FDA cau­tions over young donor blood trans­fu­sions to treat ag­ing, mem­o­ry loss

In a warn­ing to con­sumers on Tues­day, the FDA said con­sumers should be cau­tious about es­tab­lish­ments of­fer­ing in­fu­sions of plas­ma ob­tained from young hu­man donors with the claim that the in­fused plas­ma will pre­vent ag­ing to mem­o­ry loss.

At a cost of up to thou­sands of dol­lars per in­fu­sion, the FDA said these es­tab­lish­ments, lo­cat­ed in sev­er­al dif­fer­ent states, are cur­rent­ly of­fer­ing in­fu­sions of plas­ma with no proven clin­i­cal ben­e­fit.

For in­stance, a start­up called Am­brosia, which has clin­ics in New York and Cal­i­for­nia, of­fers such treat­ments for $8,000 for one liter and $12,000 for two liters, al­though sci­en­tists called the pay-to-par­tic­i­pate tri­al a scam.

FDA al­so notes es­tab­lish­ments claim­ing that blood trans­fu­sions can pre­vent not on­ly ag­ing or mem­o­ry loss, or for the treat­ment of such con­di­tions as de­men­tia, Parkin­son’s dis­ease, mul­ti­ple scle­ro­sis, Alzheimer’s dis­ease, heart dis­ease, or post-trau­mat­ic stress dis­or­der.

“The dos­ing of these in­fu­sions, which can in­volve large ad­min­is­tered vol­umes, is al­so not guid­ed by ev­i­dence from ad­e­quate and well con­trolled tri­als. In ad­di­tion, the in­fu­sion of plas­ma can be as­so­ci­at­ed with in­fec­tious, al­ler­gic, res­pi­ra­to­ry, and car­dio­vas­cu­lar risks, among oth­ers,” the FDA said. “In some in­di­vid­u­als, par­tic­u­lar­ly those with pre­ex­ist­ing heart dis­ease, the in­fu­sion of plas­ma can cause over­load of the cir­cu­la­to­ry sys­tem lead­ing to swelling of the body and dif­fi­cult breath­ing.”

In a note to health care providers, the agency al­so said the FDA-rec­og­nized Cir­cu­lar of In­for­ma­tion for the Use of Hu­man Blood and Blood Com­po­nents pub­lished by AABB lists the fol­low­ing in­di­ca­tions for the ad­min­is­tra­tion of plas­ma:

  • “Man­age­ment of pre­op­er­a­tive or bleed­ing pa­tients who re­quire re­place­ment of mul­ti­ple plas­ma co­ag­u­la­tion fac­tors (e.g., liv­er dis­ease, dis­sem­i­nat­ed in­travas­cu­lar co­ag­u­la­tion)
  • Pa­tients un­der­go­ing mas­sive trans­fu­sion who have clin­i­cal­ly sig­nif­i­cant co­ag­u­la­tion de­fi­cien­cies
  • Pa­tients tak­ing war­farin who are bleed­ing or need to un­der­go an in­va­sive pro­ce­dure be­fore vi­t­a­min K could re­verse the war­farin ef­fect or who need on­ly tran­sient re­ver­sal of war­farin ef­fect
  • Trans­fu­sion or plas­ma ex­change in pa­tients with throm­bot­ic throm­bo­cy­topenic pur­pu­ra
  • Trans­fu­sion or plas­ma ex­change with se­lect­ed co­ag­u­la­tion fac­tor de­fi­cien­cies, con­gen­i­tal or ac­quired, for which no spe­cif­ic co­ag­u­la­tion con­cen­trates are avail­able
  • Man­age­ment of pa­tients with rare spe­cif­ic plas­ma pro­tein de­fi­cien­cies, when re­com­bi­nant prod­ucts are un­avail­able.”

First pub­lished in Reg­u­la­to­ry Fo­cus™ by the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety, the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care prod­ucts. Click here for more in­for­ma­tion.


Zachary Brennan

managing editor, RAPS

Seer adds ex-FDA chief Mark Mc­Clel­lan to the board; Her­cules Cap­i­tal makes it of­fi­cial for new CEO Scott Bluestein

→ On the same day it announced a $17.5 million Series C, life sciences and health data company Seer unveiled that it had lured former FDA commissioner and ex-CMS administrator Mark McClellan on to its board. “Mark’s deep understanding of the health care ecosystem and visionary insights on policy reform will be crucial in informing our thinking as we work to bring our liquid biopsy and life sciences products to market,” said Seer chief and founder Omid Farokhzad in a statement.

Daniel O'Day

No­var­tis hands off 3 pre­clin­i­cal pro­grams to the an­tivi­ral R&D mas­ters at Gilead

Gilead CEO Daniel O’Day’s new task hunting up a CSO for the company isn’t stopping the industry’s dominant antiviral player from doing pipeline deals.

The big biotech today snapped up 3 preclinical antiviral programs from pharma giant Novartis, with drugs promising to treat human rhinovirus, influenza and herpes viruses. We don’t know what the upfront is, but the back end has $291 million in milestones baked in.

Vas Narasimhan, AP Images

On a hot streak, No­var­tis ex­ecs run the odds on their two most im­por­tant PhI­II read­outs. Which is 0.01% more like­ly to suc­ceed?

Novartis CEO Vas Narasimhan is living in the sweet spot right now.

The numbers are running a bit better than expected, the pipeline — which he assembled as development chief — is performing and the stock popped more than 4% on Thursday as the executive team ran through their assessment of Q2 performance.

Year-to-date the stock is up 28%, so the investors will be beaming. Anyone looking for chinks in their armor — and there are plenty giving it a shot — right now focus on payer acceptance of their $2.1 million gene therapy Zolgensma, where it’s early days. And CAR-T continues to underperform, but Novartis doesn’t appear to be suffering from it.

So what could go wrong?

Actually, not much. But Tim Anderson at Wolfe pressed Narasimhan and his development chief John Tsai to pick which of two looming Phase III readouts with blockbuster implication had the better odds of success.

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Norbert Bischofberger. Kronos

Backed by some of the biggest names in biotech, Nor­bert Bischof­berg­er gets his megaround for plat­form tech out of MIT

A little over a year ago when I reported on Norbert Bischofberger’s jump from the CSO job at giant Gilead to a tiny upstart called Kronos, I noted that with his connections in biotech finance, that $18 million launch round he was starting off with could just as easily have been $100 million or more.

With his first anniversary now behind him, Bischofberger has that mega-round in the bank.

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Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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Novotech CRO Ex­pands Chi­na Team as Biotech De­mand for Clin­i­cal Tri­als In­creas­es up to 79%

An increase in demand of up to 79% for clinical trials in China has prompted Novotech the Asia-Pacific CRO to rapidly expand the China team, appointing expert local clinical executives to their Shanghai and Hong Kong offices. The company is planning to expand their team by 30% over the next quarter.

Novotech China has seen considerable demand recently which is borne out by research from GlobalData:
A global migration of clinical research is occurring from high-income countries to low and middle-income countries with emerging economies. Over the period 2017 to 2018, for example, the number of clinical trial sites opened by biotech companies in Asia-Pacific increased by 35% compared to 8% in the rest of the world, with growth as high as 79% in China.
Novotech CEO Dr John Moller said China offers the largest population in the world, rapid economic growth, and an increasing willingness by government to invest in research and development.
Novotech’s 23 years of experience working in the region means we are the ideal CRO partner for USA biotechs wanting to tap the research expertise and opportunities that China offers.
There are over 22,000 active investigators in Greater China, with about 5,000 investigators with experience on at least 3 studies (source GlobalData).

On a glob­al romp, Boehringer BD team picks up its third R&D al­liance for Ju­ly — this time fo­cused on IPF with $50M up­front

Boehringer Ingelheim’s BD team is on a global deal spree. The German pharma company just wrapped its third deal in 3 weeks, going back to Korea for its latest pipeline pact — this time focused on idiopathic pulmonary fibrosis.

They’re handing over $50 million to get their hands on BBT-877, an ATX inhibitor from Korea’s Bridge Biotherapeutics that was on display at a science conference in Dallas recently. There’s not a whole lot of data to evaluate the prospects here.

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Servi­er scoots out of an­oth­er col­lab­o­ra­tion with Macro­Gen­ics, writ­ing off their $40M

Servier is walking out on a partnership with MacroGenics $MGNX — for the second time.

After the market closed on Wednesday MacroGenics put out word that Servier is severing a deal — inked close to 7 years ago — to collaborate on the development of flotetuzumab and other Dual-Affinity Re-Targeting (DART) drugs in its pipeline.

MacroGenics CEO Scott Koenig shrugged off the departure of Servier, which paid $20 million to kick off the alliance and $20 million to option flotetuzumab — putting a heavily back-ended $1 billion-plus in additional biobuck money on the table for the anti-CD123/CD3 bispecific and its companion therapies.