CMS will now cov­er pricey CAR-T ther­a­pies for Medicare pa­tients

The Cen­ters for Medicare and Med­ic­aid (CMS) has been busy. Ear­li­er this week, the agency pre­scribed ways to re­sus­ci­tate the fledg­ling an­tibi­ot­ic in­dus­try by re­struc­tur­ing the pay­ment ap­pa­ra­tus for new an­tibi­otics. On Wednes­day, CMS un­veiled its de­ci­sion to cov­er CAR-T can­cer ther­a­pies — which can cost up to $1 mil­lion per pa­tient — for Medicare ben­e­fi­cia­ries.

The cell ther­a­pies — No­var­tis’ $NVS Kym­ri­ah and Gilead’s $GILD Yescar­ta — se­cured FDA ap­proval in 2017 for blood can­cers, in­clud­ing lym­phoma and leukemia.

These prod­ucts re­quire the man­u­fac­tur­ers to first iso­late cells from the pa­tient, ma­nip­u­late them in the lab by adding chimeric anti­gen re­cep­tors to di­rect T cells to snuff out can­cer cells and then re-in­fuse them back in­to the pa­tient. The price tags car­ried by the ther­a­pies re­flect that com­plex per­son­al­ized man­u­fac­tur­ing process.

The CMS’ de­ci­sion will help cov­er the ther­a­pies in health­care fa­cil­i­ties that have the in­fra­struc­ture in place to track pa­tient out­comes. CMS ad­min­is­tra­tor Seema Ver­ma in a re­cent in­ter­view with STAT sug­gest­ed that the agency had strug­gled to come up with a CAR-T spe­cif­ic re­im­burse­ment pol­i­cy part­ly be­cause by law it re­quires a cer­tain amount of pa­tient out­comes da­ta that so far has not been avail­able. By agree­ing to cov­er costs for hos­pi­tals that will track pa­tients, the agency is in a po­si­tion to kill two birds with one stone.

Un­til now, the CMS was help­ing hos­pi­tals with new tech­nol­o­gy add-on pay­ments (NTAP) — a pro­vi­sion ush­ered in 2000 to help smooth the en­try for fresh prod­ucts while a spe­cif­ic re­im­burse­ment par­a­digm was be­ing worked out for them. NTAPs are usu­al­ly capped at 50% but last week the CMS raised that bar to 65% for CAR-T ther­a­pies — al­though the move was char­ac­ter­ized in­ad­e­quate. In re­sponse, the Amer­i­can So­ci­ety of Hema­tol­ogy said it was dis­ap­point­ed as it had re­quest­ed the CMS to in­crease this pay­ment to 80%.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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FDA to Sarep­ta: Your wide­ly an­tic­i­pat­ed fol­lowup to Ex­ondys 51 is not get­ting an ac­cel­er­at­ed OK for Duchenne MD

In one of the least anticipated moves of the year, the FDA has rejected Sarepta’s application for an accelerated approval of its Duchenne MD drug golodirsen after fretting over safety issues.

In a statement that arrived after the bell on Monday, Sarepta explained the CRL, saying:

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Novartis CEO Vas Narasimhan [via Bloomberg/Getty]

I’m not per­fect: No­var­tis chief Vas Narasimhan al­most apol­o­gizes in the wake of a new cri­sis

Vas Narasimhan has warily stepped up with what might pass as something close to a borderline apology for the latest scandal to engulf Novartis.

But he couldn’t quite get there.

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Levi Garraway. Broad Institute via Youtube

Roche raids Eli Lil­ly for its next chief med­ical of­fi­cer as San­dra Horn­ing plans to step down

We found out Monday morning where Levi Garraway was headed after he left Eli Lilly as head of oncology R&D a few days ago. Roche named Garraway as their new chief medical officer, replacing Sandra Horning, who they say is retiring from the company.

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Af­ter a posse of Wall Street an­a­lysts pre­dict a like­ly new win for Sarep­ta, we're down to the wire on a crit­i­cal FDA de­ci­sion

As Bloomberg notes, most of the Wall Street analysts that cover Sarepta $SRPT are an upbeat bunch, ready to cheer on the team when it comes to their Duchenne MD drugs, or offer explanations when an odd setback occurs — as happened recently with a safety signal that was ‘erroneously’ reported last week.

Ritu Baral Cowen
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UP­DAT­ED: No­var­tis spin­off Nabri­va fi­nal­ly scores its first an­tibi­ot­ic ap­proval

In May, Nabriva Therapeutics suffered a setback after the FDA rejected its antibiotic for complicated urinary tract infections — the Novartis spinoff has now had some better luck with the US agency, which on Monday approved its other drug for community-acquired bacterial pneumonia.

The drug, lefamulin, has been developed as an intravenous and oral formulation and been tested in two late-stage clinical trials. The semi-synthetic compound, whose dosing can be switched between the two formulations, is engineered to inhibit the synthesis of bacterial protein by binding to a part of the bacterial ribosome.

Saqib Islam. CheckRare via YouTube

Spring­Works seeks $115M to push Pfiz­er drugs across fin­ish line while Sat­suma sells mi­graine play in $86M IPO

SpringWorks and Satsuma — both biotech spinouts that have closed B rounds in April — are loading up with IPO cash to boost their respective late-stage plans.
SpringWorks

Bain-backed SpringWorks is the better-known company of the two, and it’s gunning for a larger windfall of $115 million to add to $228 million from previous financings. In the process, the Stamford, CT-based team is also drawing the curtains on the partnerships it has in mind for the pair of assets it had initially licensed from Pfizer.

Mi­nor­i­ty racial groups con­tin­ue to be dis­mal­ly rep­re­sent­ed in can­cer tri­als — study

Data reveal that different racial and ethnic groups — by nature and/or nurture — can respond differently in terms of pharmacokinetics, efficacy, or safety to therapeutics, but this disparity is not necessarily accounted for in clinical trials. A fresh analysis of the last decade of US cancer drug approvals suggests the trend continues, cementing previous research that suggests oncology trials are woefully under-representative of the racial makeup of the real world.

Van­da shares slide af­ter FDA spurns their big end­point and re­jects a pitch on jet lag re­lief

Back in the spring of last year, Vanda Pharmaceuticals $VNDA served up a hot stew of mixed data for a slate of endpoints related to what they called clear evidence that their melatonin sleep drug Hetlioz (tasimelteon) could help millions of travelers suffering from jet lag.

Never mind that they couldn’t get a planned 90 people in the study, settling for 25 instead; Vanda CEO Mihael H. Polymeropoulos said they were building on a body of data to prove it would help jet-lagged patients looking for added sleep benefits. And that, they added, would be worth a major upgrade from the agency as they sought to tackle a big market.