Sen. Patty Murray (D-WA) (Graeme Sloan/Sipa USA/Sipa via AP Images)

CDER di­rec­tor on ac­cel­er­at­ed ap­proval re­forms and a court de­ci­sion that will 'send a chill' across rare dis­ease drug de­vel­op­ment

At the sec­ond of two hear­ings be­fore the Sen­ate Health, Ed­u­ca­tion, La­bor & Pen­sions Com­mit­tee on the must-pass leg­is­la­tion (by the end of Sep­tem­ber) re­gard­ing the funds from bio­phar­ma in­dus­try ap­pli­ca­tions that will keep the FDA afloat over the next five years, CDER di­rec­tor Pa­trizia Cavaz­zoni raised par­tic­u­lar con­cerns about a re­cent court de­ci­sion while lay­ing out sev­er­al re­quests for con­gres­sion­al ac­cel­er­at­ed ap­proval re­forms.

Pa­trizia Cavaz­zoni

Re­spond­ing to a ques­tion from Sen. Tam­my Bald­win (D-WI), Cavaz­zoni said she wants to work with Con­gress to find a so­lu­tion af­ter a re­cent court de­ci­sion against the FDA “will send a chill in­to the de­vel­op­ment of rare dis­eases and it will dis­pro­por­tion­ate­ly af­fect chil­dren with rare dis­eases. It’s es­sen­tial to con­tin­ue to gen­er­ate the study of drugs in chil­dren, so this de­ci­sion will re­al­ly go counter to that.”

The case in ques­tion from last Oc­to­ber saw a US ap­peals court over­turn a pri­or FDA court win, say­ing that the agency nev­er should’ve ap­proved a rare dis­ease drug be­cause a pre­vi­ous­ly ap­proved but more ex­pen­sive drug with the same ac­tive in­gre­di­ent has or­phan drug ex­clu­siv­i­ty bar­ring such an ap­proval.

The sit­u­a­tion right now, Cavaz­zoni said, fol­low­ing the court’s de­ci­sion “is a spon­sor could study a dis­ease in a very nar­row seg­ment of the pop­u­la­tion and then be able to block fur­ther ap­provals through­out the en­tire con­di­tion that that drug could ad­dress.”

Sen. Su­san Collins (R-ME) al­so raised ques­tions with Cavaz­zoni re­gard­ing CMS’ re­cent de­ci­sion to not cov­er Bio­gen’s con­tro­ver­sial Alzheimer’s drug, and its dis­cus­sion of the safe­ty of the drug.

Cavaz­zoni de­fend­ed the ap­proval, say­ing the da­ta “are sol­id and the drug is ap­pro­pri­ate­ly made avail­able to pa­tients based on FDA’s de­ci­sion. It’s im­por­tant to dis­tin­guish FDA and CMS’ roles,” she said, adding that FDA is sole­ly re­spon­si­ble for de­ter­min­ing safe­ty and ef­fec­tive­ness, and CMS has a dif­fer­ent stan­dard, rea­son­able and nec­es­sary, which trans­late in­to the set­ting in how the drug is cov­ered.

“There are some ar­eas when it comes to ac­cel­er­at­ed ap­proval where we could use some help from Con­gress,” Cavaz­zoni said, not­ing that FDA doesn’t have the au­thor­i­ty to re­quire that con­fir­ma­to­ry tri­als are start­ed or be un­der­way by the time the drug is ap­proved, or at least have a de­tailed plan to con­duct those tri­als.

“An­oth­er area we can use some help is in ex­pe­dit­ing the with­draw­al of drugs when the con­fir­ma­to­ry tri­als do not con­firm the drug is as­so­ci­at­ed with clin­i­cal ben­e­fit,” Cavaz­zoni said. “Right now the ex­pe­dit­ed with­draw­al path is any­thing but ex­pe­dit­ed. It can take up to 2 years and re­quire lots of re­sources and lots of ad­min­is­tra­tive bur­den.”

Com­mit­tee Chair Sen. Pat­ty Mur­ray of Wash­ing­ton ac­knowl­edged that the user fee pro­grams in gen­er­al have an im­por­tant role, and they en­sure that more drugs and de­vices cross the fin­ish line and that the FDA gets the ap­pro­pri­ate re­sources.

Richard Burr

“It should be un­think­able that af­ter 2 years, when FDA’s work has been more im­por­tant than ever, that we would fail to get this done or force the agency to send pink slips,” Mur­ray said, while say­ing that they should look back at this pan­dem­ic — from FDA’s work to re­view and ap­prove vac­cines quick­ly to oth­er is­sues like test­ing strug­gles and the hy­drox­y­chloro­quine de­ba­cle.

The top Re­pub­li­can mem­ber of the com­mit­tee, Richard Burr of North Car­oli­na, raised con­cerns about the cur­rent lack of funds flow­ing in­to biotech in gen­er­al, and said to­day’s hear­ing was about ac­count­abil­i­ty, while men­tion­ing the need for FDA “to speed not on­ly the re­view of prod­ucts but their de­vel­op­ment as well.”

He al­so raised sev­er­al con­cerns, high­light­ing sev­er­al bench­marks the agency failed to meet in the last user fee agree­ment.

“Now FDA wants dou­ble the mon­ey for mediocre per­for­mance im­prove­ments,” Burr said. “The more you use the user fee process to bul­ly dol­lars out of in­dus­try, hold­ing them hostage, the less ac­count­able the FDA is.”

Pe­ter Marks

CBER di­rec­tor Pe­ter Marks al­so ex­plained the cur­rent sit­u­a­tion of Covid-19 vac­cines for the youngest group of chil­dren (un­der 5), say­ing that as soon as the FDA re­ceives an ap­pli­ca­tion, “we’ll move quick­ly. It’s one of our high­est pri­or­i­ties.”

Again he said that an ad­comm of out­side ex­perts will meet and re­view the da­ta on the vac­cines too, and in the next week, Marks said, the FDA will re­lease a ten­ta­tive time­line for those meet­ing(s) as the agency re­views these 2 ap­pli­ca­tions from Pfiz­er and Mod­er­na. He al­so stressed that the FDA can’t start or fin­ish its re­views un­til the com­plete ap­pli­ca­tions are sub­mit­ted.

Com­pa­nies of­ten re­lease state­ments ahead of the FDA on when such ap­pli­ca­tions are ful­ly sub­mit­ted, he not­ed.

On the top­ic of va­can­cies, which is al­ways a ten­u­ous top­ic at the FDA as sev­er­al se­nior vac­cine and oth­er lead­ers moved on re­cent­ly, Cavaz­zoni said CDER is look­ing to fill roles for about 7-8% of its staff, which “isn’t a high rate for a large or­ga­ni­za­tion,” she said.

In re­sponse to ques­tions over the re­cent­ly un­veiled McK­in­sey con­flicts of in­ter­est with FDA re­gard­ing their opi­oid work, Cavaz­zoni stressed that CDER doesn’t have any con­tracts with McK­in­sey, and while McK­in­sey had worked with FDA, its work with FDA did not en­tail spe­cif­ic sci­en­tif­ic re­views of prod­ucts or process­es.

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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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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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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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Douglas Love, Annexon CEO

An­nex­on’s GA drug miss­es on pri­ma­ry goal but win on vi­su­al acu­ity will be fo­cus of planned late-stage tri­al

Annexon’s complement inhibitor didn’t prove better than sham at reducing lesion growth in a leading cause of blindness, but the biotech still plans to move forward on the back of secondary endpoints showing visual acuity preservation, which will “certainly” be the primary goal in a late-stage trial to be discussed shortly with the FDA, CEO Douglas Love told Endpoints News. 

The California biotech’s ANX007 was not statistically significant compared to pooled sham, the comparator, at 12 months in patients with geographic atrophy, per a Wednesday presentation. In every-month dosing, the GA lesion area changed about 6.2% from baseline (p=0.526) and 1.3% (p=0.896) in the every-other-month group. In a March note, Jefferies analyst Suji Jeong said a reduction of 20% to 30% would be “encouraging.”

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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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