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.

UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Stephen Hahn, AP

The FDA has de­val­ued the gold stan­dard on R&D. And that threat­ens every­one in drug de­vel­op­ment

Bioregnum Opinion Column by John Carroll

A few weeks ago, when Stephen Hahn was being lightly queried by Senators in his confirmation hearing as the new commissioner of the FDA, he made the usual vow to maintain the gold standard in drug development.

Neatly summarized, that standard requires the agency to sign off on clinical data — usually from two, well-controlled human studies — that prove a drug’s benefit outweighs any risks.

Over the last few years, biopharma has enjoyed an unprecedented loosening over just what it takes to clear that bar. Regulators are more willing to drop the second trial requirement ahead of an accelerated approval — particularly if they have an unmet medical need where patients are clamoring for a therapy.

That confirmatory trial the FDA demands can wait a few years. And most everyone in biopharma would tell you that’s the right thing for patients. They know its a tonic for everyone in the industry faced with pushing a drug through clinical development. And it’s helped inspire a global biotech boom.

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UP­DAT­ED: New play­ers are jump­ing in­to the scram­ble to de­vel­op a vac­cine as pan­dem­ic pan­ic spreads fast

When the CNN news crew in Wuhan caught wind of the Chinese government’s plan to quarantine the city of 11 million people, they made a run for one of the last trains out — their Atlanta colleagues urging them on. On the way to the train station, they were forced to skirt the local seafood market, where the coronavirus at the heart of a brewing outbreak may have taken root.

And they breathlessly reported every moment of the early morning dash.

In shuttering the city, triggering an exodus of masked residents who caught wind of the quarantine ahead of time, China signaled that they were prepared to take extreme actions to stop the spread of a virus that has claimed 17 lives, sickened many more and panicked people around the globe.

CNN helped illustrate how hard all that can be.

The early reaction in the biotech industry has been classic, with small-cap companies scrambling to headline efforts to step in fast. But there are also new players in the field with new tech that has been introduced since the last of a series of pandemic panics that could change the usual storylines. And they’re volunteering for a crash course in speeding up vaccine development — a field where overnight solutions have been impossible to prove.

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Mer­ck KGaA spin­out gets first fund­ing to bring dual-act­ing can­cer mol­e­cules in­to the clin­ic

Two and a half years after launch, Merck KGaA spinout iOnctura is getting its first major round of funding.

The oncology startup raised €15 million ($16.6 million) to put its lead drug into the clinic and get its second drug past IND-enabling tests. INKEF Capital and VI Partners co-led the round and were joined by the biotech’s longtime backer M Ventures, an arm of Merck KGaA, and Schroder Adveq.

Eli Lil­ly’s $1.6B can­cer drug failed to spark even the slight­est pos­i­tive gain for pa­tients in its 1st PhI­II

Eli Lilly had high hopes for its pegylated IL-10 drug pegilodecakin when it bought Armo last year for $1.6 billion in cash. But after reporting a few months ago that it had failed a Phase III in pancreatic cancer, without the data, its likely value has plunged. And now we’re getting some exact data that underscore just how little positive effect it had.

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Am­gen aug­ments Asia foothold by tak­ing over Astel­las joint ven­ture in Japan

California-based Amgen, which does the bulk of its business in the United States, made its ambition to reinvigorate its growth prospects by expanding its presence in Asia clear at the sidelines of the JP Morgan healthcare conference in San Francisco earlier this month.

The Thousand Oaks-based company on Thursday executed its plan to dissolve the joint venture with Astellas — created in 2013 — to operate the unit independently in Japan. With its rapidly aging population, the region represents an appealing market for Amgen’s osteoporosis treatments Prolia and Evenity as well as a cholesterol-lowering injection Repatha.

Daphne Zohar (PureTech)

PureTech bags $200M from sale of Karuna shares — still siz­zling from promis­ing schiz­o­phre­nia da­ta

Cashing in on the exuberance around Karuna Therapeutics and its potential blockbuster CNS drug, PureTech has sold a chunk of the biotech’s shares to Goldman Sachs for $200 million.

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