Brett Giroir, HHS

Azar keen on nom­i­nat­ing Brett Giroir, di­vi­sive as­sis­tant HHS sec­re­tary, to top job at FDA — re­port

HHS sec­re­tary Alex Azar re­port­ed­ly has his eye on nom­i­nat­ing Brett Giroir — con­tro­ver­sial­ly con­firmed as as­sis­tant sec­re­tary of HHS last year — to head the FDA as judg­ment day on Ned Sharp­less’ term as act­ing com­mis­sion­er looms.

Pres­i­dent Don­ald Trump must make a de­ci­sion about the com­mis­sion­er of food and drugs by No­vem­ber 1, as man­dat­ed by the Fed­er­al Va­can­cies Re­form Act of 1998  that en­ti­tles a Sen­ate-con­firmed po­si­tion to be filled in an act­ing ca­pac­i­ty for a max­i­mum of 210 days, af­ter the po­si­tion is va­cat­ed.  The ef­fer­ves­cent Scott Got­tlieb re­signed as FDA com­mis­sion­er ear­li­er this year, de­part­ing of­fi­cial­ly on April 5.

Sharp­less, a physi­cian/sci­en­tist who pre­vi­ous­ly served as head of the Na­tion­al Can­cer In­sti­tute, was wide­ly seen as Got­tlieb’s cho­sen suc­ces­sor and was warm­ly em­braced by the bio­phar­ma in­dus­try when he as­cend­ed to the FDA throne. Back­ing him will be a smoother choice by the White House.

A re­port by Bio­Cen­tu­ry — which re­port­ed Azar is keen on Giroir, cit­ing ad­min­is­tra­tion of­fi­cials — has al­so sug­gest­ed in­flu­en­tial pa­tient groups are cir­cu­lat­ing a let­ter urg­ing Trump to pick Sharp­less per­ma­nent­ly.

Un­like Sharp­less who is gen­er­al­ly seen as par­ty-ag­nos­tic, Giroir is un­abashed­ly Re­pub­li­can — his stance on re­pro­duc­tive rights sparked De­mo­c­ra­t­ic op­po­si­tion that held up his con­fir­ma­tion as as­sis­tant HHS sec­re­tary for months. He was whisked in­to the Trump ad­min­is­tra­tion by for­mer Texas Gov­er­nor and cur­rent Sec­re­tary of En­er­gy Rick Per­ry, Bio­Cen­tu­ry not­ed.

His ap­point­ment as FDA com­mis­sion­er will trig­ger con­cerns that the agency will end up en­tan­gled in the ten­ta­cles of pol­i­tics re­gard­ing drug im­por­ta­tion, re­pro­duc­tive and women’s health, FDA civ­il ser­vants told Bio­Cen­tu­ry.

End­points News has con­tact­ed Azar and Got­tlieb for com­ment on the re­port.

Giroir, a Texas pe­di­a­tri­cian, su­per­vis­es HHS’ Of­fice of Pop­u­la­tion Af­fairs, which ad­min­is­ters Ti­tle X grants — fund­ing for fam­i­ly plan­ning clin­ics and re­pro­duc­tive health ser­vices — as well as the Of­fice of Ado­les­cent Health, which over­sees the teen preg­nan­cy pre­ven­tion pro­gram.

Un­der Giroir, the HHS launched a re­view in­to the use of fe­tal tis­sue for re­search last year. This June, med­ical re­search fund­ing for NIH sci­en­tists us­ing fe­tal tis­sue —  har­vest­ed from dead fe­tus­es, from in­duced or nat­ur­al abor­tions — was abort­ed. Ear­li­er this month, Planned Par­ent­hood re­ject­ed Ti­tle X fund­ing — in re­sponse to a gag rule im­posed by the Trump ad­min­is­tra­tion that cur­tails re­cip­i­ents from giv­ing abor­tion re­fer­rals.

Af­ter se­cur­ing con­fir­ma­tion to take on the role of HHS as­sis­tant sec­re­tary, Giroir left his post as chief of Texas-based biotech Vira­Cyte (now re­named AlloVir), which is de­vel­op­ing im­munother­a­pies for vi­ral dis­eases.

The hope is the Trump ad­min­is­tra­tion does, in fact, take steps to nom­i­nate a per­ma­nent suc­ces­sor — whether Sharp­less, Giroir or an­oth­er per­son al­to­geth­er. Of 731 key lead­er­ship po­si­tions (out of rough­ly 1,200 po­si­tions) re­quir­ing Sen­ate con­fir­ma­tion — 145 po­si­tions have no nom­i­nee cho­sen by the Trump ad­min­is­tra­tion, ac­cord­ing to a track­er set up by The Wash­ing­ton Post and Part­ner­ship for Pub­lic Ser­vice, a non­prof­it or­ga­ni­za­tion.

Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Inside FDA HQ (File photo)

The FDA just ap­proved the third Duchenne MD drug. And reg­u­la­tors still don’t know if any of them work

Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

On Wednesday the FDA approved the third Duchenne MD drug, based on the same biomarker. And regulators were ready to act yet again despite the lack of efficacy data.

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A lab technician works during research on coronavirus at Johnson & Johnson subsidiary Janssen Pharmaceutical in Beerse, Belgium, Wednesday, June 17, 2020. (Virginia Mayo/AP Images)

UP­DAT­ED: End­points News ranks all 28 play­ers in the Covid-19 vac­cine race. Here's how it stacks up to­day

(This piece was last updated on August 13. Endpoints News will continue to track the latest developments through the FDA’s marketing decisions.)

The 28 players now in or close to the clinical race to get a Covid-19 vaccine over the finish line are angling for a piece of a multibillion-dollar market. And being first — or among the leaders — will play a big role in determining just how big a piece.

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Stéphane Bancel speaks to President Donald Trump at the White House meeting on March 2 (AP Images)

UP­DAT­ED: Mod­er­na of­fers steep dis­count in US sup­ply deal — but still takes the crown with close to $2.5B in vac­cine con­tracts

The US pre-order for Moderna’s Covid-19 vaccine is in.

Operation Warp Speed is reserving $1.525 billion for 100 million doses of Moderna’s Phase III mRNA candidate, rounding out to about $15 per dose — including $300 million in incentive payments for timely delivery. Given that Moderna has a two-dose regimen, it’s good for vaccinating 50 million people. The US government also has the option to purchase another 400 million doses for a total of $6.6 billion, or $16.5 per dose.

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James Wilson, WuXi Global Forum at JPM20

FDA puts up a red light for Pas­sage Bio’s first gene ther­a­py pro­gram, de­lay­ing a pro­gram from James Wilson's group at Penn

Gene therapy pioneer James Wilson spearheaded animal studies demonstrating the potential of new treatments injected directly into the brain, looking to jumpstart a once-and-done fix for an extraordinarily rare disease called GM1 gangliosidosis in infants. His team at the University of Pennsylvania published their work on monkeys and handed it over to Passage Bio, a Wilson-inspired startup building a pipeline of gene therapies — with an IND for PBGM01 to lead the way.

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FDA's ODAC shrugs off ob­jec­tions to Mesoblast's GVHD drug for chil­dren, vot­ing 8-2 in fa­vor and im­prov­ing the odds of an ap­proval

The FDA’s Oncologic Drugs Advisory Committee once again waved through an investigational drug, clearing the potential final hurdle before the agency’s decision.

Thursday’s winner was Mesoblast $MESO, an Australian stem-cell company that submitted its Ryoncil drug for the treatment of steroid refractory acute graft-versus-host disease in children younger than 12. ODAC gave Ryoncil the thumbs up by an 8-2 vote, shrugging off concerns about trial design and pushing aside the need for an additional study.

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Cal­lid­i­tas bets up to $102M on a biotech buy­out, snag­ging a once-failed PBC drug

After spending years developing its oral formulation of the corticosteroid budesonide, Sweden’s Calliditas now has its sights set on the primary biliary cholangitis field.

The company will buy out France-based Genkyotex, and it’s willing to bet up to €87 million ($102 million) that Genkyotex’s failed Phase II drug, GKT831, will do better in late-stage trials.

Under the current agreement, Calliditas $CALT will initially pay €20.3 million in cash for 62.7% of Genkyotex (or €2.80 a piece for 7,236,515 shares) in early October, then circle back for the rest of Genkyotex’s shares under the same terms. If nothing changes, the whole buyout will cost Calliditas €32.3 million, plus up to  €55 million in contingent rights.

Qi­a­gen in­vestors spurn Ther­mo Fish­er’s takeover of­fer, de­rail­ing a $12B+ deal

Thermo Fisher Scientific had announced an $11.5 billion takeover of Dutch diagnostics company Qiagen back in March, but the deal apparently did not sit well with Qiagen investors.

After getting hammered by critics who contended that Qiagen $QGEN was worth a lot more than what Thermo Fisher wanted to spend, investors turned thumbs down on the offer — derailing the buyout even after Thermo Fisher increased its offer to $12.6 billion in July. Qiagen’s share price has been boosted considerably by Covid-19 as demand for its testing kits surged.

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Xuefeng Yu in Hong Kong, 2019 (Imaginechina via AP Images)

CanSi­no reaps $748M wind­fall from Shang­hai IPO — as it warns Covid-19 vac­cine won't be a huge mon­ey mak­er

CanSino began the year with a clear goal to secure a secondary listing on Shanghai’s STAR market. Then something more urgent came along: As a rising vaccine developer on a mission to bring global standard immunizations to China, it heeded the call to make a vaccine to protect against a virus that would paralyze the whole world.

Xuefeng Yu and his team managed to keep doing both.

More than a month after CanSino’s Covid-19 vaccine candidate is authorized for military use in China, the Hong Kong-listed company has made a roaring debut in Shanghai. It fetched $748 million (RMB$5.2 billion) by floating 24.8 million shares, and soared 88% on its first trading day.