears to now be the rule. 

In a note to in­vestors, Jef­feries’ Michael Yee not­ed that who­ev­er steps in will find an agency that has es­tab­lished a “pro-in­no­va­tion” ap­proach that won’t sud­den­ly end now, re­gard­less of who gets the job.

That sounds rea­son­able, but it won’t stop the fret­ting that’s go­ing on now. Ap­proach­es are one thing, ex­e­cu­tion is an­oth­er.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Albert Bourla, Pfizer CEO (Efren Landaos/Sipa USA/Sipa via AP Images)

Pfiz­er makes an­oth­er bil­lion-dol­lar in­vest­ment in Eu­rope and ex­pands again in Michi­gan

Pfizer is continuing its run of manufacturing site expansions with two new large investments in the US and Europe.

The New York-based pharma giant’s site in Kalamazoo, MI, has seen a lot of attention over the past year. As a major piece of the manufacturing network for Covid-19 vaccines and antivirals, Pfizer is gearing up to place more money into the site. Pfizer announced it will place $750 million into the facility, mainly to establish “modular aseptic processing” (MAP) production and create around 300 jobs at the site.

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Vas Narasimhan, Novartis CEO (Thibault Camus/AP Images, Pool)

No­var­tis bol­sters Plu­vic­to's case in prostate can­cer with PhI­II re­sults

The prognosis is poor for metastatic castration-resistant prostate cancer (mCRPC) patients. Novartis wants to change that by making its recently approved Pluvicto available to patients earlier in their course of treatment.

The Swiss pharma giant unveiled Phase III results Monday suggesting that Pluvicto was able to halt disease progression in certain prostate cancer patients when administered after androgen-receptor pathway inhibitor (ARPI) therapy, but without prior taxane-based chemotherapy. The drug is currently approved for patients after they’ve received both ARPI and chemo.

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Rick Modi, Affinia Therapeutics CEO

Ver­tex-part­nered gene ther­a­py biotech Affinia scraps IPO plans

Affinia Therapeutics has ditched its plans to go public in a relatively closed-door market that has not favored Nasdaq debuts for the drug development industry most of this year. A pandemic surge in 2020 and 2021 opened the doors for many preclinical startups, which caught Affinia’s attention and gave the gene therapy biotech confidence in the beginning days of 2022 to send in its S-1.

But on Friday, Affinia threw in the S-1 towel and concluded now is not the time to step onto Wall Street. The biotech has put out few public announcements since the spring of this year. Endpoints News picked the startup as one of its 11 biotechs to watch last year.

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Af­ter M&A fell through, Ther­a­peu­tic­sMD sells hor­mone ther­a­py, con­tra­cep­tive ring for $140M cash plus roy­al­ties

TherapeuticsMD, a women’s health company whose one-time billion-dollar valuation seems a distant memory as its blockbuster aspirations petered out, is finally cashing out.

Australia’s Mayne Pharma is paying $140 million upfront to license essentially TherapeuticsMD’s whole portfolio, including two prescription drugs that treat conditions relating to menopause, a contraceptive vaginal ring as well as its prescription prenatal vitamin brands.

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Big week for Alzheimer’s da­ta; As­traZeneca buys cell ther­a­py start­up; Dig­i­tal ther­a­peu­tics hits a pay­er wall; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

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Yuling Li, Innoforce CEO

In­no­force opens new man­u­fac­tur­ing site in Chi­na

Innoforce is off to the races at its new site in the city of Hangzhou, China.

The Chinese CDMO announced last week that it has started manufacturing at the new facility, which was built to offer process development and manufacturing operations for RNA, plasmid DNA, viral vectors and other cell therapeutics. It will also serve as Innoforce’s corporate HQ.

The company said it’s investing more than $200 million in the 550,000-square-foot manufacturing base for advanced therapies. The GMP manufacturing facility features space for producing plasmids with three 30-liter bioreactors. For viral vector manufacturing, Innoforce also has 200- and 500-liter bioreactors at its disposal, along with eight suites to make cell therapies. The site also includes several labs and warehouse spaces.

FDA grants or­phan drug des­ig­na­tion to Al­ger­non's ifen­prodil, while ex­clu­siv­i­ty re­mains un­clear

As the FDA remains silent on orphan drug exclusivity in the wake of a controversial court case, the agency continues to hand out new designations. The latest: Algernon Pharmaceuticals’ experimental lung disease drug ifenprodil.

The Vancouver-based company announced on Monday that ifenprodil received orphan designation in idiopathic pulmonary fibrosis (IPF), a chronic lung condition that results in scarring of the lungs.  Most IPF patients suffer with a dry cough, and breathing can become difficult.