Emer Cooke, ICRMA chair (AP Photo/Geert Vanden Wijngaert)

ICM­RA to launch sev­er­al reg­u­la­to­ry pi­lot pro­grams cen­tered around man­u­fac­tur­ing in­spec­tions

As reg­u­la­to­ry agen­cies look to catch up on in­spec­tions amid the Covid-19 pan­dem­ic, ICM­RA is un­veil­ing sev­er­al pi­lot pro­grams to ad­dress in­dus­try ap­pli­ca­tions and in­spec­tions.

ICM­RA, which is made up of the world’s top drug reg­u­la­tors, is launch­ing mul­ti­ple pi­lot pro­grams, in­clud­ing two reg­u­la­to­ry pi­lots ad­dress­ing fa­cil­i­ty in­spec­tions for chem­istry and man­u­fac­tur­ing con­trols (CMC) and post-ap­proval change (PAC) sub­mis­sion as­sess­ments and re­lat­ed reg­u­la­to­ry ac­tions.

These pi­lots will ex­plore the po­ten­tial for col­lab­o­ra­tion be­tween reg­u­la­tors when it comes to spe­cif­ic da­ta ex­pec­ta­tions and as­sess­ment ap­proach­es when as­sess­ing man­u­fac­tur­ing fa­cil­i­ties for PAIs and PLIs as well as re­view­ing PACs and PAC man­age­ment pro­to­cols. Each pi­lot will last rough­ly 1 to 1.5 years, and will in­volve two or more reg­u­la­to­ry au­thor­i­ties that will con­duct three as­sess­ments or in­spec­tions.

“ICM­RA rec­og­nizes that de­vel­op­ment, man­u­fac­ture, and sup­ply of med­i­cines is now glob­al, and a more glob­al reg­u­la­to­ry phar­ma­ceu­ti­cal qual­i­ty knowl­edge man­age­ment (PQ KM) ca­pa­bil­i­ty would im­prove reg­u­la­tors’ abil­i­ty to read­i­ly share and uti­lize the in­for­ma­tion that may al­ready be re­ceived or col­lect­ed. This in­cludes in­for­ma­tion on man­u­fac­tur­ing fa­cil­i­ties, prod­ucts, mar­ket­ing au­tho­riza­tion ap­pli­ca­tions, and mar­ket­ing ap­pli­ca­tion hold­ers,” ICM­RA said in a state­ment.

The oth­er pi­lots fo­cus on both col­lab­o­ra­tive as­sess­ments of CMC-re­lat­ed post-ap­proval changes and hy­brid in­spec­tions.

Ac­cord­ing to the or­ga­ni­za­tion, the main ob­jec­tives of the two pi­lots in­clude “the de­vel­op­ment of an ini­tial com­mon frame­work for col­lab­o­ra­tive as­sess­ment and hy­brid in­spec­tions,” and iden­ti­fy­ing “best prac­tices and stan­dards in the qual­i­ty as­sess­ment of CMC-re­lat­ed post-ap­proval changes and col­lab­o­ra­tive hy­brid in­spec­tions to in­form rel­e­vant qual­i­ty as­sess­ments.”

An­oth­er main goal is to de­vel­op rec­om­men­da­tions for a “fu­ture cross-re­gion­al path­way(s) to be pur­sued by ICM­RA.”

A re­port sum­ma­riz­ing these pro­grams will be pub­lished in 2023.

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

As you start planning for #JPM23, we hope you will consider joining Endpoints News for our live and virtual events. For those who are celebrating Thanksgiving, we hope you are enjoying the long weekend with loved ones. And if you’re not — we’ll see you next week!

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,000+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,000+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,000+ biopharma pros reading Endpoints daily — and it's free.

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.

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.

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.”

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,000+ biopharma pros reading Endpoints daily — and it's free.