Mallinck­rodt be­comes the lat­est vic­tim of the FDA's pan­dem­ic-re­lat­ed de­lays as it push­es off ap­pli­ca­tion for skin graft

With Covid-19 lock­ing down trav­el across the world, not even drug safe­ty reg­u­la­tors have been spared from cost­ly de­lays. For the FDA, whose in­spec­tion sched­ule keeps the trains on time for new mar­ket­ing ap­provals, those de­lays are con­tin­u­ing to prove cost­ly.

The FDA has pushed re­view of Mallinck­rodt’s al­lo­gene­ic skin graft for burns, dubbed Strat­a­Graft, af­ter the agency ad­mit­ted it wasn’t able to con­duct a plant in­spec­tion in the need­ed time­frame for a de­ci­sion, the drug­mak­er said last week.

Steven Ro­mano

Mallinck­rodt tied the de­lay to “Covid-19 trav­el re­stric­tions” and said it had no hang-ups about its graft’s chances at an ap­proval.

“We are con­fi­dent in the ef­fi­ca­cy and safe­ty of Strat­a­Graft for the treat­ment of deep par­tial-thick­ness burns based on our piv­otal Phase 3 clin­i­cal tri­al re­sults,” CSO Steven Ro­mano said in a re­lease. “We plan to work close­ly with the FDA to com­plete the re­view and sched­ule its site in­spec­tion.”

With the pan­dem­ic con­tin­u­ing to rage, the FDA has been forced to pri­or­i­tize planned in­spec­tions for new drug ap­pli­ca­tions. Of­ten, that sys­tem has fa­vored drug­mak­ers with new mol­e­cules with a high clin­i­cal need and shoved to the side low­er-im­por­tance prod­ucts, par­tic­u­lar­ly in aes­thet­ics.

In late No­vem­ber, the FDA de­layed its re­view of Re­vance’s frown-line in­jec­tion dax­i­bot­u­linum­tox­i­nA, say­ing it didn’t have enough time to look over its Newark, Cal­i­for­nia, man­u­fac­tur­ing fa­cil­i­ty on time. In Oc­to­ber, the agency pushed back Spec­trum’s neu­trope­nia can­di­date Rolon­tis af­ter of­fi­cials twice de­layed a plant in­spec­tion of South Ko­rea’s Han­mi, which holds the drug’s li­cense.

Mean­while, in­spec­tion de­lays have al­so nixed time­ly ap­provals for big­ger-name prod­ucts as well. The FDA re­peat­ed­ly de­layed a site in­spec­tion for Bris­tol My­ers Squibb’s liso-cel, even­tu­al­ly con­duct­ing a site vis­it at Lon­za Hous­ton’s con­tract site in De­cem­ber that re­sult­ed in a Form 483. That de­lay pushed liso-cel’s ap­proval past the Dec. 31 cut­off point tied to a $9 CVR in­vestors held from Bris­tol’s ac­qui­si­tion of Cel­gene.

Lon­za af­ter the fact said the FDA’s de­lays didn’t give ei­ther com­pa­ny enough time to ad­dress any par­tic­u­lar man­u­fac­tur­ing is­sues, which are quite com­mon for CAR-Ts on the whole. Liso-cel was even­tu­al­ly ap­proved ear­li­er this month and will be mar­ket­ed as Breyanzi.

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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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.

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.

Fu­ji­film to build $188M man­u­fac­tur­ing plant in North Car­oli­na’s re­search tri­an­gle

As the Japanese conglomerate Fujifilm continues to invest heavily in its CDMO arm, one of its manufacturing divisions is teeing up a major investment.

Fujifilm Irvine Scientific announced on Tuesday that parent Fujifilm is making a $188 million investment to build a cell culture media manufacturing site in the Research Triangle Park in North Carolina. The new site will mark Fujifilm Irvine’s fifth manufacturing site globally and its second in the US.

Dermavant Sciences' first consumer TV ad for its Vtama psoriasis med shows people ready for a new topical treatment.

Roivant’s Der­ma­vant de­buts first-ever TV com­mer­cial for pso­ri­a­sis cream Vta­ma

Dermavant Sciences has been marketing its first product, psoriasis med Vtama, to dermatologists for months, but on Tuesday it rolled out its first consumer campaign. The debut DTC effort including a streaming TV commercial encourages patients to a “Topical Uprising” in a nod to Vtama being a topical cream.

In the new commercial, a swell of people discards scarves and jacket coverings, gathering in the street to converge on a pharmacy to demand a steroid-free prescription. A moment of levity follows when a pharmacist says, “You know you can just talk to your doctor, right?” The gathered crowds collectively says, “Oh.”

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Sus­pend­ed Cal­i­for­nia cell ther­a­py man­u­fac­tur­ing site hit with FDA warn­ing let­ter over ma­jor qual­i­ty con­cerns

A cell therapy outfit in California that manufactures a human umbilical cord derived cellular product and exosome products is facing a warning from the FDA over several major observations related to quality.

The FDA notes the site’s “deficient donor screening practices, inadequate aseptic practices, unvalidated manufacturing,” and the “risk that your products may be contaminated with microorganisms or have other serious product quality defects.”