FDA ex­tends full ap­proval for Mer­ck­'s Keytru­da in 1st-line blad­der can­cer af­ter mixed ad­comm vote

Mer­ck’s ac­cel­er­at­ed ap­proval for Keytru­da in first-line blad­der can­cer was one of a slate of ear­ly nods put un­der the mi­cro­scope by an FDA ad­vi­so­ry com­mit­tee ear­li­er this year af­ter flop­ping con­fir­ma­to­ry tests. Keytru­da passed that test, and now the FDA is mak­ing its ac­cel­er­at­ed OK of­fi­cial.

The FDA on Tues­day gave its full ap­proval to Keytru­da as a treat­ment for first-line ad­vanced blad­der can­cer pa­tients who are not el­i­gi­ble for plat­inum-based chemo de­spite a con­fir­ma­to­ry out­comes tri­al in an ex­pand­ed pa­tient pop­u­la­tion turn­ing up a dud, Mer­ck said in a state­ment.

Keytru­da earned an ac­cel­er­at­ed nod in first-line lo­cal­ly ad­vanced or metasta­t­ic urothe­lial can­cer (UC) pa­tients who were not el­i­gi­ble for chemo back in May 2017. But the con­fir­ma­to­ry KEYNOTE-361 study, which aimed to ex­pand Keytru­da in­to pa­tients who had been pre­vi­ous­ly treat­ed with plat­inum-based chemo, failed both of its key pri­ma­ry end­points, ef­fec­tive­ly shut­ting down Keytru­da’s broad­er use.

Ear­li­er this year, Keytru­da’s first-line ap­proval in blad­der can­cer was one of the in­di­ca­tions con­sid­ered by an FDA ad­comm for con­tin­ued sup­port. The ad­comm vot­ed 5-3 to main­tain the ac­cel­er­at­ed ap­proval. Mean­while, three oth­er in­di­ca­tions re­viewed at that meet­ing have since been with­drawn af­ter fur­ther con­sul­ta­tion with the FDA.

In a re­lease, Mer­ck chalked up the full ap­proval to the con­tin­ued lack of suc­cess for PD-1 im­munother­a­pies in first-line pa­tients, which Roche and As­traZeneca both hav­ing racked up loss­es:

“While the treat­ment land­scape has evolved, an un­met need re­mains for ap­pro­pri­ate pa­tients new­ly di­ag­nosed with cer­tain types of ad­vanced urothe­lial car­ci­no­ma who are not el­i­gi­ble for plat­inum-con­tain­ing chemother­a­py,” Scot Ebbing­haus, Mer­ck Re­search Lab­o­ra­to­ries’ VP of clin­i­cal re­search said in a state­ment. “We are con­fi­dent in the role Keytru­da will con­tin­ue to play for these pa­tients who have few oth­er treat­ment op­tions and are work­ing with ur­gency to ad­vance stud­ies to help more pa­tients liv­ing with blad­der and oth­er types of can­cer.”

Se­cur­ing a full ap­proval de­spite con­tro­ver­sy could be seen as a win, but not for Keytru­da, the world’s sec­ond-best­selling drug that has soaked up in­di­ca­tions on its way to dom­i­nat­ing the im­mune check­point in­hibitor space.

In blad­der can­cer, in par­tic­u­lar, Mer­ck has tak­en an ag­gres­sive ap­proach, se­cur­ing ap­provals in UC pa­tients whose dis­ease pro­gress­es dur­ing or af­ter plat­inum-based chemo or with­in 12 months of neoad­ju­vant or ad­ju­vant chemo, and as a treat­ment of pa­tients with Bacil­lus Cal­mette-Guerin-un­re­spon­sive, high-risk, non-mus­cle in­va­sive blad­der can­cer with car­ci­no­ma in situ with or with­out pap­il­lary tu­mors who are in­el­i­gi­ble for or have elect­ed not to un­der­go a cys­tec­to­my.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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FDA hands ac­cel­er­at­ed nod to Seagen, Gen­mab's so­lo ADC in cer­vi­cal can­cer, but com­bo stud­ies look even more promis­ing

Biopharma’s resident antibody-drug conjugate expert Seagen has scored a clutch of oncology approvals in recent years, finding gold in what are known as “third-gen” ADCs. Now, another of their partnered conjugates is ready for prime time.

The FDA on Monday handed an accelerated approval to Seagen and Genmab’s Tivdak (tisotumab vedotin-tftv, or “TV”) in second-line patients with recurrent or metastatic cervical cancer who previously progressed after chemotherapy rather than PD-(L)1 systemic therapy, the companies said in a release.

Jay Bradner (Jeff Rumans for Endpoints News)

Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a rather narrow market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

But that was just the first deal that whet their appetite.

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The biggest ques­tions fac­ing gene ther­a­py, the XLMTM com­mu­ni­ty, and Astel­las af­ter fourth pa­tient death

After three patients died last year in an Astellas gene therapy trial, the company halted the study and began figuring out how to safely get the program back on track. They would, executives eventually explained, cut the dose by more than half and institute a battery of other measures to try to prevent the same thing from happening again.

Then tragically, Astellas announced this week that the first patient to receive the new regimen had died, just weeks after administration.

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Ex­elix­is pulls a sur­prise win in thy­roid can­cer just days ahead of fi­nal Cabome­tyx read­out

Exelixis added a thyroid cancer indication to its super-seller Cabometyx’s label on Friday — months before the FDA was expected to make a decision, and days before the company was set to unveil the final data at #ESMO21.

At a median follow-up of 10.1 months, differentiated thyroid cancer patients treated with Cabometyx (cabozantinib) lived a median of 11 months without their disease worsening, compared to just 1.9 months for patients given a placebo, Exelixis said on Monday.

Dave Lennon, former president of Novartis Gene Therapies

Zol­gens­ma patent spat brews be­tween No­var­tis and Re­genxbio as top No­var­tis gene ther­a­py ex­ec de­parts

Regenxbio, a small licensor of gene therapy viral vectors spun out from the University of Pennsylvania, is now finding itself in the middle of some major league patent fights.

In addition to a patent suit with Sarepta Therapeutics from last September, Novartis, is now trying to push its smaller partner out of the way. The Swiss biopharma licensed Regenxbio’s AAV9 vector for its $2.1 million spinal muscular atrophy therapy Zolgensma.

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Ex-My­lan em­ploy­ee pleads guilty to in­sid­er trad­ing, il­le­gal­ly deal­ing on FDA ap­provals, earn­ings and Up­john merg­er

A former Mylan IT executive pleaded guilty Friday to an insider trading scheme where he bought and sold stock options on another executive’s advice.

Prosecutors secured the plea from Dayakar Mallu, Mylan’s former VP of global operations information technology, after uncovering the plan. Mallu collaborated with an unnamed “senior manager,” the SEC said, to trade options ahead of Mylan public announcements regarding FDA approvals, revenue reports and its merger with the Pfizer generics subsidiary Upjohn. The two subsequently shared profits.

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