Af­ter two years of ne­go­ti­a­tions, FDA gets its user fee pro­grams au­tho­rized through 2022

Pres­i­dent Don­ald Trump signed the FDA Reau­tho­riza­tion Act of 2017 in­to law late Fri­day, qui­et­ly end­ing a two-year long process that un­til sev­er­al weeks ago threat­ened to dis­rupt agency op­er­a­tions.

Al­though the leg­is­la­tion was passed by both hous­es of Con­gress with over­whelm­ing bi­par­ti­san sup­port — Sen­a­tor Bernie Sanders was the on­ly reg­is­tered no vote — it faced a num­ber of threats, in­clud­ing the White House’s clear de­sire to fund the FDA en­tire­ly with user fees from the com­pa­nies the agency reg­u­lates and a sen­a­tor’s long stand­ing cru­sade to un­der­cut the agency with a sweep­ing new “right-to-try” di­rec­tive.

The FDA is ex­pect­ed to col­lect $9 bil­lion in fees — $8 bil­lion for pre­scrip­tion drugs and $1 bil­lion for de­vices — be­tween 2018 and 2022, based on the fee lev­el set in the Sen­ate bill.

This marks the sixth it­er­a­tion of the PDU­FA pro­gram and the sec­ond for GDU­FA, which gov­erns gener­ic drug pro­grams. GDU­FA II cre­ates a new user fee struc­ture aimed at faster re­views and more in­ter­ac­tion be­tween the agency and gener­ics de­vel­op­ers work­ing on com­plex mol­e­cules. It comes on the heels of the FDA’s new record-set­ting pace on gener­ics ap­provals, spurred by new com­mis­sion­er Scott Got­tlieb.

Greg Walden (R-OR), the Re­pub­li­can chair­man of the House En­er­gy & Com­merce com­mit­tee had this to say on Medi­um: “FDARA is a win for pa­tients and the mil­lions of Amer­i­cans work­ing to cre­ate new cures and ther­a­pies. The bill is com­posed of four in­di­vid­ual user fee pro­grams, that pack­aged to­geth­er will de­liv­er re­sults for pa­tients and en­sure we re­main the glob­al leader in med­ical in­no­va­tion.”

 

Adap­tive De­sign Meth­ods Of­fer Rapid, Seam­less Tran­si­tion Be­tween Study Phas­es in Rare Can­cer Tri­als

Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no universally accepted definition for a “rare” cancer. Moreover, with the evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy.

Adaptive designs, which allow for prospectively planned modifications to study design based on accumulating data from subjects in the trial, can be used to optimize rare oncology trials (see Figure 1). Adaptive design studies may include multiple cohorts and multiple tumor types. In addition, numerous adaptation methods may be used in a single trial and may facilitate a more rapid, seamless transition between study phases.

Marianne De Backer (L) and Jeff Hatfield

Bay­er nabs star biotech Vi­vid­ion with a $2B buy­out and an ‘arms-length’ pact, pulling a part­ner out of the IPO con­ga line

Vividion is canceling that IPO it filed. Instead of following the industry-wide migration to Nasdaq, the biotech that has captured considerable attention for its still-preclinical work finding cryptic pockets to bind to on proteins is going to work for Bayer now.

The pharma giant is putting out word today that it has bought out Vividion for $1.5 billion in cash and another half-billion dollars in milestones.

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Tadataka Yamada (Photographer: Kiyoshi Ota/Bloomberg via Getty Images)

Sci­ence pi­o­neer, phar­ma re­search chief, glob­al health ad­vo­cate and biotech en­tre­pre­neur Tadata­ka ‘Tachi’ Ya­ma­da has died

Tadataka Yamada, a towering physician-scientist who made his name in academia before transforming drug development at GlaxoSmithKline and developing vaccines for malaria and meningitis at the Gates Foundation, died unexpectedly of natural causes at his home in Seattle Wednesday morning.

He was 76. Frazier Healthcare Partners’ David Socks confirmed his death.

Known widely by the mononym “Tachi,” Yamada had a globetrotting career and arrived in industry relatively late in life. A 2004 Independent article noted GSK had asked Yamada to stay on beyond his approaching 60th birthday, the company’s usual retirement age. Yamada would continue working for the next 17 years, steering the Gates Foundation’s global health division for 6 years, funding Jim Wilson’s gene therapy work when few would touch it, launching Takeda Vaccines and co-founding a series of high-profile biotechs.

Tien Lee, Aardvark Therapeutics

Emerg­ing from stealth mode, Aard­vark rounds up enough cash to put its lead drug through Prad­er-Willi PhII

When Aardvark Therapeutics CEO Tien Lee started his work on the biotech’s lead candidate, appetite suppression was the goal for the small molecule.  Soon after, his team started to see added benefits with lower blood glucose levels and anti-inflammatory activity. On the tail end of that, the company has emerged from stealth mode and announced today that they’ve raised enough cash in the B round to cover mid-stage development work.

Josh Hoffman, outgoing Zymergen CEO (Zymergen)

UP­DAT­ED: Syn­bio uni­corn Zymer­gen jet­ti­sons found­ing CEO, cuts guid­ance as cus­tomers re­port lead prod­uct does­n't work

Zymergen, just months off a $500 million IPO that put the synthetic bio firm in rarified air, has now ejected its founding CEO and downgraded its revenue forecasts after customers reported its lead film product doesn’t work as advertised, the company said Tuesday afternoon.

CEO Josh Hoffman will leave his role and sacrifice his board seat immediately in favor of Jay Flatley, the former CEO of Illumina who will take the lead role on an interim basis as the company conducts a search for its next leader.

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UK re-in­ves­ti­gates Pfiz­er's eye-pop­ping price goug­ing on an epilep­sy drug

When a drugmaker raises the price of a drug in the US by more than 2,000% overnight, and without any particular reason for that increase, nothing typically happens to the company. No fines, no court orders, just business as usual.

Martin Shkreli’s decades-old anti-parasitic drug Daraprim was the perfect example — massive price spike on an old drug, lots of media attention, public outcry, Congressional committees dragging his former company through multiple hearings, and at the end of it? Nothing happened to the price or the company (until generic competition came).

Thomas Lingelbach, Valneva CEO

A small vac­cine de­vel­op­er fa­vored by the UK gov­ern­ment in Covid-19 touts a PhI­II first in chikun­gun­ya

Before Valneva garnered the favor of the UK government as a potential supplier of Covid-19 vaccines, the French biotech prided itself on being the first company to bring a chikungunya vaccine into Phase III.

It now has positive pivotal results to back up the breakthrough therapy designation the FDA granted just weeks ago.

There are currently no approved jabs to prevent chikungunya virus infection despite decades of R&D efforts, a fact that underscores just how arduous traditional vaccine development can be, particularly for neglected tropical disease. In a absence of a major commercial market, the US government and NGOs such as CEPI have deployed various grants and incentives to spur on a small crew of academics and industry players, with Merck, via its acquisition of Themis, claiming a spot in that race.

Zymergen co-founders Zach Serber, Josh Hoffman, and Jed Dean (Zymergen via website)

Zymer­gen's sud­den im­plo­sion shocked biotech. A lin­ger­ing loan could make things even worse

As former synbio unicorn Zymergen picks up the pieces from its spectacular implosion Tuesday, an outstanding loan from Perceptive Advisors — the only blue-chip biotech crossover investor to touch Zymergen’s fundraising efforts — could make the situation worse, according to public documents.

In December 2019, more than a year before Zymergen filed for what would eventually become a $500 million IPO, the “biofacturing” firm signed a $100 million credit facility with Perceptive to help supplement the nearly $700 million the company had raised across four VC rounds.

Bio­gen, Ei­sai are push­ing for an­oth­er ac­cel­er­at­ed Alzheimer's OK — this time for BAN2401

Now that the door at the FDA has been opened wide for Alzheimer’s drugs that can demonstrate a reduction in amyloid, Biogen and its partners at Eisai are pushing for a quick OK on the next drug to follow in the controversial path of aducanumab.

In a presentation to analysts, Eisai neurology chief Ivan Cheung outlined some bullish expectations for their newly-approved treatment and set the stage for what he believes will be a fast follow for BAN2401 (lecanemab) — after a dry spell in new drug development that’s lasted close to 20 years.

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