Rob Califf (AP Photo/Pablo Martinez Monsivais, File)

Biden like­ly to nom­i­nate Ver­i­ly's Rob Califf to lead the FDA again

Cap­ping a con­tro­ver­sial­ly long pe­ri­od for the FDA to go with­out a per­ma­nent leader, Pres­i­dent Joe Biden is like­ly to se­lect Ver­i­ly’s Rob Califf, a for­mer FDA com­mis­sion­er un­der Pres­i­dent Oba­ma, as the next FDA com­mis­sion­er nom­i­nee.

A for­mer Duke car­di­ol­o­gist and mem­ber of the pres­ti­gious Na­tion­al Acad­e­my of Med­i­cine, Califf will be a wel­come face for an agency grap­pling with high-pro­file re­tire­ments in CBER and CDER. He’ll al­so re­turn to a role that he was com­fort­able in for a short stint at the end of Oba­ma’s pres­i­den­cy. The Wash­ing­ton Post first re­port­ed the news.

Califf, un­like oth­er po­ten­tial nom­i­nees like act­ing FDA com­mis­sion­er Janet Wood­cock, isn’t like­ly to draw much ire from De­moc­rats even though he’s most re­cent­ly been work­ing for Google’s Ver­i­ly and as Sens. Joe Manchin (D-WV) and Richard Blu­men­thal (D-CT) pre­vi­ous­ly op­posed his nom­i­na­tion. But as the agency saw more re­cent­ly with Scott Got­tlieb, a for­mer FDA com­mis­sion­er with in­dus­try con­nec­tions who im­me­di­ate­ly fol­lowed Califf in his pre­vi­ous run, a for­mer po­si­tion does not nec­es­sar­i­ly af­fect how well one per­forms in the top role at FDA.

Califf’s ex­pect­ed nom­i­na­tion al­so caps a long and in­creas­ing­ly con­tro­ver­sial tenure for Janet Wood­cock at the top of FDA as an act­ing com­mis­sion­er. In some cir­cles of De­moc­rats Wood­cock was a non­starter be­cause of her back­ing from bio­phar­ma in­dus­try and be­cause of her role in the opi­oid cri­sis.

As a 35-year vet­er­an of the FDA, it’s un­clear what Wood­cock will do now with Califf mov­ing to the helm of FDA. She pre­vi­ous­ly led the FDA’s Cen­ter for Drug Eval­u­a­tion and Re­search since 1994, but that po­si­tion has been tak­en by Pa­trizia Cavaz­zoni, and Wood­cock might take a role re­lat­ed to Covid-19 as she served re­cent­ly as the top leader of Op­er­a­tion Warp Speed’s de­vel­op­ment of ther­a­peu­tics.

A source close to the de­ci­sion mak­ing process said that Ni­rav Shah, for­mer health com­mis­sion­er for New York, and Mon­i­ca Bertag­nol­li, a pro­fes­sor of surgery at Har­vard Med­ical School who al­so works at Dana-Far­ber, were al­so vet­ted for the per­ma­nent FDA com­mis­sion­er role.

White House press sec­re­tary Jen Psa­ki said at Thurs­day’s press brief­ing, “Well, the Pres­i­dent is def­i­nite­ly ea­ger to make a de­ci­sion about an FDA nom­i­nee and, of course, make that de­ci­sion pub­lic once it’s made. We’re just not quite at that point yet. In terms of the time­line, I’m not aware of what ex­act­ly that time­line is.”

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

Pfiz­er, Sarep­ta and two oth­ers sug­gest Duchenne drug safe­ty is­sues tied to "class ef­fect"

Since the first experimental Duchenne gene therapy programs came about, the space has proven rife with safety issues and patient deaths in clinical trials. Pfizer and three biotechs now think they’ve found a reason why.

The four companies suggested there may be a “class effect” causing the adverse events in Duchenne gene therapies, they wrote in a new study. They specifically highlighted how side effects in five patients across three trials, who all showed muscle weakness with cardiac involvement, were “strikingly similar.”

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Pre­sent­ing a live End­points News event: Man­ag­ing a biotech in tur­bu­lent times

Biotech is one of the smartest, best educated industries on the planet. PhDs abound. We’ve had a long enough track record to see a new generation of savvy, experienced execs coming together to run startups.

And in these times, they are being tested as never before.

Biotech is going through quite a rough patch right now. For 2 years, practically anyone with a decent resume and some half-baked ideas on biotech could start a company and get it funded. The pandemic made it easy in many ways to pull off an IPO, with traditional road shows shut down in exchange for a series of quick Zoom meetings. Generalist investors flocked as the numbers raised soared into the stratosphere.

Martin Shkreli (Dennis Van Tine/MediaPunch/IPX)

In­fa­mous biotech ex­ec Mar­tin Shkre­li gets out of prison, hits the street

Martin Shkreli, the infamous biotech CEO who made headlines for his jeering assault on a legion of critics in and out of Congress, is back on the streets after 4 years inside a federal penitentiary.

Shkreli’s attorney put out a statement Wednesday afternoon saying that the “pharma bro” had been transferred to a halfway house in New York with a few more months to go under federal custody, slated to end September 14. Attorney Benjamin Brafman acknowledged the release and vowed that he and Shkreli are keeping quiet.

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Frank Pallone (D-NJ), House Energy and Commerce Committee chair (Kevin Dietsch/Pool via AP Images)

House com­mit­tee unan­i­mous­ly ad­vances FDA user fee leg­is­la­tion with ac­cel­er­at­ed ap­proval tweaks

The House Energy and Commerce Committee on Wednesday offered a rare show of bipartisan support for a bill that would provide the FDA with user fees for the next five years.

The committee voted 55-0 to advance the quinquennial user fee bill to the full House floor, which if approved, will allow the FDA to use biopharma funds to hire new reviewers, and hit new marks as outlined in the user fee deals that the FDA and biopharma companies forged over the past several years.

Patty Murray (D-WA) (Graeme Sloan/Sipa USA)(Sipa via AP Images)

Sen­ate user fee reau­tho­riza­tion bill omits ac­cel­er­at­ed ap­proval re­forms, shows wide gaps with House ver­sion

The Senate health committee on Tuesday released its first version of the bill to reauthorize all the different FDA user fees. But unlike the House version, there are only a few controversial items in the Senate’s version, which does not address either accelerated approval reforms or clinical trial diversity (as the House did).

While it’s still relatively early in the process of finalizing this legislation (the ultimate statutory deadline is the end of September), the House and Senate, at least initially, appear to be starting off in different corners on what should be included.

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FDA lob­bies Con­gress over rare dis­ease court rul­ing with wide im­pli­ca­tions

Usually reserved for making decisions on drug applications or enforcing what Congress stipulates, the FDA is now dipping its toe into the wild world of congressional politics as it attempts to fix a major court decision that could have a chilling effect on rare disease R&D.

The case in question from last October saw a US appeals court overturn a prior FDA court win, saying that the agency never should’ve approved a rare disease drug because a previously approved but more expensive drug with the same active ingredient has orphan drug exclusivity barring such an approval.

Peter Marks (Greg Nash/Pool via AP)

Even FDA's Pe­ter Marks is wor­ried about the com­mer­cial vi­a­bil­i­ty of gene and cell ther­a­pies

When bluebird bio’s gene therapy to treat beta thalassemia won European approval in 2019, the nearly $2 million per patient price tag for the potential cure seemed like a surmountable hurdle.

Fast forward two years later, and bluebird has withdrawn Zynteglo, the beta thal drug, along with the rest of its gene therapy portfolio from Europe, which the company said is generally unwilling to pay a fair price for the treatment.

Pri­cy in­halers re­main ex­pen­sive due to de­vice tweaks that keep com­peti­tors at bay, re­searchers find

New research published in Health Affairs today highlights the way in which the FDA’s inhaler regulations have rewarded incremental adjustments to older products, thereby enabling companies to skirt around cheaper competition.

A DC appeals court clerk and researchers from Harvard and the University of Calgary dug through all the patents and regulatory exclusivities granted to inhalers approved by the FDA between 1986 and 2020, finding that of the 62 inhalers approved, 53 (or 85%) were brand-name products, with a median of 16 years of protection from generic competition.