ODAC votes to keep Keytru­da and Tecen­triq ac­cel­er­at­ed ap­provals in blad­der can­cer

The FDA’s On­co­log­ic Drugs Ad­vi­so­ry Com­mit­tee is con­tin­u­ing to find it dif­fi­cult to say no to can­cer pa­tients, es­pe­cial­ly as blad­der can­cer pa­tients af­fect­ed by Wednes­day’s votes have few oth­er treat­ment op­tions.

Pe­ter Kang

On Wednes­day morn­ing, ODAC vot­ed 5-3 in fa­vor of keep­ing Keytru­da’s (pem­brolizum­ab) ac­cel­er­at­ed ap­proval alive as a first line blad­der can­cer treat­ment for those who are cis­platin-in­el­i­gi­ble and car­bo­platin-in­el­i­gi­ble, even af­ter a Mer­ck con­fir­ma­to­ry tri­al failed.

And on Wednes­day af­ter­noon, ODAC vot­ed 10-1 in fa­vor of keep­ing Genen­tech’s Tecen­triq (ate­zolizum­ab) as a first-line treat­ment of cis­platin-in­el­i­gi­ble pa­tients with ad­vanced/metasta­t­ic blad­der can­cer pend­ing fi­nal over­all sur­vival re­sults from a con­fir­ma­to­ry tri­al, known as IMvig­or130.

Among those vot­ing in fa­vor of Keytru­da — Mer­ck’s mega-block­buster drug, with 19 full ap­provals, in­clud­ing a full ap­proval as a sec­ond-line blad­der can­cer treat­ment, and 10 ac­cel­er­at­ed ap­provals — sev­er­al physi­cians point­ed to the lack of oth­er treat­ments for cer­tain pa­tients.

While the con­fir­ma­to­ry tri­al did not show a ben­e­fit for Keytru­da as a first-line treat­ment, much of the dis­cus­sion cen­tered on these sub­groups of pa­tients who might have no treat­ment op­tions with­out Keytru­da.

“Ac­cess to pem­brolizum­ab re­mains im­por­tant for these pa­tients, and FDA ap­proval should be main­tained un­til ad­di­tion­al con­fir­ma­to­ry da­ta are avail­able,” Pe­ter Kang, VP of clin­i­cal re­search at Mer­ck, told the com­mit­tee.

Laleh Amiri-Ko­rdestani

As far as how Mer­ck might still con­firm ben­e­fit in this in­di­ca­tion, Kang point­ed to one study that would look at Keytru­da in com­bi­na­tion with Ei­sai’s Lenvi­ma, com­pared with Keytru­da and place­bo. That tri­al is ex­pect­ed to be com­plet­ed in Oc­to­ber 2023. But the FDA said it would be chal­leng­ing to use this tri­al as it does not iso­late the ef­fect of Keytru­da. Two oth­er po­ten­tial con­fir­ma­to­ry tri­als from Mer­ck may al­so be used to con­firm its ben­e­fit, and are ex­pect­ed to fin­ish in mid-2025 and 2027.

Lay­ing out the case for FDA, Laleh Amiri-Ko­rdestani, di­vi­sion di­rec­tor in FDA’s Of­fice of On­co­log­ic Dis­eases, not­ed that the clin­i­cal tri­al ev­i­dence does not sup­port con­tin­ued ap­proval for Keytru­da in this set­ting.

Ronac Mam­tani

She al­so ex­plained how the treat­ment land­scape has changed, as last June, FDA ap­proved EMD Serono’s Baven­cio (avelum­ab) for those with blad­der can­cer who have not pro­gressed with first-line plat­inum-con­tain­ing chemother­a­py.

But Ronac Mam­tani, blad­der can­cer doc­tor at the Uni­ver­si­ty of Penn­syl­va­nia, took to the pub­lic fo­rum at the meet­ing and stressed what oth­er pan­elists had been hint­ing at — that re­tain­ing this in­di­ca­tion could be crit­i­cal for some pa­tients who are cis­platin-in­el­i­gi­ble.

For the af­ter­noon ses­sion, pan­elists were over­whelm­ing­ly in fa­vor of wait­ing for the over­all sur­vival re­sults from a con­fir­ma­to­ry tri­al to read out for Tecen­triq be­fore de­cid­ing on whether to pull the blad­der can­cer in­di­ca­tion.

Mo­hum­mad Sid­diqui, di­rec­tor of uro­log­ic on­col­o­gy at the Uni­ver­si­ty of Mary­land Med­ical Cen­ter, vot­ed to keep this ac­cel­er­at­ed ap­proval for Tecen­triq and said the in­ter­im analy­sis looks promis­ing and the FDA should wait for the fi­nal analy­sis from IMvig­or130. And Rick Paz­dur, FDA’s top on­col­o­gy head, sweet­ened the of­fer for Roche/Genetech, not­ing that if the com­pa­ny hits on the over­all sur­vival in the tri­al, they’ll be able to con­vert the Tecen­triq monother­a­py ac­cel­er­at­ed ap­proval to full ap­proval and the com­pa­ny will win a new in­di­ca­tion for Tecen­triq in com­bi­na­tion with chemo.

Mo­hum­mad Sid­diqui

Philip Hoff­man, chair of ODAC, was the on­ly com­mit­tee mem­ber to vote against keep­ing this Tecen­triq ac­cel­er­at­ed ap­proval, and he said he was con­cerned by with­draw­al of the Roche/Genen­tech drug in the sec­ond line set­ting as the pri­ma­ry end­point of over­all sur­vival was not met.

The votes on Wednes­day fol­lowed Tues­day’s meet­ing, in which com­mit­tee mem­bers vot­ed 7-2 to keep alive the ac­cel­er­at­ed ap­proval for Genen­tech’s Tecen­triq (ate­zolizum­ab) plus Abrax­ane (nab-pa­cli­tax­el) in metasta­t­ic triple neg­a­tive breast can­cer (mTNBC) while ad­di­tion­al con­fir­ma­to­ry tri­als are on­go­ing.

Three more ac­cel­er­at­ed ap­provals (in­clud­ing two for Keytru­da and one for Bris­tol My­ers Squibb’s Op­di­vo) are set for votes on Thurs­day. While the FDA does not have to fol­low the ad­vice of ODAC, it gen­er­al­ly does.

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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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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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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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EMA rec­om­mends re­vok­ing au­tho­riza­tion of No­var­tis' sick­le cell drug

The European Medicines Agency’s committee for medicinal products for human use (CHMP) on Friday recommended revoking the marketing authorization for Novartis’ treatment for a painful complication related to sickle cell, after a recent study did not confirm its clinical benefit.

CHMP’s review looked at results of the STAND study, finding that Adakveo (crizanlizumab) did not reduce the number of painful crises leading to a healthcare visit, and patients treated with Adakveo had slightly more painful crises on average, with a subsequent healthcare visit, over the first year of treatment, compared with those on placebo.

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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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