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.

UP­DAT­ED: Chica­go biotech ar­gues blue­bird, Third Rock 'killed' its ri­val, pi­o­neer­ing tha­lassemia gene ther­a­py in law­suit

Blue­bird bio $BLUE chief Nick Leschly court­ed con­tro­ver­sy last week when he re­vealed the com­pa­ny’s be­ta tha­lassemia treat­ment will car­ry a jaw-drop­ping $1.8 mil­lion price tag over a 5-year pe­ri­od in Eu­rope — mak­ing it the plan­et’s sec­ond most ex­pen­sive ther­a­py be­hind No­var­tis’ $NVS fresh­ly ap­proved spinal mus­cu­lar at­ro­phy ther­a­py, Zol­gens­ma, at $2.1 mil­lion. A Chica­go biotech, mean­while, has been fum­ing at the side­lines. In a law­suit filed ear­li­er this month, Er­rant Gene Ther­a­peu­tics al­leged that blue­bird and ven­ture cap­i­tal group Third Rock un­law­ful­ly prised a vi­ral vec­tor, de­vel­oped in part­ner­ship with the Memo­r­i­al Sloan Ket­ter­ing Can­cer Cen­ter (MSK), from its grasp, and thwart­ed the de­vel­op­ment of its sem­i­nal gene ther­a­py.

The top 10 block­buster drugs in the late-stage pipeline — Eval­u­ate adds 6 new ther­a­pies to heavy-hit­ter list

Vertex comes in for a substantial amount of criticism for its no-holds-barred tactical approach toward wresting the price it wants for its commercial drugs in Europe. But the flip side of that coin is a highly admired R&D and commercial operation that regularly wins kudos from analysts for their ability to engineer greater cash flow from the breakthrough drugs they create.

Both aspects needed for success in this business are on display in the program backing Vertex’s triple for cystic fibrosis. VX-659/VX-445 + Tezacaftor + Ivacaftor — it’s been whittled down to 445 now — was singled out by Evaluate Pharma as the late-stage therapy most likely to win the crown for drug sales in 5 years, with a projected peak revenue forecast of $4.3 billion.

The latest annual list, which you can see here in their latest world preview, includes a roster of some of the most closely watched development programs in biopharma. And Evaluate has added 6 must-watch experimental drugs to the top 10 as drugs fail or go on to a first approval. With apologies to the list maker, I revamped this to rank the top 10 by projected 2024 sales, instead of Evaluate's net present value rankings.

It's how we roll at Endpoints News.

Here is a quick summary of the rest of the top 10:

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Arc­turus ex­pands col­lab­o­ra­tion, adding $30M cash; Ku­ra shoots for $100M raise

→  Rare dis­ease play­er Ul­tragenyx $RARE is ex­pand­ing its al­liance with Arc­turus $ARCT, pay­ing $24 mil­lion for eq­ui­ty and an­oth­er $6 mil­lion in an up­front as the two part­ners ex­pand their col­lab­o­ra­tion to in­clude up to 12 tar­gets. “This ex­pand­ed col­lab­o­ra­tion fur­ther so­lid­i­fies our mR­NA plat­form by adding ad­di­tion­al tar­gets and ex­pand­ing our abil­i­ty to po­ten­tial­ly treat more dis­eases,” said Emil Kakkis, the CEO at Ul­tragenyx. “We are pleased with the progress of our on­go­ing col­lab­o­ra­tion. Our most ad­vanced mR­NA pro­gram, UX053 for the treat­ment of Glyco­gen Stor­age Dis­ease Type III, is ex­pect­ed to move in­to the clin­ic next year, and we look for­ward to fur­ther build­ing up­on the ini­tial suc­cess of this part­ner­ship.”

Neil Woodford. Woodford Investment Management via YouTube

Wood­ford braces po­lit­i­cal storm as UK fi­nan­cial reg­u­la­tors scru­ti­nize fund sus­pen­sion

The shock of Neil Wood­ford’s de­ci­sion to block with­drawals for his flag­ship fund is still rip­pling through the rest of his port­fo­lio — and be­yond. Un­der po­lit­i­cal pres­sure, UK fi­nan­cial reg­u­la­tors are now tak­ing a hard look while in­vestors con­tin­ue to flee.

In a re­sponse let­ter to an MP, the Fi­nan­cial Con­duct Au­thor­i­ty re­vealed that it’s opened an in­ves­ti­ga­tion in­to the sus­pen­sion fol­low­ing months of en­gage­ment with Link Fund So­lu­tions, which tech­ni­cal­ly del­e­gat­ed Wood­ford’s firm to man­age its funds.

John Chiminski, Catalent CEO - File Photo

'It's a growth play': Catal­ent ac­quires Bris­tol-My­er­s' Eu­ro­pean launch pad, ex­pand­ing glob­al CD­MO ops

Catalent is staying on the growth track.

Just two months after committing $1.2 billion to pick up Paragon and take a deep dive into the sizzling hot gene therapy manufacturing sector, the CDMO is bouncing right back with a deal to buy out Bristol-Myers’ central launchpad for new therapies in Europe, acquiring a complex in Anagni, Italy, southwest of Rome, that will significantly expand its capacity on the continent.

There are no terms being offered, but this is no small deal. The Anagni campus employs some 700 staffers, and Catalent is planning to go right in — once the deal closes late this year — with a blueprint to build up the operations further as they expand on oral solid, biologics, and sterile product manufacturing and packaging.

This is an uncommon deal, Catalent CEO John Chiminski tells me. But it offers a shortcut for rapid growth that cuts years out of developing a green fields project. That’s time Catalent doesn’t have as the industry undergoes unprecedented expansion around the world.

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Gilead baits new al­liance with $45M up­front, div­ing in­to the busy pro­tein degra­da­tion field

Gilead is jump­ing on board the pro­tein degra­da­tion band­wag­on. And they’re turn­ing to a low-pro­file Third Rock start­up for the ex­per­tise. But if you were look­ing for a trans­for­ma­tion­al deal to kick up fresh en­thu­si­asm for Gilead, you’ll have to re­main pa­tient.

This one will have a long way to go be­fore they get in­to the clin­ic.

The big biotech said Wednes­day morn­ing that it is pay­ing $45 mil­lion up­front and re­serv­ing a whop­ping $2.3 bil­lion in biotech bucks if San Fran­cis­co-based Nurix can point the way to new can­cer ther­a­pies, as well as drugs for oth­er, un­spec­i­fied dis­eases.

A new num­ber 1 drug? Keytru­da tapped to top the 10 biggest block­busters on the world stage by 2024

Analysts may be fretting about Keytruda’s longterm prospects as a host of rival therapies elbow their way to the market. But the folks at Evaluate Pharma are confident that last year’s $7 billion earner is headed for glory, tapping it to beat out the current #1 therapy Humira as AbbVie watches that franchise swoon over the next 5 years.

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In­vestor day prep at Mer­ck in­cludes a new strat­e­gy to pick up the pace on M&A — re­port

Mer­ck’s re­cent deals to buy up two bolt-on biotechs — Ti­los and Pelo­ton — weren’t an aber­ra­tion. In­stead, both ac­qui­si­tions mark a new strat­e­gy to beef up its dom­i­nant can­cer drug op­er­a­tions cen­tered on Keytru­da while look­ing to ad­dress grow­ing con­cerns that too many of its eggs are in the one I/O bas­ket for their PD-1 pro­gram. And Mer­ck is go­ing af­ter more small- and mid-sized buy­outs to calm those fears.

Dave Barrett, Brian Chee, Amir Nashat, Amy Schulman. Polaris

Bob Langer's first port of call — Po­laris Part­ners — maps $400M for ninth fund

Health and tech ven­ture group Po­laris Part­ners, which counts Alec­tor, Al­ny­lam and Ed­i­tas Med­i­cine as part of its port­fo­lio, is set­ting up its ninth fund, rough­ly two years af­ter it closed Po­laris VI­II with $435 mil­lion in the bank, sur­pass­ing its tar­get by $35 mil­lion.

The Boston-based firm, in an SEC fil­ing, said it in­tends to raise $400 mil­lion for the fund. Po­laris — which rou­tine­ly backs com­pa­nies mold­ed out of the work done in the lab of pro­lif­ic sci­en­tist Bob Langer of MIT  — typ­i­cal­ly in­vests ear­ly, and sticks around till com­pa­nies are in the green. Like its peers at Flag­ship and Third Rock, Po­laris is all about cham­pi­oning the lo­cal biotech scene with a steady flow of start­up cash.