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.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

Editor’s note: This is a live story and will be updated.

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

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Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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See­los Ther­a­peu­tics 'tem­porar­i­ly' stops study in rare neu­ro dis­or­der for busi­ness rea­sons

Microcap biotech Seelos Therapeutics is halting enrollment of its study in spinocerebellar ataxia type 3 (also known as Machado-Joseph disease) because of “financial considerations,” and in order to focus on other studies, the company said today, adding that the pause would be temporary.

The study will continue with the patients who have already enrolled, and the data from them will be used to decide whether to continue enrolling others in the future.

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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CSL CEO Paul McKenzie (L) and CMO Bill Mezzanotte

Q&A: New­ly-mint­ed CSL chief ex­ec­u­tive Paul McKen­zie and chief med­ical of­fi­cer Bill Mez­zan­otte

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.

Boehringer re­ports ro­bust sales led by type 2 di­a­betes and pul­monary drugs, promis­es more to come high­light­ing obe­si­ty

Boehringer Ingelheim reported human pharma sales of €18.5 billion on Wednesday, led by type 2 diabetes and heart failure drug Jardiance and pulmonary fibrosis med Ofev. Jardiance sales reached €5.8 billion, growing 39% year over year, while Ofev took in €3.2 billion, notching its own 20.6% annual jump.

However, Boehringer is also looking ahead with its pipeline, estimating “In the next seven years the company expects about 20 regulatory approvals in human pharma.”

FDA ap­proves Nar­can opi­oid over­dose re­ver­sal spray for over-the-counter sale

The FDA today approved Emergent BioSolutions’ Narcan brand naloxone nasal spray for over-the-counter sales. The nod was expected and comes on the heels of a unanimous 19-0 advisory committee vote in favor of approval last month.

The move to OTC means the opioid overdose reversal agent will now be available on grocery, convenience and gas stations shelves, as well as potentially for purchase online.

J&J bows out of RSV vac­cine race, end­ing PhI­II study and ced­ing to Pfiz­er, GSK

Johnson & Johnson announced Wednesday morning it is ending development of its adult RSV vaccine that was in the middle of a 27,200-patient trial, giving up a big slice of what’s expected to be the next multibillion-dollar pharma market.

The decision came down to the shifting RSV “competitive landscape,” a company spokesperson tells Endpoints News, adding the “breadth of options” was much different than when J&J first started its pivotal study. The spokesperson declined to comment on the Phase III data, saying only the shot is undergoing an “ongoing assessment.”

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