Robert Califf (L) shakes hands with Sen. Mitt Romney (R-UT) at Califf's confirmation hearing for FDA commissioner, Dec. 14, 2021 (Graeme Sloan/Sipa via AP Images)

Sen­ate health com­mit­tee again of­fers its sup­port for FDA com­mis­sion­er nom­i­nee Rob Califf

Rob Califf, the fa­mous car­di­ol­o­gist from Duke Uni­ver­si­ty, is like­ly to re­turn to the top of the FDA, this time un­der the Biden ad­min­is­tra­tion.

At his con­fir­ma­tion hear­ing Tues­day, De­moc­rats and Re­pub­li­cans on the Sen­ate health com­mit­tee of­fered their sup­port for Califf, with Chair Pat­ty Mur­ray (D-WA) stress­ing the need for an ex­pe­ri­enced leader, like Califf, who can en­sure that sci­ence comes first.

“I’m not sure you could write a re­sume for some­one who’s more qual­i­fied to be FDA com­mis­sion­er,” Sen. Richard Burr (R-NC) said while in­tro­duc­ing Califf and say­ing he thought there was enough Re­pub­li­can sup­port to get Califf across the fin­ish line.

While crit­i­ciz­ing Pres­i­dent Joe Biden for leav­ing this com­mis­sion­er po­si­tion open for al­most a year, Burr called on Califf to en­sure that FDA does “not go back to the ways be­fore Covid. We need a war time com­mis­sion­er.”

Califf, who re­cent­ly worked as a se­nior ad­vis­er to Google Health and Ver­i­ly, and is well-known for his clin­i­cal re­search knowl­edge, called the pan­dem­ic “dev­as­tat­ing,” and said he was ready to “hit the ground run­ning” while thank­ing act­ing FDA com­mis­sion­er Janet Wood­cock and the agency’s staffers for their “ex­em­plary ser­vice” over the past sev­er­al years.

“I’m go­ing to be very ag­gres­sive in this area of med­ical mis­in­for­ma­tion,” Califf said.

He stressed that all of the FDA’s ac­tions must fo­cus on pro­tect­ing con­sumers and pa­tients, call­ing for a more “sys­tem­at­ic ap­proach to im­prove pa­tient safe­ty and bet­ter un­der­stand ben­e­fits and risks” of med­ical prod­ucts.

Califf was al­so ques­tioned re­peat­ed­ly on re­al weal world ev­i­dence, one of his long­time pet projects. He said the FDA needs to get sys­tems in place to pro­duce re­al world da­ta more quick­ly, give peo­ple the con­fi­dence to share da­ta, and “get those rules right.”

When sev­er­al De­moc­rats raised con­cerns with the FDA’s han­dling of the con­tin­ued opi­oid epi­dem­ic, Califf com­mit­ted to con­duct­ing a re­view of opi­oid la­bels, adding, “We need to learn from the past sev­er­al decades and re­assess be­cause what­ev­er we’ve done un­til now is not enough.”

At­tract­ing and re­tain­ing the sci­en­tif­ic work­force at FDA, mean­while, “may be more im­por­tant than any oth­er pol­i­cy,” Califf said. Two se­nior vac­cine of­fi­cials re­cent­ly re­tired and CBER in par­tic­u­lar is strug­gling to re­tain and re­cruit tal­ent.

While Califf has his foes, most­ly for his web of fi­nan­cial ties to in­dus­try, in­clud­ing De­moc­rats like Joe Manchin (WV), Ed Markey (MA), Richard Blu­men­thal (CT), Bernie Sanders, and po­ten­tial­ly Ben Lu­ján (NM), Re­pub­li­cans who pre­vi­ous­ly sup­port­ed him in 2016 — Sens. Burr, Bill Cas­sidy (LA), Su­san Collins (ME), and Mitt Rom­ney (UT) — seemed to of­fer their sup­port again in Tues­day’s hear­ing.

And should more De­moc­rats de­fect from vot­ing for Biden’s se­lec­tion for FDA, a to­tal of 34 Re­pub­li­cans who pre­vi­ous­ly vot­ed to con­firm Califf on his first time around are still in the Sen­ate.

“The Covid-19 pan­dem­ic has made clear­er than ever the choice be­tween phar­ma­ceu­ti­cal prof­its and the health of our peo­ple. In this crit­i­cal mo­ment, Dr. Califf is not the leader Amer­i­cans need at the FDA,” Sanders said in a state­ment on Tues­day.

In ques­tions from Burr and oth­ers on the ac­cel­er­at­ed ap­proval path­way, which is un­der scruti­ny in­side the agency and with­in HHS, Califf said that he’s “a fan of ac­cel­er­at­ed ap­proval un­der the right con­di­tions,” such as for peo­ple with rare ge­net­ic dis­eases or life-threat­en­ing can­cer. But he con­ced­ed the US needs a bet­ter sys­tem to eval­u­ate ac­cel­er­at­ed ap­proval prod­ucts as they’re used on the mar­ket.

Six for­mer FDA com­mis­sion­ers, as well as more than 90 oth­er or­ga­ni­za­tions of­fered their sup­port for Califf late Mon­day, in­clud­ing a let­ter of sup­port from the Na­tion­al Health Coun­cil, which has PhRMA and BIO ex­ec­u­tives on its board.

Oth­er Re­pub­li­cans ques­tioned Califf over what the FDA might do with the birth con­trol drug mifepri­s­tone, as the Biden ad­min­is­tra­tion pre­vi­ous­ly sig­naled that the FDA may re­move a decades-old pro­hi­bi­tion on dis­pens­ing such pills via mail. But Califf said he would lis­ten to the ca­reer ex­perts and the lat­est da­ta.

And while the FDA doesn’t usu­al­ly dip its toes in­to ar­eas like drug pric­ing, Califf said that he’s on record sup­port­ing Medicare’s abil­i­ty to ne­go­ti­ate prices. Sanders al­so ques­tioned Califf on how to make drugs more af­ford­able and Califf men­tioned the need for a more ro­bust gener­ics in­dus­try. The biosim­i­lars pro­gram needs to do bet­ter too, he said.

“Wealthy coun­tries need to pay their fair share,” Califf said. But he not­ed the US has not a hit a sweet spot, as fi­nan­cial tox­i­c­i­ty re­mains a se­ri­ous is­sue for can­cer pa­tients. Al­most one-quar­ter of those pa­tients skip their chemo treat­ments be­cause of the cost, he said.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

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Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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FDA ap­proves Nar­can opi­oid over­dose re­ver­sal spray for over-the-counter sale

The FDA today approved Emergent BioSolutions’ Narcan brand naloxone nasal spray for over-the-counter sales. The nod was expected and comes on the heels of a unanimous 19-0 advisory committee vote in favor of approval last month.

The move to OTC means the opioid overdose reversal agent will now be available on grocery, convenience and gas stations shelves, as well as potentially for purchase online.

CSL CEO Paul McKenzie (L) and CMO Bill Mezzanotte

Q&A: New­ly-mint­ed CSL chief ex­ec­u­tive Paul McKen­zie and chief med­ical of­fi­cer Bill Mez­zan­otte

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.

Boehringer re­ports ro­bust sales led by type 2 di­a­betes and pul­monary drugs, promis­es more to come high­light­ing obe­si­ty

Boehringer Ingelheim reported human pharma sales of €18.5 billion on Wednesday, led by type 2 diabetes and heart failure drug Jardiance and pulmonary fibrosis med Ofev. Jardiance sales reached €5.8 billion, growing 39% year over year, while Ofev took in €3.2 billion, notching its own 20.6% annual jump.

However, Boehringer is also looking ahead with its pipeline, estimating “In the next seven years the company expects about 20 regulatory approvals in human pharma.”

Blue­bird to miss Q1 dead­line for lo­vo-cel fil­ing, shares sliced

Bluebird bio had expected to submit a BLA for its sickle cell disease gene therapy lovotibeglogene autotemcel, or lovo-cel, by Friday. Now, that deadline is getting punted for at least a couple of weeks.

CEO Andrew Obenshain detailed the update during the company’s full-year earnings call Wednesday morning after the gene therapy specialist reported the development in its Q4 and 2022 report.

“We will likely miss the Q1 2023 submission goal,” Obenshain said, noting the application is fully written and ready to go. But bluebird is still waiting on FDA feedback for the company’s CMC module submitted in early March.

Jeff Bluestone (R), Sonoma Biotherapeutics CEO

Jef­frey Blue­stone brings his start­up haul to $400M+, join­ing forces with Re­gen­eron on cell ther­a­pies

These days, when Jeffrey Bluestone gets together with his contemporaries in science, the conversation often turns to retirement plans.

But a little more than three years ago, Bluestone reached a momentous turning point in his career, exiting a prestigious post at UCSF, where he had spent decades in the scientific pursuit of new therapies. And it had nothing to do with retirement anytime in the near future.

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