Got­tlieb, Marks de­tail plans to ad­vance de­vel­op­ment of cell and gene ther­a­pies

FDA Com­mis­sion­er Scott Got­tlieb and Cen­ter for Bi­o­log­ics Eval­u­a­tion and Re­search (CBER) Di­rec­tor Pe­ter Marks on Tues­day de­tailed plans for the agency to keep pace with an ex­pect­ed in­flux of ap­pli­ca­tions for cell and gene ther­a­pies over the com­ing years.

“The FDA is wit­ness­ing a surge of cell and gene ther­a­py prod­ucts en­ter­ing ear­ly de­vel­op­ment, ev­i­denced by a large up­swing in the num­ber of in­ves­ti­ga­tion­al new drug (IND) ap­pli­ca­tions,” Got­tlieb and Marks say.

Scott Got­tlieb

By 2020, Got­tlieb and Marks say they ex­pect to be re­ceiv­ing up­wards of 200 INDs for cell and gene ther­a­pies each year, adding to the 800 ac­tive INDs for such prod­ucts al­ready filed with the agency. By 2025, they pre­dict the agency will be ap­prov­ing be­tween 10 and 20 cell and gene ther­a­py prod­ucts an­nu­al­ly.

The two FDA lead­ers draw a com­par­i­son be­tween the cur­rent land­scape for cell and gene ther­a­pies to the pro­lif­er­a­tion of an­ti­body drugs in the late 1990s once plat­forms for cre­at­ing an­ti­bod­ies that wouldn’t be re­ject­ed by a pa­tient’s im­mune sys­tem were wide­ly avail­able.

“In this case, it was the ad­vent of safe and ef­fec­tive vec­tors for the de­liv­ery of gene ther­a­py prod­ucts, such as the adop­tion of ade­no-as­so­ci­at­ed virus (AAV) vec­tors,” Got­tlieb and Marks say.

Pe­ter Marks

To pro­mote the con­tin­ued de­vel­op­ment of new cell and gene ther­a­pies, Got­tlieb and Marks say that the FDA will take a num­ber of steps, in­clud­ing adding as many as 50 ad­di­tion­al re­view­ers to its staff, lever­ag­ing ex­pe­dit­ed path­ways, is­su­ing new guid­ance and tak­ing en­force­ment ac­tion against clin­ics that fail to com­ply with FDA reg­u­la­tions.

Ac­cord­ing to Got­tlieb and Marks, ac­cel­er­at­ed ap­proval is par­tic­u­lar­ly rel­e­vant for gene ther­a­pies as the agency can man­date post­mar­ket stud­ies to mea­sure the dura­bil­i­ty of the treat­ment and long-term safe­ty is­sues that would be dif­fi­cult to ad­dress in the pre­mar­ket set­ting.

As for new guid­ance, Got­tlieb and Marks say the agency will de­vel­op a se­ries of clin­i­cal guid­ance doc­u­ments for dif­fer­ent ar­eas of prod­uct de­vel­op­ment, such as gene ther­a­pies for in­her­it­ed blood dis­or­ders and neu­rode­gen­er­a­tive dis­eases.

In some cas­es, Got­tlieb and Marks say that a more tra­di­tion­al ap­proach to drug de­vel­op­ment may be war­rant­ed for cer­tain gene ther­a­pies, such as those in­tend­ed to ad­dress the symp­toms of a neu­rode­gen­er­a­tive dis­ease or tar­get an “ex­pres­sion of a pro­tein or en­zyme be­lieved to play a role in the ad­vance of the dis­ease.”

In an­oth­er planned guid­ance, Got­tlieb and Marks say the agency will tack­le the man­u­fac­tur­ing com­plex­i­ties of cell-based gene ther­a­pies, such as chimeric anti­gen re­cep­tor (CAR) T-cell ther­a­pies, in a fu­ture guid­ance.

“The guid­ance that we in­tend to is­sue will pro­mote a bet­ter un­der­stand­ing of the crit­i­cal qual­i­ty at­trib­ut­es and oth­er fac­tors re­lat­ed to prod­uct man­u­fac­tur­ing,” they write, adding that one of their goals will be to min­i­mize the need for bridg­ing stud­ies for mi­nor man­u­fac­tur­ing changes for cell ther­a­pies.

The two al­so say that the FDA will is­sue a guid­ance lat­er this year out­lin­ing how re­searchers can “pool their clin­i­cal da­ta af­ter fol­low­ing a com­mon man­u­fac­tur­ing pro­to­col, and there­by de­vel­op a more ro­bust da­ta set for pur­pos­es of gain­ing a [Bi­o­log­ics Li­cense Ap­pli­ca­tion] BLA.”


First pub­lished here. Reg­u­la­to­ry Fo­cus is the flag­ship on­line pub­li­ca­tion of the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety (RAPS), the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care and re­lat­ed prod­ucts, in­clud­ing med­ical de­vices, phar­ma­ceu­ti­cals, bi­o­log­ics and nu­tri­tion­al prod­ucts. Email news@raps.org for more in­for­ma­tion.

Susan Galbraith, AstraZeneca EVP, oncology R&D, at EUBIO22 (Rachel Kiki for Endpoints News)

Up­dat­ed: As­traZeneca jumps deep­er in­to cell ther­a­py 2.0 space with $320M biotech M&A

Right from the start, the execs at Neogene had some lofty goals in mind when they decided to try their hand at a cell therapy that could tackle solid tumors.

Its founders have helped hone a new approach that would pack in multiple neoantigen targets to create a personalized TCR treatment that would not just make the leap from blood to solid tumors, but do it with durability. And they managed to make their way rapidly to the clinic, unveiling their first Phase I program for advanced tumors just last May.

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Ei­sai’s ex­pand­ed Alzheimer’s da­ta leave open ques­tions about safe­ty and clin­i­cal ben­e­fit

Researchers still have key questions about Eisai’s investigational Alzheimer’s drug lecanemab following the publication of more Phase III data in the New England Journal of Medicine Tuesday night.

In the paper, which was released in conjunction with presentations at an Alzheimer’s conference, trial investigators write that a definition of clinical meaningfulness “has not been established.” And the relative lack of new information, following topline data unveiled in September, left experts asking for more — setting up a potential showdown to precisely define how big a difference the drug makes in patients’ lives.

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Illustration: Assistant Editor Kathy Wong for Endpoints News

Twit­ter dis­ar­ray con­tin­ues as phar­ma ad­ver­tis­ers ex­tend paus­es and look around for op­tions, but keep tweet­ing

Pharma advertisers on Twitter are done — at least for now. Ad spending among the previous top spenders flattened even further last week, according to the latest data from ad tracker Pathmatics, amid ongoing turmoil after billionaire boss Elon Musk’s takeover now one month ago.

Among 18 top advertisers tracked for Endpoints News, only two are spending: GSK and Bayer. GSK spending for the full week through Sunday was minimal at just under $1,900. Meanwhile, German drugmaker Bayer remains the industry outlier upping its spending to $499,000 last week from $480,000 the previous week. Bayer’s spending also marks a big increase from a month ago and before the Musk takeover, when it spent $16,000 per week.

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Vi­a­tris with­draws ac­cel­er­at­ed ap­proval for top­i­cal an­timi­cro­bial 24 years lat­er

After 24 years without confirming clinical benefit, the FDA announced Tuesday morning that Viatris (formed via Mylan and Pfizer’s Upjohn) has decided to withdraw a topical antimicrobial agent, Sulfamylon (mafenide acetate), after the company said conducting a confirmatory study was not feasible.

Sulfamylon first won FDA’s accelerated nod in 1998 as a topical burn treatment, with the FDA noting that last December, Mylan told the agency that it wasn’t running the trial.

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Catal­ent to cut about 200 jobs in Mary­land and Texas

Contract manufacturing company Catalent is cutting about 200 jobs in Maryland and Texas, according to WARN notices, trimming back some of its pandemic-era expansion.

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Mar­ket­ingRx roundup: Pfiz­er, BioN­Tech re-up iHeartRa­dio hol­i­day spon­sor­ship; WHO re­names mon­key­pox to 'm­pox'

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Mark Schneider, Nestlé CEO (AP Images)

Nestlé re­con­sid­ers peanut al­ler­gy pro­gram two years af­ter $2.6B buy­out

It seems Nestlé is experiencing some buyer’s remorse two years after throwing down $2.6 billion for Aimmune Therapeutics and its peanut allergy pill Palforzia.

CEO Mark Schneider announced on Tuesday that Nestlé is “exploring strategic options” for Palforzia following lower-than-expected demand. A company spokesperson declined to confirm whether a potential sale is in consideration.

“The review is expected to be completed in the first half of 2023. Going forward, Nestlé Health Science will sharpen its focus on Consumer Care and Medical Nutrition,” the company said in a news release.

Bris­tol My­ers scraps gene ther­a­py deal with uniQure for car­dio­vas­cu­lar dis­eases

Bristol Myers Squibb is hitting the exit on a collaboration with a gene therapy biotech.

The Big Pharma company will no longer partner with uniQure on finding new treatments for cardiovascular diseases, the biotech reported to the SEC last week, following a rocky relationship that saw the pair break off an earlier agreement — before coming back to the table. The deal will officially terminate on Feb. 21, 2023.

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iECURE CEO Joe Truitt and founder Jim Wilson

Jim Wil­son biotech iECURE gets fresh $65M to push pe­di­atric liv­er dis­ease gene ther­a­py in­to the clin­ic

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This round was co-led by Novo Holdings and LYFE Capital, followed by initial investors Versant and OrbiMed as well. In September 2021, iECURE raised a $50 million Series A led by the latter two. The new cash infusion will get iECURE through an initial in-human trial, which CEO Joe Truitt told Endpoints News iECURE hopes to read out in 2024.

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