The Trump ef­fect: PwC re­port says the FDA has slammed the brakes on sig­nif­i­cant new drug reg­u­la­tions

Don­ald Trump launched his pres­i­den­cy breath­ing fire on fed­er­al reg­u­la­tions. And the top of­fi­cials at the FDA heard that call loud and clear.

Scott Got­tlieb

A new re­port out from Price­wa­ter­house­C­oop­ers shows that the agency reined in its is­suance of new fed­er­al reg­u­la­tions to a 20-year low in 2017, as the Scott Got­tlieb stepped up to the helm as com­mis­sion­er.

In­stead, the agency has shift­ed to new guid­ances in pref­er­ence to hard­line reg­u­la­tions, says PwC. And that will like­ly come as a wel­come em­pha­sis in the bio­phar­ma in­dus­try, where we’ve seen the FDA re­peat­ed­ly demon­strate its will­ing­ness to re­lax old stan­dards to ac­cel­er­ate new drug de­vel­op­ment.

Over the 4-year run of the Oba­ma ad­min­is­tra­tion, says the re­port, the agency is­sued 132 “eco­nom­i­cal­ly sig­nif­i­cant” reg­u­la­tions for the in­dus­try as a whole — an av­er­age of 33 per year. Un­der Trump and Got­tlieb, that dropped to a low of just 6.

And while the num­ber of new drug ap­provals has soared to a record high, with 53 new ap­provals — a com­bi­na­tion of bi­o­log­ics and new chem­i­cal en­ti­ties — PwC sees the FDA large­ly stay­ing the course on in­dus­try over­sight, in­spec­tions and en­force­ment.

For the phar­ma­ceu­ti­cal in­dus­try, the FDA un­der Got­tlieb’s tenure has been pre­dictable, sta­ble and con­sis­tent, al­low­ing com­pa­nies to con­tin­ue in­vest­ments in­to drug re­search and de­vel­op­ment and ex­ist­ing com­pli­ance pro­grams with­out fear of a ma­jor change in reg­u­la­to­ry ap­proach. While Got­tlieb’s reg­u­la­to­ry phi­los­o­phy is per­haps more in­dus­try-friend­ly than past FDA com­mis­sion­ers, the agency’s broad­er ap­proach from en­force­ment to ap­provals has not been an abrupt de­par­ture from ex­ist­ing trends and norms.

A few oth­er high­lights:

  • Or­phan drug ap­provals rock­et­ed to a new high, with 64 new ap­provals in 2017, un­der­scor­ing a trend dri­ven by small­er clin­i­cal tri­als and steep prices for new roll­outs re­strict­ed to tiny pa­tient pop­u­la­tions.
  • Da­ta in­tegri­ty re­mains a key con­cern. And reg­u­la­tors re­main vig­i­lant about is­su­ing qual­i­ty re­lat­ed warn­ing let­ters.

Will the agency’s move to brake new reg­u­la­tions last through 2018?

Per­haps not, says the PwC re­port. A look at his­tor­i­cal trends show a dra­mat­ic in­crease in the sec­ond year of the George W Bush ad­min­is­tra­tion, with a more mod­est 15.4% in­crease in the sec­ond year of the Oba­ma ad­min­is­tra­tion. And their an­a­lysts flagged a list of sig­nif­i­cant new regs now un­der de­vel­op­ment at the FDA that could have a big im­pact on bio­phar­ma com­pa­nies.

As we re­port­ed re­cent­ly, Scott Got­tlieb and the FDA are held in high re­gard by drug de­vel­op­ers. And the PwC group found that there’s still a big push on for fur­ther eas­ing FDA stan­dards for ap­proval, as well as clin­i­cal and pre­clin­i­cal reg­u­la­tions.


Im­age: Pres­i­dent Don­ald Trump speaks with re­porters in No­vem­ber, 2017. Shut­ter­stock. Charts: PWC

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Fangliang Zhang (Imaginechina via AP Images)

The big mon­ey: Poised to make drug R&D his­to­ry, a Chi­na biotech un­veils uni­corn rac­ing am­bi­tions in a bid to raise $350M-plus on Nas­daq

Almost exactly three years after Shanghai-based Legend came out of nowhere to steal the show at ASCO with jaw-dropping data on their BCMA-targeted CAR-T for multiple myeloma, the little player with Big Pharma connections is taking a giant step toward making it big on Wall Street. And this time they want to seal the deal on a global rep after staking out a unicorn valuation in what’s turned out to be a bull market for biotech IPOs — in the middle of a pandemic.

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Gilead re­leas­es an­oth­er round of murky remde­sivir re­sults

A month after the NIH declared the first trial on remdesivir in Covid-19 a success, Gilead is out with new results on their antiviral. But although the study met one of its primary endpoints, the data are likely to only add to a growing debate over how effective the drug actually is.

In a Phase III trial, patients given a 5-day dose of remdesivir were 65% more likely to show “clinical improvement” compared to an arm given standard-of-care. The trial, though, gave little indication for whether the drug had an impact on key endpoints such as survival or time-to-recovery. And in a surprising twist, a 10-day dosing arm of remdesivir didn’t lead to a statistically significant improvement over standard of care.

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Len Schleifer (left) and George Yancopoulos, Regeneron (Vimeo)

Eyes on he­mo­phil­ia prize, Re­gen­eron adds a $100M wa­ger on joint de­vel­op­ment cam­paign with In­tel­lia

When George Yancopoulos first signed up Intellia to be its CRISPR/Cas9 partner on gene editing projects 4 years ago, the upstart smartly ramped up its IPO at the same time. Today, Regeneron $REGN is coming back in, adding $100 million in an upfront fee and equity to significantly boot up a whole roster of new development projects.

And they’re highlighting some clinical hemophilia research plans in the process.

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Iterum's fu­ture looks un­cer­tain, af­ter lead an­tibi­ot­ic fails con­sec­u­tive piv­otal stud­ies

While the market for antibiotics remains in tatters — unlike many of its bankrupt (or at the brink of bankruptcy) peers — Iterum is suffering not because its antibiotic isn’t selling, but because the compound has now failed back-to-back late-stage studies.

The experimental drug, sulopenem, was designed to tackle drug-resistant infections with an outpatient focus (in addition to hospitals), to avert those reimbursement challenges that incentivize hospitals to prescribe cheaper, generic broad-spectrum antibiotics.

Jean-Jacques Bienaimé, BioMarin chairman and CEO

Bio­Marin holds the line on bleeds with 4-year val­rox up­date on he­mo­phil­ia A — but what's this about an­oth­er de­cline in Fac­tor 8 lev­els?

BioMarin has posted some top-line results for their 4-year followup on the most advanced gene therapy for hemophilia A — extending its streak on keeping a handful of patients free of bleeds and off Factor VIII therapy, but likely stirring fresh worries over a continued drop in Factor VIII levels.

We just don’t know how big a drop.

We’ll see more data when the results are presented at the World Federation of Hemophilia in a couple of weeks. But in a statement out Sunday night, BioMarin $BMRN reported that none of the patients required Factor VIII treatment, adding:

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Covid-19 roundup: Did in­sid­ers cash in on pos­i­tive news re­port about Gilead be­fore pub­li­ca­tion?

A series of bullish trades on Gilead options just before the release of a favorable news story is raising questions among regulatory experts, Reuters reported.

On April 16, just hours before STAT published anecdotes from a Chicago hospital that served as one of the clinical sites to test Gilead’s remdesivir in Covid-19 patients, the California-based company’s shares were trading at around $75. Four large blocks of options were purchased for about $1.5 million each, betting that the stock would rise beyond that to as much as $87.5 by mid-August.

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Roche nabs front­line OK for Avastin/Tecen­triq in com­mon liv­er can­cer, best­ing an old Bay­er drug

For the first time in 12 years, the FDA has approved a new frontline treatment for the most common form of liver cancer.

The agency okayed a combination of Roche’s anti-VEGF antibody Avastin and their immunotherapy Tecentriq for patients with unresectable or metastatic hepatocellular carcinoma (HCC). The approval comes two weeks after Roche and their big biotech sub Genentech published Phase III results showing the combo improved both progression-free survival and, crucially, helped patients live longer than the long-running standard-of-care, Bayer’s Nexavar.

As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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