FDA ush­ers in a new era in can­cer treat­ment with 'his­toric' CAR-T ap­proval for No­var­tis

The FDA has ap­proved the world’s first CAR-T ther­a­py, giv­ing a green light to No­var­tis for Kym­ri­ah (ti­s­agen­le­cleu­cel) in what reg­u­la­tors them­selves de­scribe as an his­toric event.

The ear­ly ap­proval — about a month ahead of the PDU­FA date — came through for cer­tain pe­di­atric and young adult pa­tients with a form of acute lym­phoblas­tic leukemia.

No­var­tis set the price for the one-time treat­ment at $475,000, right in line with low-end es­ti­mates and one that will put pres­sure on Gilead to rein in its own price for a ri­val ther­a­py ex­pect­ed to be ap­proved in the near fu­ture.

Scott Got­tlieb, FDA Com­mis­sion­er

None oth­er than FDA com­mis­sion­er Scott Got­tlieb did the hon­ors in the agency’s state­ment, not­ing:

We’re en­ter­ing a new fron­tier in med­ical in­no­va­tion with the abil­i­ty to re­pro­gram a pa­tient’s own cells to at­tack a dead­ly can­cer. New tech­nolo­gies such as gene and cell ther­a­pies hold out the po­ten­tial to trans­form med­i­cine and cre­ate an in­flec­tion point in our abil­i­ty to treat and even cure many in­tractable ill­ness­es. At the FDA, we’re com­mit­ted to help­ing ex­pe­dite the de­vel­op­ment and re­view of ground­break­ing treat­ments that have the po­ten­tial to be life-sav­ing.

A group of bio­phar­ma com­pa­nies have been rac­ing to this break­through point. Kite Phar­ma $KITE, new­ly bought out in a $12 bil­lion deal by Gilead $GILD, is com­ing in a close sec­ond with its own ap­pli­ca­tion for axi­cel.

No­var­tis’ in­ves­ti­ga­tors reg­is­tered a game-chang­ing 83% re­mis­sion rate in its piv­otal study.

CAR-T, though, is al­so as­so­ci­at­ed with se­vere and po­ten­tial­ly dead­ly side ef­fects, in­clud­ing lethal in­stances of cy­tokine re­lease syn­drome with some pa­tients dy­ing from brain swelling in sep­a­rate stud­ies from the No­var­tis drug.

Reg­u­la­tors not­ed that they will re­quire spe­cial train­ing for any­one in­volved in de­liv­er­ing this ther­a­py, while ex­pand­ing the ap­proval of Actem­ra (tocilizum­ab) to treat CAR T-cell-in­duced se­vere or life-threat­en­ing CRS in pa­tients 2 years of age or old­er. “In clin­i­cal tri­als in pa­tients treat­ed with CAR-T cells,” the FDA re­port­ed, “69% of pa­tients had com­plete res­o­lu­tion of CRS with­in two weeks fol­low­ing one or two dos­es of Actem­ra.”

This ther­a­py, which is made us­ing a pa­tient’s own im­mune cells, won’t be cheap.

By its own reck­on­ing, the UK’s tough watch dog on drug pric­ing has said that these drugs would be worth up to $649,000 a year, giv­en the young pa­tients it’s tar­get­ing first. An­a­lysts, mean­while, have pegged the price at any­where from $400,000 to $750,000.

In a call with an­a­lysts, No­var­tis ex­ecs said it would keep the price well be­low the high end, set­ting the cost at $475,000 for a drug that will be avail­able in 20 cen­ters with­in one month, and 35 soon af­ter.

In a state­ment the com­pa­ny said it “is col­lab­o­rat­ing with CMS to make an out­comes-based ap­proach avail­able to al­low for pay­ment on­ly when pe­di­atric and young adult ALL pa­tients re­spond to Kym­ri­ah by the end of the first month. Fu­ture po­ten­tial in­di­ca­tions would be re­viewed for the most rel­e­vant out­comes-based ap­proach.”

Joe Jimenez

Said Joseph Jimenez, CEO of No­var­tis:

Five years ago, we be­gan col­lab­o­rat­ing with the Uni­ver­si­ty of Penn­syl­va­nia and in­vest­ed in fur­ther de­vel­op­ing and bring­ing what we be­lieved would be a par­a­digm-chang­ing im­muno­cel­lu­lar ther­a­py to can­cer pa­tients in dire need. With the ap­proval of Kym­ri­ah, we are once again de­liv­er­ing on our com­mit­ment to change the course of can­cer care.

This ap­proval marks a ma­jor shift in on­col­o­gy, with a tru­ly rev­o­lu­tion­ary ap­proach to treat­ing can­cer. That hasn’t es­caped the no­tice of big play­ers and small, with a host of de­vel­op­ers look­ing to do much bet­ter, more safe­ly, with new drugs in the pipeline.

It’s a big day in the biotech world, but it’s just the of­fi­cial start date for a new era.

Biotech and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

No­var­tis' sec­ond at­tempt to repli­cate a stun­ning can­cer re­sult falls flat

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

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AstraZeneca CEO Pascal Soriot (Raphael Lafargue/Abaca/Sipa USA)

A com­bo of As­traZeneca's Imfinzi and chemo wins where oth­ers have failed in piv­otal bil­iary tract test

Looking to run with the big dogs in the PD-(L)1 class, AstraZeneca’s Imfinzi has a tall hill to climb to compete in an increasingly bustling market. An aggressive combo strategy for the drug has paid off so far, and now AstraZeneca is adding another notch to its belt.

A combo of Imfinzi (durvalumab) and chemotherapy significantly extended the lives of first-line patients with advanced biliary tract cancer over chemo alone, according to topline results from the Phase III TOPAZ-1 study revealed Monday.

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Sean Ianchulev, Eyenovia CEO and CMO

Re­cent court de­ci­sion push­es FDA to re­ject and re­clas­si­fy drug-de­vice com­bo, crush­ing shares

Back in April, the FDA lost a crucial court case in which its broad discretion of regulating medical products that might satisfy the legal definitions of either “drug” and/or “medical device” was sharply curtailed.

In addition to the appeals court ruling that Genus Medical Technologies’ contrast agent barium sulfate (aka Vanilla SilQ) should not be considered a drug, as the FDA had initially ruled, but as a medical device, the agency also was forced to spell out which drugs would transition to devices as a result of the ruling.

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Robert Califf (Pablo Martinez Monsivais, AP Images, File)

As buzz on Califf FDA nom heats up, in­dus­try and agency in­sid­ers of­fer a strong nod for the ‘per­fect’ choice

For once in this long, dramatic road to finding a new FDA commissioner, there’s been some continuity. Both CNN and Politico reported this weekend that Rob Califf met with President Biden to discuss the permanent commish role, following earlier news broken by the Washington Post that all signs point to Califf.

Although there may be a few Democrats who continue to grandstand about the dangers of COI (Califf has worked for Verily, sits on the board of Centessa Pharmaceuticals, and has other ties to industry research), with the pandemic ongoing and the need for some kind of continuity at FDA mounting, Califf is likely to meet the same fate as when he first won Senate confirmation in 2016, by a vote of 89-4 — Bernie Sanders and 6 others didn’t vote.

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Sim­mer­ing feud be­tween blue­bird and a ri­val con­tin­ues with al­le­ga­tions of patent fraud

Bluebird bio was hit with a patent infringement lawsuit last week from a Chicago-based biotech it has had an ongoing beef with calling for $2 billion to help cure the “irreparable harm” caused by alleged willful infringement.

Bluebird bio is facing a lawsuit from Errant Gene Therapeutics for violating patent law in two instances, the company says.

The suit alleges that bluebird infringed the rights of EGT’s recombinant vectors used in the gene therapy treatment of rare diseases such as sickle cell disease and beta thalassemia for its drugs Zynteglo and LentiGlobin. EGT has an exclusive license from the Memorial Sloan Kettering Cancer Center to patents titled “vector encoding human global gene and use thereof in treatment of Hemoglobinopathies.”

Sanofi, Re­gen­eron gear up for new Dupix­ent fil­ing af­ter clear­ing sec­ond PhI­II for eosinophilic esophagi­tis

This fall’s harvest is proving fruitful for Dupixent.

Days after touting a clean Phase III sweep in prurigo nodularis, Sanofi and Regeneron report that their blockbuster anti-inflammatory drug has passed another Phase III test with flying colors, paving the way for regulatory filings in 2022.

It’s the second trial where Dupixent has proven effective against eosinophilic esophagitis, meeting the co-primary endpoints by spurring significant improvements in both clinical and histologic disease measures.

Peter Greenleaf, Aurinia CEO

Af­ter pass­ing on Ac­celeron, Bris­tol My­ers eyes bolt-on ac­qui­si­tion of au­toim­mune spe­cial­ist — re­port

Bristol Myers Squibb is looking to beef up its autoimmune portfolio by scooping up Aurinia Pharmaceuticals, Bloomberg reported.

The recent overtures to Aurinia, relayed by anonymous insiders, came just as Bristol Myers turned down buyout talks with partners at Acceleron — which Merck ultimately struck a deal to acquire for $11.5 billion. Bristol Myers has reportedly decided to cash out on its minority stake, likely bagging $1.3 billion in the process, while keeping the royalty deals on two of Acceleron’s blood disorder drugs.

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So — that pig-to-hu­man trans­plant; Po­ten­tial di­a­betes cure reach­es pa­tient; Ac­cused MIT sci­en­tist lash­es back; and more

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