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.

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

Pfiz­er, Sarep­ta and two oth­ers sug­gest Duchenne drug safe­ty is­sues tied to "class ef­fect"

Since the first experimental Duchenne gene therapy programs came about, the space has proven rife with safety issues and patient deaths in clinical trials. Pfizer and three biotechs now think they’ve found a reason why.

The four companies suggested there may be a “class effect” causing the adverse events in Duchenne gene therapies, they wrote in a new study. They specifically highlighted how side effects in five patients across three trials, who all showed muscle weakness with cardiac involvement, were “strikingly similar.”

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Pre­sent­ing a live End­points News event: Man­ag­ing a biotech in tur­bu­lent times

Biotech is one of the smartest, best educated industries on the planet. PhDs abound. We’ve had a long enough track record to see a new generation of savvy, experienced execs coming together to run startups.

And in these times, they are being tested as never before.

Biotech is going through quite a rough patch right now. For 2 years, practically anyone with a decent resume and some half-baked ideas on biotech could start a company and get it funded. The pandemic made it easy in many ways to pull off an IPO, with traditional road shows shut down in exchange for a series of quick Zoom meetings. Generalist investors flocked as the numbers raised soared into the stratosphere.

De­spite fed­er­al ef­forts to di­ver­si­fy clin­i­cal tri­als, progress re­mains 'stag­nan­t' — re­port

While calls to diversify clinical trials have grown louder in recent years — gaining support from federal agencies such as the FDA and NIH — progress has largely stalled, according to a new report from the National Academies of Sciences, Engineering and Medicine.

Swaths of patients in racial and ethnic minority groups, as well as LGBTQIA+, pregnant and older adult populations continue to be left out of clinical trials. While some advances have been made in the last 30 years — women now account for roughly half of clinical trial participants — growth in other areas remains stagnant, according to the report, which was mandated by Congress and sponsored by the NIH.

Paul Chaplin, Bavarian Nordic president and CEO

Bavar­i­an Nordic se­cures BAR­DA con­tract for small­pox vac­cine

It seems that smallpox vaccination production is weighing on the mind of the US government. And manufacturer Bavarian Nordic is the latest company to benefit.

Just a few days after Emergent, a company that has made government contracts its lifeblood, acquired the exclusive rights to Tembexa from Chimerix, with a $225 million cash payment and an expected BARDA contract, the agency has offered a contract for smallpox vaccine production.

Lina Khan, FTC chair (Saul Loeb/Pool via AP)

New FTC com­mis­sion­er could turn the tide for an in­ves­ti­ga­tion in­to PBMs

The Senate last week voted along party lines, 51-50, with Vice President Kamala Harris casting the tie-breaker, to make President Biden appointee Alvaro Bedoya the deciding vote on a split 2-2 Federal Trade Commission.

The addition of Bedoya to the FTC could not only spell more trouble for biopharma M&A activity, as he may align with his Democrat partners to break the FTC ties, but it may also mean that FTC Chair Lina Khan has what she needs to move forward on a study around the pharma middlemen known as pharmacy benefit managers.

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

Sen­ate user fee reau­tho­riza­tion bill omits ac­cel­er­at­ed ap­proval re­forms, shows wide gaps with House ver­sion

The Senate health committee on Tuesday released its first version of the bill to reauthorize all the different FDA user fees. But unlike the House version, there are only a few controversial items in the Senate’s version, which does not address either accelerated approval reforms or clinical trial diversity (as the House did).

While it’s still relatively early in the process of finalizing this legislation (the ultimate statutory deadline is the end of September), the House and Senate, at least initially, appear to be starting off in different corners on what should be included.

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Warren Buffett, Berkshire Hathaway CEO

Berk­shire Hath­away pulls out of Ab­b­Vie, Bris­tol My­ers Squibb in­vest­ments

It looks like Warren Buffett is sticking to ice cream and railroads for the moment.

The billionaire CEO of Berkshire Hathaway backed out of two major holdings in the pharma industry, Forexlive first reported, including a $410 million investment in AbbVie and a $324.4 million stake in Bristol Myers Squibb.

The move comes after Berkshire abandoned its Teva shares just last quarter, Bloomberg reported.

Long-ex­pect­ed UK lay­offs im­mi­nent for No­var­tis fol­low­ing sale

Nearly a year ago, more than 200 workers at Novartis’ Grimsby, UK, facility were able to hang on to their jobs after the pharma closed a Switzerland site as a part of its workforce restructuring plan. Now, it looks like those employees’ time is up, as the site has been sold, Grimsby Telegraph reported today.

The manufacturing site has been sold to Humber Industrials, a subsidiary of International Process Plants. None of the current staff members will be working with the new owners, however.