Re­al-time re­views for cell and gene ther­a­pies? Pfiz­er to FDA: Yes, please

The FDA’s On­col­o­gy Cen­ter of Ex­cel­lence has a pro­gram, known as Re­al-Time On­col­o­gy Re­view (RTOR), that speeds up can­cer drug ap­provals, some­times by months, but has al­ways been re­strict­ed to can­cer drugs — first with sup­ple­ments and then mov­ing on to full ap­provals.

How RTOR works: Ear­li­er sub­mis­sions of topline ef­fi­ca­cy and safe­ty re­sults from spon­sors, pri­or to the sub­mis­sion of the com­plete ap­pli­ca­tion, help to sup­port an ear­li­er start to the FDA’s eval­u­a­tion of the com­plete ap­pli­ca­tion.

In draft guid­ance from this sum­mer on RTOR, how­ev­er, the agency qui­et­ly (via a foot­note) men­tioned that:

RTOR may not be suit­able for cer­tain bi­o­log­i­cal prod­ucts, such as cell and gene ther­a­pies, for which com­plex man­u­fac­tur­ing and prod­uct char­ac­ter­is­tics need to be con­sid­ered in eval­u­at­ing the safe­ty and ef­fi­ca­cy of the prod­uct. For these types of prod­ucts, we rec­om­mend that a dis­cus­sion of whether the prod­uct is suit­able for RTOR take place with the ap­pro­pri­ate re­view di­vi­sion.

In com­ments on the draft re­leased ear­li­er this month, Pfiz­er point­ed to the foot­note and said it is en­cour­aged that the FDA “is di­rect­ing spon­sors to dis­cuss the po­ten­tial for us­ing RTOR for cell and gene ther­a­pies with the ap­pro­pri­ate re­view di­vi­sion. Pfiz­er sup­ports the use of RTOR on these new and promis­ing ar­eas of drug de­vel­op­ment where it has the po­ten­tial to stream­line fu­ture re­views for the ben­e­fits of pa­tients.”

But the Al­liance for Re­gen­er­a­tive Med­i­cine said its mem­ber com­pa­nies “en­cour­age the Agency not to cre­ate a dif­fer­ent stan­dard for cell and gene ther­a­pies (CGT) but in­stead con­sid­er, and pro­vide ad­vice on, the ex­tent of CMC in­for­ma­tion that will need to be avail­able at each of the par­tial sub­mis­sions al­lowed in RTOR pro­gram to al­low it to be rou­tine­ly suit­able to CGTs. We sug­gest that the re­view process­es ap­plied for Kym­ri­ah for DL­B­CL, Zol­gens­ma, and Evrys­di may be use­ful in es­tab­lish­ing a base­line.”

RTOR in­spired an up­com­ing and high­ly an­tic­i­pat­ed PDU­FA VII pi­lot, known as the Split-Time Ap­pli­ca­tion Re­view (STAR) pi­lot, which will be ap­plic­a­ble to all ther­a­peu­tic ar­eas. PhRMA said in its com­ments on the draft that it en­cour­ages the FDA “to con­sid­er and share its think­ing re­gard­ing the in­ter­play be­tween the RTOR and STAR pro­grams.” More de­tails will like­ly be com­ing soon on the STAR pi­lot too, which looks to be sim­i­lar to the break­through des­ig­na­tion pro­gram.

The in­dus­try lob­by­ing group al­so called for hu­man cell and tis­sue prod­ucts re­viewed by CBER, as well as prod­ucts uti­liz­ing dig­i­tal health tech­nolo­gies to be con­sid­ered for RTOR.

The push for ex­pand­ing the pro­gram comes as re­cent stud­ies have shown that al­though RTOR does not guar­an­tee an ear­li­er ac­tion date, three out of the four cas­es in one study were ap­proved months ear­li­er than the PDU­FA date, “2.8, 3.0, and 4.1 months for Piqray, Qin­lock, and Tukysa, re­spec­tive­ly.”

Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; and more

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Paul Perreault, CSL Behring CEO

CSL lands FDA ap­proval for he­mo­phil­ia B gene ther­a­py, sets $3.5M list price

The FDA has approved the world’s first gene therapy for hemophilia B, ushering into the market a treatment that’s historic in both what it promises to do and how much it will cost.

CSL will be marketing the drug, Hemgenix, at a list price of $3.5 million — which sets a new record for the most expensive single-use gene therapy in the US.

In a statement provided to Endpoints News, the Australian company noted that the current costs of treating people with moderate to severe hemophilia B can be significant over a lifetime. By some estimates, healthcare systems could spend more than $20 million per person.

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Image: Shutterstock

MIT re­searchers re­veal DNA "Paste" tech be­hind lat­est gene edit­ing start­up

MIT scientists have developed a tool that they say can insert large gene sequences where they want in the genome.

In a paper published Thursday in Nature Biotechnology, MIT fellows Omar Abudayyeh, Jonathan Gootenberg and colleagues detail a technology they call PASTE, which they say can potentially be used to insert long strands of DNA and treat genetic diseases caused by many different mutations, such as cystic fibrosis and Leber congenital amaurosis, a rare eye disorder that causes blindness.

Elon Musk (GDA via AP Images)

Biggest drug com­pa­nies halt­ed Twit­ter ad buys af­ter Lil­ly in­sulin spoof

Almost all of the drug industry’s biggest advertisers cut their spending on Twitter to zero or near-zero over the last two weeks amid worries about impersonation of their brands by pranksters and the future of the social media company.

Among 18 of the biggest pharmaceutical advertisers in the US market, 12 cut their Twitter ad spending to nothing for the week beginning Nov. 14, according to Pathmatics, which tracks data on prescription drug ad spending as well as general corporate advertising. The list of drugmakers cutting spending to zero includes Merck, AstraZeneca, Eli Lilly, Novartis, Pfizer and others.

Rob Davis, Merck CEO

Up­dat­ed: No Seagen here: 'Do more' means a small $1.35B pur­chase of Ima­go for Mer­ck

Merck is making an acquisition, the Big Pharma announced before Monday’s opening bell. No, Seagen is not entering the fold, as had been speculated for quarters.

Folding under Merck’s wings will be Pfizer-backed Imago BioSciences. For nearly a year, Merck CEO Rob Davis has been saying the pharma giant needs to “do more” on the business development front after its 2021 $11.5 billion acquisition of Acceleron.

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FDA preps for DMD drug gener­ics as Sarep­ta has yet to fin­ish its con­fir­ma­to­ry tri­al

The FDA typically releases guidance to help generic drug manufacturers develop new copycats of small molecule drugs, oftentimes in preparation for a brand name product’s patents or exclusivity to expire.

This week, FDA released such bioequivalence guidance for any generic drugmakers looking to take on Sarepta’s Duchenne muscular dystrophy (DMD) drug Exondys 51 (eteplirsen), even though the drug’s sponsor has yet to convert the accelerated approval to a full approval, showing clinical benefit.

Alzheimer’s drug bites the dust; Re­struc­ture, re­struc­ture, re­struc­ture; Land­mark di­a­betes OK; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Being in the news business can give one a warped sense of time — it feels like quite a while since we published some of these stories below. But next Saturday’s Endpoints Weekly will definitely be shorter, as we take off Thursday and Friday for Thanksgiving. We will still have the abbreviated edition in your inbox at the usual time.

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Days af­ter re­port­ing PhI­II fail­ure, GSK pulls BC­MA drug from US mar­ket — but it's not giv­ing up en­tire­ly yet

GSK is pulling its BCMA-targeting drug from the US market, ending a short, two-year run for a high-profile product that, among other things, was hailed for marking the pharma giant’s return to oncology.

The company is initiating the process for withdrawal at the request of the FDA, which in turn was based on the negative readout of a confirmatory Phase III trial earlier this month. In that trial, GSK’s Blenrep failed to extend progression-free survival over standard of care for patients with multiple myeloma who have received at least two prior lines of therapy.

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Karen Aiach, Lysogene CEO (RE(ACT) Discovery Institute)

Gene ther­a­py flunks PhII/III study, but for­mer Sarep­ta part­ner sees a path for­ward — if it can find the cash

The development path for Lysogene’s gene therapy for MPS IIIA has been a rocky one. After the FDA slapped a partial clinical hold on a Phase II/III study, a patient already dosed in the trial died, although it was deemed unrelated to treatment. Then earlier this year, Sarepta pulled out of their three-year partnership due to disagreements on who will handle commercial supply.

And now, Lysogene reported the trial has failed its primary endpoint.

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