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.

The Federal Trade Commission has gotten involved in a patent feud over Supernus’ Parkinson’s drug Apokyn, a case the agency said may have ‘‘significant implications” for patients who rely on the drug.

Sage Chemical won the first generic approval for its Apokyn formulation (also known as apomorphine hydrochloride injection) back in 2022. The non-ergoline dopamine agonist is approved to treat Parkinson’s symptoms during “off episodes,” such as difficulty moving, tremors and intense cramping. However, regulators specified that the approval pertained to the generic drug cartridges only, not the injector pen required for administration.

The Brussels-based European Federation of Pharmaceutical Industries and Associations (EFPIA) this morning called on the European Commission to ensure that it adequately assesses how its re-writing of the 20+-year-old pharma legislation, which is due to be released before the end of the month, damages the competitiveness of the pharma industry.

EFPIA’s opposition to the changes has lingered since the beginning, and its latest estimate is that the rewritten legislation could cost the industry €640 million ($688 million).

The White House’s Office of Science and Technology Policy (OSTP) last year sought to find ways to better coordinate large-scale clinical trials in the US — as the UK lead by example during the pandemic — especially for these emergency clinical trials.

The lobbying group PhRMA Tuesday called for more clinical trial diversity in underserved areas, including by making participation less of a burden, and expanding eligibility criteria when appropriate.