The De­part­ment of De­fense could un­der­mine FDA, giv­ing the green light for ex­per­i­men­tal prod­ucts

A de­fense bill mak­ing its way through the Sen­ate would give the Pen­ta­gon pow­er to ap­prove new drugs and med­ical de­vices in some emer­gency in­stances, with clear­ance to ig­nore ad­vice from the FDA.

The pro­posed pol­i­cy would give the De­fense De­part­ment un­prece­dent­ed lee­way, thanks to the bill’s vague lan­guage. The con­cept is alarm­ing con­gres­sion­al staff and Health and Hu­man Ser­vices of­fi­cials, ac­cord­ing to Polit­co, who first re­port­ed the news.

Specif­i­cal­ly, the bill gives the Pen­ta­gon au­thor­i­ty to OK the use of ex­per­i­men­tal and un­ap­proved drugs and med­ical de­vices for emer­gency use on mil­i­tary per­son­nel and “in­di­vid­u­als as­so­ci­at­ed with de­ployed mem­bers of the armed forces,” the bill states. The idea is emer­gency use of some prod­ucts could “re­duce deaths and sever­i­ty of in­juries caused by agents of war.”

One way the DoD might flex this new pow­er is to ap­prove the use of prod­ucts like freeze-dried plas­ma to treat mil­i­tary per­son­nel who have suf­fered blood loss on the bat­tle­field. Freeze-dried plas­ma, un­ap­proved for use in the US, is a sta­ble and long-last­ing dry prod­uct that could be kept at room tem­per­a­ture. Use of the prod­uct, it’s thought, could save lives in the field.

Joe Panet­ta

Joe Panet­ta, pres­i­dent and CEO of Cal­i­for­nia’s biotech in­dus­try group Bio­com, tells me he’s not sur­prised by the DoD’s move.

“I think the mil­i­tary has his­tor­i­cal­ly been giv­en unique priv­i­leges in a num­ber of ar­eas out­side of what ap­plies to in­dus­try and the gen­er­al pub­lic,” Panet­ta said in an email. “This sit­u­a­tion is no dif­fer­ent. I know that in­dus­try wants a process that en­sures safe­ty and ef­fi­ca­cy thanks to a stan­dard­ized and uni­ver­sal­ly ac­cept­ed reg­u­la­to­ry process, i.e. FDA … (But) I em­pathize with the fact that FDA nor­mal­ly pro­ceeds at a pace that does not fit the mil­i­tary’s sit­u­a­tion at times.”

Still, con­cerns arise from how broad the lan­guage is in the bill. For in­stance, the phrase “agents of war” is not a le­gal de­f­i­n­i­tion. Politi­co re­ports that it could open the door for the Pen­ta­gon to ap­prove a wide range of prod­ucts and treat­ments.

The bill does spell out two re­quire­ments the DoD has to meet be­fore sign­ing off on an ex­per­i­men­tal treat­ment or de­vice. First, the DoD would cre­ate a new com­mit­tee of health care ex­perts who must rec­om­mend the use of an un­ap­proved prod­uct. Sec­ond, the as­sis­tant sec­re­tary of de­fense for health af­fairs would need to au­tho­rize the prod­uct’s use af­ter con­sult­ing with the FDA.

Panet­ta said he wasn’t sure what oth­er in­dus­try ex­perts might say, but per­son­al­ly he thinks this sort of pro­vi­sion is war­rant­ed.

“I think that if there is an im­mi­nent need for a ther­a­py or de­vice … then they should be al­lowed to do this,” he wrote in the email.

But, Panet­ta has caveats. The DoD should waive all com­pa­ny li­a­bil­i­ty for the prod­uct, and they should have a re­view pro­ce­dure that ad­e­quate­ly eval­u­ates the risk to their per­son­nel, Panet­ta said. He said an at­trac­tive al­ter­na­tive might be to work with the FDA to set up a spe­cial ex­pidit­ed re­view process for these sit­u­a­tions.

The FDA ac­tu­al­ly of­fered such an al­ter­na­tive to the DoD, but the lan­guage wasn’t ac­cept­ed for the bill, ac­cord­ing to Politi­co.

At an event Tues­day, FDA Com­mis­sion­er Scott Got­tlieb said con­trol over med­ical de­vice and drug ap­provals should stay in the hands of the FDA, The Hill re­ports. Still, he said he sup­ports of an ac­cel­er­at­ed ap­proval process for prod­ucts used on the bat­tle­field, and is will­ing to mod­i­fy the lan­guage the agency pro­posed for the DoD’s bill.

“I’m ful­ly com­mit­ted to try­ing to ex­pe­dite prod­ucts for the war fight­er, and … if they pass the lan­guage that has been put for­ward — the al­ter­na­tive lan­guage — we will com­mit to very quick­ly putting in place the im­ple­ment­ing guid­ance to stand up that process,” Got­tlieb said at the event.


Check out the full text of the Na­tion­al De­fense Au­tho­riza­tion Act for Fis­cal Year 2018 bill. Jump to Sec­tion 732 for more on emer­gency use of un­ap­proved prod­ucts.

Hal Barron and Rick Klausner (GSK, Lyell)

Ex­clu­sive: GSK’s Hal Bar­ron al­lies with Rick Klaus­ner’s $600M cell ther­a­py start­up, look­ing to break new ground blitz­ing sol­id tu­mors

LONDON — Chances are, you’ve heard little or nothing about Rick Klausner’s startup Lyell. But that ends now.

Klausner, the former head of the National Cancer Institute, former executive director for global health at the Gates Foundation, co-founder at Juno and one of the leaders in the booming cell therapy field, has brought together one of the most prominent teams of scientists tackling cell therapy 2.0 — highlighted by a quest to bridge a daunting tech gap that separates some profound advances in blood cancers with solid tumors. And today he’s officially adding Hal Barron and GlaxoSmithKline as a major league collaborator which is pitching in a large portion of the $600 million he’s raised in the past year to make that vision a reality.

“We’ve being staying stealth,” Klausner tells me, then adding with a chuckle: “and going back to stealth after this.”

“Cell therapy has a lot of challenges,” notes Barron, the R&D chief at GSK, ticking off the resistance put up by solid tumors to cell therapies, the vein-to-vein time involved in taking immune cells out of patients, engineering them to attack cancer cells, and getting them back in, and more. “Over the years Rick and I talked about how it would be wonderful to take that on as a mission.”

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First place fin­ish: Eli Lil­ly just moved to fran­chise leader with their sec­ond mi­graine drug OK in 1 year

In a rare twist for Eli Lilly’s historically slow-moving R&D group, the pharma giant has seized bragging rights to a first-in-class new drug approval. And all signs point to an aggressive marketing followup as they look to outclass some major franchise rivals hobbled by internal dissension.

The FDA came through with an OK for lasmiditan on Friday evening, branding it as Reyvow and lining it up — once a substance classification comes through from the DEA — for a major market release. The oral drug binds to 5-HT1F receptors and is designed to stop an acute migraine after it starts. That makes it a complementary therapy to their CGRP drug Emgality, which has a statistically significant impact on preventing attacks.

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Pfizer, South San Francisco — Jeff Rumans for Endpoints News

Pfiz­er takes aim at a flag­ship fran­chise at Sanofi and Re­gen­eron — and scores a few di­rect hits

Count Pfizer in as a top player in the blockbuster game of JAK1 inhibitors.

Over the weekend the pharma giant posted some stellar Phase III efficacy data for their heavyweight contender abrocitinib in atopic dermatitis (eczema) that lines up ahead of a booming Dupixent (dupilumab), a blockbuster in the portfolios of Regeneron and Sanofi. And they put some real distance ahead of Eli Lilly’s trailing Olumiant, which made a delayed initial arrival on the market for rheumatoid arthritis after the FDA hobbled it with some additional hurdles on safety concerns.

JADE-MONO-1 scores well for Pfizer, teeing up what will be an intensely followed breakdown of the JADE MONO-2 data, which the pharma giant recently top-lined as “similar” to the first Phase III when tested against a placebo — a control group that has been easily outclassed by all the drugs in this market niche.

As of now, Pfizer looks to be equipped to run into the review stage — advantaged by a breakthrough therapy designation that is intended to speed up the regulatory process.

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Allogene HQ Open House on September 17, 2019 in South San Francisco. (Jeff Rumans, Endpoints News)

The next 10 years: Where is biotech head­ed?

The last 10 years have seen a revolution in drug development. Timelines have shortened, particularly in oncology. Regulators have opened up. Investment has skyrocketed. China became a player. Biotechs have multiplied as gene and cell therapy has exploded — offering major new advances in the way diseases are treated, and sometimes cured.

So where are we headed from here? I journeyed out to San Francisco in September to discuss the answer to that question at Allogene’s open house. If the last 10 years have been an eye-opener, what does the next decade hold in store?

Patrick Mahaffy, Getty Images

Court green-lights Clo­vis case af­ter de­tail­ing ev­i­dence the board ‘ig­nored red flags’ on false safe­ty and ef­fi­ca­cy da­ta

Clovis investors have cleared a major hurdle in their long-running case against the board of directors, with a Delaware court making a rare finding that they had a strong enough case against the board to proceed with the action.

In a detailed ruling at the beginning of the month that’s been getting careful scrutiny at firms specializing in biotech and corporate governance, the Delaware Court of Chancery found that the attorneys for the investors had made a careful case that the board — a collection of experts that includes high-profile biotech entrepreneurs, a Harvard professor and well-known investigator as well as Clovis CEO Patrick Mahaffy — repeatedly ignored obvious warnings that Mahaffy’s executive crew was touting inflated, unconfirmed data for their big drug Roci. Serious safety issues were also reportedly overlooked while the company continued a fundraising campaign that brought in more than a half-billion dollars. And that leaves the board open to claims related to their role in the fiasco.

The bottom line:

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Bill Gates backs Gink­go Biowork­s' $350M raise to fu­el the buzzy syn­thet­ic bi­ol­o­gy 'rev­o­lu­tion'

If you want to understand Ginkgo Bioworks, the name should suffice: Bioworks, a spin off “ironworks,” that old industrial linchpin devoted to leveraging scale as a wellspring for vast new industries capable of remaking society. Ginkgo wants to be the ironworks for the revolution it’s heralded with as much fanfare as they can, playing off of one of the buzziest technologies in biotech.

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UCB bags a ri­val to Soliris in $2.1B buy­out deal — but will an in­creas­ing­ly vig­i­lant FTC sign off?

UCB is buying out Ra Pharma $RARX, announcing an acquisition deal that rings up at $48 a share, or $2.1 billion net of cash, and puts them toe-to-toe with Alexion on a clinical showdown.

Ra shares closed at $22.70 on Wednesday.

There’s a small pipeline in play at Ra, but UCB is going for the lead drug — a C5 inhibitor called zilucoplan in Phase III for myasthenia gravis (MG) looking to play rival to Alexion’s Soliris. Soliris has the market advantage, though, with a much earlier approval in MG in late 2017 that UCB feels confident in challenging.

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A new play­er is tak­ing the field in a push for a he­mo­phil­ia A gene ther­a­py, and it’s a big one

BioMarin, the execs at Spark (and buyer-to-be Roche) as well as the Sangamo/Pfizer team have a new rival striding onto the hemophilia block. And it’s a big one.

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Stuck with a PhI­II gene ther­a­py fail­ure at 96 weeks, Gen­Sight prefers the up­beat as­sess­ment

Two years after treatment, the best thing that GenSight Biologics $SIGHT can say about their gene therapy for vision-destroying cases of Leber Hereditary Optic Neuropathy is that it’s just a bit better than a placebo — just maybe because one treatment can cover both eyes.

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