FDA press­es its cam­paign against rogue re­gen­er­a­tive med play­ers, cites Stem­Genex for il­le­gal sales

The FDA’s cam­paign against the “bad ac­tors” op­er­at­ing in re­gen­er­a­tive med­i­cine is far from com­plete.

The reg­u­la­tor on Tues­day ad­mon­ished San Diego-based Stem­Genex Bi­o­log­ic Lab­o­ra­to­ries LLC for mar­ket­ing an un­ap­proved stem cell prod­uct and point­ed out sig­nif­i­cant vi­o­la­tions that could cor­rupt the man­u­fac­tur­ing process, po­ten­tial­ly cul­mi­nat­ing in mi­cro­bial con­t­a­m­i­na­tion that put pa­tients at risk.

The crack­down on clin­ics sell­ing re­gen­er­a­tive med­i­cines for a host of un­ap­proved in­di­ca­tions con­tin­ues to gath­er steam, as the agency makes good on its 2017 promise to en­sure un­scrupu­lous play­ers mar­ket­ing un­proven and of­ten dan­ger­ous prod­ucts are held to ac­count.

The field en­ticed a grow­ing list of com­pa­nies that have “op­por­tunis­ti­cal­ly seized on the clin­i­cal po­ten­tial of re­gen­er­a­tive med­i­cine” to make du­bi­ous claims to pa­tients that of­ten suf­fer from in­cur­able, de­bil­i­tat­ing dis­eases, the agency un­der­scored in a state­ment in Au­gust 2017.


In Stem­Genex’s case, the FDA found the com­pa­ny was pro­cess­ing body fat in­to stro­mal vas­cu­lar frac­tion (SVF) — a cel­lu­lar prod­uct — and “il­le­gal­ly” mar­ket­ing it for se­ri­ous, of­ten life-threat­en­ing dis­eases, in­clud­ing di­a­betes, fi­bromyal­gia, spinal cord in­jury, chron­ic ob­struc­tive pul­monary dis­ease and mus­cu­lar dy­s­tro­phy. And they ad­min­is­ter it by IV, in­hala­tion and di­rect­ly in­to the spinal canal.

This prod­uct, which in­volves more than the min­i­mal ma­nip­u­la­tion of a pa­tient’s adi­pose tis­sue, is reg­u­lat­ed as both a drug and bi­o­log­i­cal prod­uct, but the agency said that no such li­cens­es or ap­provals have been sanc­tioned or ap­plied for.

On the com­pa­ny’s web­site, its pro­pri­etors claim:

Stem­Genex Stem Cell Re­search Cen­tre and af­fil­i­ates do not claim that treat­ment us­ing au­tol­o­gous stem cells are a cure for any con­di­tion, dis­ease, or in­jury. Adi­pose-de­rived stem cell ther­a­py is not a part of FDA ap­proved stem cell ther­a­pies and is not a con­sid­ered a cure for any med­ical con­di­tion. All state­ments and opin­ions pro­vid­ed on this web­site are for ed­u­ca­tion­al and in­for­ma­tion­al pur­pos­es on­ly and we do not di­ag­nose or give med­ical ad­vise via this web­site.

As part of its warn­ing let­ter sent to Stem­Genex on Oc­to­ber 31, FDA al­so list­ed a num­ber of de­vi­a­tions from cur­rent good man­u­fac­tur­ing prac­tice re­quire­ments in the man­u­fac­ture of the SVF prod­uct, in­clud­ing un­val­i­dat­ed man­u­fac­tur­ing process­es and an un­con­trolled en­vi­ron­ment.

The warn­ing let­ter is the lat­est in a slate of warn­ings is­sued by the reg­u­la­tor since last year. Last Jan­u­ary, the FDA slapped a sim­i­lar warn­ing on New Jer­sey-based Amer­i­can CryoStem Corp, in ad­di­tion to warn­ings is­sued to or­ga­ni­za­tions in Flori­da and Cal­i­for­nia in 2017 to ad­dress such prod­ucts be­ing mar­ket­ed.

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive PhI­II for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.

Trump ad­min­is­tra­tion re­vives bid to get drug list prices on TV ads

The Trump administration is not giving up just yet. On Wednesday, the HHS filed an appeal against a judge’s decision in July to overturn a ruling obligating drug manufacturers to disclose the list price of their therapies in television adverts — hours before it was stipulated to go into effect.

In May, the HHS published a final ruling requiring drugmakers to divulge the wholesale acquisition cost— of a 30-day supply of the drug — in tv ads in a bid to enhance price transparency in the United States. The pharmaceutical industry has vehemently opposed the rule, asserting that list prices are not what a typical patient in the United States pays for treatment — that number is typically determined by the type of (or lack thereof) insurance coverage, deductibles and out-of-pocket costs. Although there is truth to that claim, the move was considered symbolic in the Trump administration’s healthcare agenda to hold drugmakers accountable in a climate where skyrocketing drug prices have incensed Americans on both sides of the aisle.