Janet Woodcock, acting FDA commissioner (Bill Clark/CQ Roll Call via AP Images)

FDA’s do­mes­tic bio­phar­ma in­spec­tions re­turn to nor­mal as agency faces back­log of 8,000+

For the first time since March 2020, the FDA’s in­spec­tions of US-based bio­phar­ma sites have re­turned to nor­mal, FDA act­ing com­mis­sion­er Janet Wood­cock said Mon­day at a small busi­ness reg­u­la­to­ry event.

“I’m pleased to say that as of this month, we’ve be­gun tran­si­tion­ing back to stan­dard op­er­a­tions for do­mes­tic in­spec­tions while con­tin­u­ing to pri­or­i­tize mis­sion-crit­i­cal work for for­eign in­spec­tions,” Wood­cock said.

The re­turn to nor­mal­cy will be wel­comed by in­dus­try, which has grap­pled with a grow­ing back­log of most­ly sur­veil­lance in­spec­tions, al­though some of those de­layed in­spec­tions have al­so de­layed the ap­proval of new drugs.

Ac­cord­ing to a re­port from FDA in May, an es­ti­mat­ed 68 ap­pli­ca­tions (in­clud­ing 48 for hu­man drugs) have been de­layed due to FDA’s in­abil­i­ty to con­duct pre-ap­proval, pre-mar­ket, or pre-li­cense in­spec­tions as of March.

The agency said it post­poned near­ly 8,000 non-mis­sion-crit­i­cal sur­veil­lance in­spec­tions in 2020 due to the pan­dem­ic. Sur­veil­lance in­spec­tions are rou­tine in­spec­tions that mon­i­tor con­for­mance to FDA re­quire­ments to iden­ti­fy qual­i­ty prob­lems and ad­verse trends, but un­like oth­ers, they’re planned in ad­vance by ap­ply­ing es­tab­lished risk fac­tors and statu­to­ry in­spec­tion fre­quen­cy man­dates.

“FDA es­ti­mates that rough­ly 14% of the 15,514 do­mes­tic sur­veil­lance in­spec­tions still to be con­duct­ed in FY21 will be achiev­able in the Base-Case sce­nario,” the re­port says, not­ing that about 3,229 of those 15,000+ in­spec­tions are re­lat­ed to hu­man and an­i­mal drugs, while the rest are re­lat­ed to hu­man and an­i­mal food.

And in terms of for-cause in­spec­tions, mean­ing in­spec­tions where there are con­sumer com­plaints or re­ports of ad­verse events, the FDA had to skip 8 of those in­spec­tions in FY 2020.

As far as how the agency is work­ing around the ris­ing cas­es of Covid-19 in cer­tain hotspots across the coun­try, FDA re­vealed ear­li­er this month that it de­vel­oped a rat­ing sys­tem to as­sist in de­ter­min­ing when and where it’s safest to con­duct do­mes­tic in­spec­tions.

The rat­ing sys­tem, which FDA said it may sus­pend through Sep­tem­ber as it re­sumes stan­dard op­er­a­tions, us­es re­al-time da­ta to qual­i­ta­tive­ly as­sess the num­ber of Covid-19 cas­es in a lo­cal area based on state and na­tion­al da­ta. FDA shared that da­ta with state agen­cies that car­ry out in­spec­tions of FDA-reg­u­lat­ed en­ti­ties on the agency’s be­half un­der con­tract.

FDA al­so pre­vi­ous­ly said that for the fore­see­able fu­ture, pri­or­i­tized do­mes­tic in­spec­tions will be pre-an­nounced to FDA-reg­u­lat­ed busi­ness­es. Wood­cock did not an­nounce any change to that pol­i­cy.

On the for­eign in­spec­tion side, where Con­gress has been par­tic­u­lar­ly point­ed with the agency, FDA’s As­so­ciate Com­mis­sion­er for Reg­u­la­to­ry Af­fairs Ju­dith McMeekin told an Al­liance for a Stronger FDA we­bi­nar last week that the agency has strug­gled.

“We have suc­cess­ful­ly con­duct­ed about 30 for­eign in­spec­tions that were mis­sion crit­i­cal. Go­ing and con­duct­ing these for­eign in­spec­tions dur­ing the pan­dem­ic, it hasn’t been with­out tremen­dous ef­forts to mit­i­gate risk to our in­ves­ti­ga­tors,” she said. “You have to re­mem­ber, there were many months be­fore we had a vac­cine and so our in­ves­ti­ga­tors were go­ing out, trav­el­ing to for­eign coun­tries. And then en­tailed in a tremen­dous for­ti­tude on be­half of our in­ves­ti­ga­tors, many had to quar­an­tine up­on ar­rival in a coun­try for 10 to 14 days and mil­i­tary hous­ing or in their ho­tel room. Some cas­es they were not able to re­al­ly move around freely.”

McMeekin al­so touched on how the agency was able to rapid­ly in­spect fa­cil­i­ties ahead of the Covid-19 vac­cine EUAs:

“Our in­ves­ti­ga­tors con­duct­ed about 26 clin­i­cal re­search fa­cil­i­ty in­spec­tions be­fore the ad­vi­so­ry com­mit­tee meet­ings and be­fore we had au­tho­rized vac­cines. So, in just four weeks, we com­plet­ed all of those in­spec­tions with a team of more than 50 in­ves­ti­ga­tors. So, how did we do this? We col­lab­o­rat­ed with our cen­ters. We col­lab­o­rate with the cen­ters for bi­o­log­ics, we planned out the in­spec­tions and the time­lines in ad­vance of the in­spec­tion as­sign­ments … We gen­er­al­ly send in one in­ves­ti­ga­tor to the fa­cil­i­ty for clin­i­cal re­search in­spec­tions. In these cas­es, we send in two so that we could en­sure the ac­cu­ra­cy, re­li­a­bil­i­ty and the da­ta in the short­est amount of time. So, we al­so im­ple­ment­ed use of portable pro­jec­tors to re­view the doc­u­ments in a so­cial­ly dis­tant man­ner.”

Oth­er Covid-re­lat­ed tran­si­tions

Fol­low­ing Wood­cock’s com­ments, FDA cen­ter di­rec­tors Jeff Shuren (de­vices), Pa­trizia Cavaz­zoni (drugs) and Pe­ter Marks (bi­o­log­ics) al­so of­fered their per­spec­tives on what Covid-re­lat­ed pro­vi­sions may car­ry over to the post-Covid FDA.

Shuren stressed the need for faster guid­ance de­vel­op­ment, not­ing that some Covid-re­lat­ed guid­ance doc­u­ments were de­vel­oped in on­ly days or weeks, and he’d like to see that ex­pe­dit­ed ap­proach car­ry over be­yond the pan­dem­ic. Marks and Cavaz­zoni con­curred, and Cavaz­zoni men­tioned the need to build off FDA’s ac­cu­mu­lat­ed ex­pe­ri­ence with de­cen­tral­ized clin­i­cal tri­als dur­ing Covid-19, and the use of al­ter­na­tive ap­proach­es, such as re­mote eval­u­a­tions, to in­spect man­u­fac­tur­ing and clin­i­cal tri­al fa­cil­i­ties.

As far as when the EUA path­way may close for spon­sors, Shuren said he didn’t think it would hap­pen any time soon, and he not­ed that there will be an “am­ple tran­si­tion pe­ri­od,” as well as guid­ance on that tran­si­tion. Marks said the EUA tran­si­tion pe­ri­od for vac­cines may look com­pli­cat­ed as there might be some pe­di­atric in­di­ca­tions still un­der EUAs for vac­cines that have won full ap­provals. He stressed that the idea is to min­i­mize any­thing that cre­ates con­fu­sion, and de­creas­es trust.

Ed­i­tor’s note: Ar­ti­cle up­dat­ed with com­ments from Dr. McMeekin.

Image courtesy of The Janssen Pharmaceutical Companies of Johnson & Johnson.

Pro­tect­ing the glob­al phar­ma­ceu­ti­cal in­no­va­tion ecosys­tem – what’s at stake?

We are living in a new era of healthcare that is rapidly advancing progress impacting patient outcomes and experiences. We’ve seen a remarkable pace of transformational innovation, applied research, and advanced clinical development over the last decade.

Despite this tremendous progress, there is much more work to be done, and patients are counting on us – now more than ever – to continue that momentum. At the heart of our industry is a focus on developing and delivering medicines for some of the world’s most challenging diseases, including those that have few or no effective treatments today.

Mi­rati’s drug sitra­va­tinib flops PhI­II in com­bo with Op­di­vo for cer­tain lung can­cer

Mirati Therapeutics’ path to a second drug approval will likely have to wait. The San Diego biotech company said Wednesday that its investigational lung cancer drug failed a Phase III trial testing it in combination with Bristol Myers Squibb’s Opdivo.

The drug, sitravatinib, and Opdivo weren’t better than the chemo drug docetaxel at keeping patients alive, Mirati said in a press release. The spectrum-selective kinase inhibitor missed the primary goal of overall survival in patients with second- or third-line advanced non-squamous, non-small cell lung cancer.

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End­points 20(+2) un­der 40, 2023; Bio­phar­ma's high­est-paid CEOs; N-of-1 CRISPR sto­ry goes on af­ter tragedy; 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.

We will be off Monday in observance of Memorial Day — and when we get back, it will be a straight march to ASCO, BIO and more. Enjoy the (long) weekend!

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FDA ap­proves Lex­i­con’s heart-fail­ure drug af­ter de­feat in di­a­betes

The FDA on Friday approved Lexicon’s heart failure drug sotagliflozin following a string of setbacks for the pharma company, including an FDA rejection in diabetes and the loss of a development deal with Sanofi.

The dual SGLT1 and SGLT2 inhibitor will be marketed as Inpefa and is a once-daily tablet. It’s been approved to reduce the risk of cardiovascular death and heart failure-related hospitalization or urgent visits in adults with heart failure or type 2 diabetes mellitus, chronic kidney disease, and other cardiovascular risk factors. The label spans the range of left ventricular ejection fraction, including preserved ejection fraction and reduced ejection fraction, as well as patients with or without diabetes, Lexicon said Friday.

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Rich Horgan (R) with his late brother, Terry

Rich Hor­gan spear­head­ed a gene ther­a­py for his broth­er. The tri­al end­ed in tragedy, but the work con­tin­ues for more pa­tients

Rich Horgan’s quest to create a custom gene therapy for his brother, Terry, ended in tragedy. But Horgan doesn’t believe it’s the end of the story.

Terry, a 27-year-old patient with Duchenne muscular dystrophy, died last October just eight days after receiving the therapy in a clinical trial in which he was the only participant. The case raised questions about the safety of certain gene therapies and what would happen to other drug programs under a nonprofit that Horgan created, called Cure Rare Disease.

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Bio­phar­ma's 20 high­est-paid CEOs of 2022, each bring­ing in $20M+ pay­days

Even in a down year for much of the biopharma market, 20 CEOs brought in pay packages valued at more than $20 million, an Endpoints News analysis found.

Endpoints collected data on more than 350 CEO compensation packages, covering a wide range of pharma, biotech, and life sciences companies. All told, the 20 largest earners made over $725 million in 2022 — an average package of $36.4 million. Three brought in paydays over $50 million, and one CEO broke the $100 million mark.

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The 20(+2) un­der 40: Your guide to the next gen­er­a­tion of biotech lead­ers

This year’s list of 20 biotech leaders under the age of 40 includes a huge range of ambitions. Some of our honorees are planning to create the next big drug giant. Others are pushing the bounds of AI. One is working to revolutionize TB testing. All are compelling talents who are still young in age, but already far along in achievement.

And, as in years past, we went over. The 20 are actually 22 because of two double profiles that reflect how important teamwork is in the industry. As one of our honorees, Joe Illingworth of DJS Antibodies, told me in our interview, “It takes a village to raise a biotech.”

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Eu­ro­pean Com­mis­sion to re­ceive few­er Pfiz­er-BioN­Tech vac­cine dos­es un­der amend­ed con­tract

The European Commission has made a few changes to its vaccine contract with Pfizer and BioNTech, reducing the dose volume while extending the delivery timeline to cope with “evolving public health needs.”

The Commission previously struck a contract in May 2021 for 900 million doses, with the option to purchase another 900 million. Of those, 450 million were expected to be delivered in 2023, though an amendment now calls for fewer doses. While neither the Commission nor Pfizer and BioNTech have revealed an exact amount, an unnamed source told Reuters that the amendment reduces the remaining expected doses by about a third.

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Teresa Bitetti, Takeda's president of the global oncology business unit

Take­da wins pri­or­i­ty re­view for $400M col­orec­tal can­cer drug, li­censed from Hutchmed in Jan­u­ary

Takeda and Hutchmed scored a priority review Thursday afternoon for a colorectal cancer drug, the companies announced.

The experimental drug in question is fruquintinib, previously approved in China in 2018 to treat metastatic colorectal cancer. Takeda and Hutchmed are aiming to bring fruquintinib to the US and other countries outside China in the same indication, and the FDA set its decision date for Nov. 30 of this year.

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