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%.

Inside FDA HQ (File photo)

The FDA just ap­proved the third Duchenne MD drug. And reg­u­la­tors still don’t know if any of them work

Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

On Wednesday the FDA approved the third Duchenne MD drug, based on the same biomarker. And regulators were ready to act yet again despite the lack of efficacy data.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 87,700+ biopharma pros reading Endpoints daily — and it's free.

Franz-Werner Haas, CureVac CEO

UP­DAT­ED: On the heels of a snap $1B raise, Cure­Vac out­lines plans to seek emer­gency OK for their Covid-19 vac­cine in a mat­ter of months

CureVac is going from being one of the quietest players in the race to develop a new vaccine to fight the worst public health crisis in a century to a challenger for the multibillion-dollar market that awaits the first vaccines to make it over the finish line. Typically low-key at a time of brash comments and incredibly ambitious development timelines from the leaders, CureVac now is jumping straight into the spotlight.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 87,700+ biopharma pros reading Endpoints daily — and it's free.

Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Sanofi vet Kather­ine Bowdish named CEO of PIC Ther­a­peu­tics; As the world Terns: Liv­er dis­ease biotech makes ex­ec­u­tive changes

PIC Therapeutics hasn’t raised much money, yet. But the fledgling biotech has attracted a high-profile player to the helm.

The Boston-based biotech has handed the reins to Katherine Bowdish as its president and CEO. Bowdish will also join the board of directors of PIC. Bowdish joins from Sanofi where she served as VP and head of R&D strategy, as well as helping launch and lead Sanofi Sunrise, a venture investment and partnering vehicle at Sanofi. Before that, Bowdish held several exec roles at Permeon Biologics, Anaphore, Alexion Pharmaceuticals and Prolifaron (acquired by Alexion).

Martin Shkreli (Shutterstock)

Mar­tin Shkre­li con­tin­ued to or­ches­trate an­ti-com­pet­i­tive schemes for Dara­prim be­hind bars — FTC

Martin Shkreli didn’t just blog, read up on drug development news and run his biotech business with a contraband cell phone in prison. According to the FTC, he was also coordinating the anticompetitive scheme to shield Daraprim — the drug at the center of a price-gouging controversy that earned him the “Pharma Bro” nickname — from generic rivals.

Back in January the FTC, together with New York’s attorney general, launched a federal lawsuit against Shkreli, who’s now serving a 7-year sentence for defrauding investors in his hedge fund, alleging that he effectively created a drug monopoly. While Shkreli’s notorious move to raise the per tablet price of Daraprim from $17.50 to $750 was perfectly legal, the tactics he allegedly deployed to box out competitors weren’t.

NIH director Francis Collins at a Senate Appropriations subcommittee hearing for Operation Warp Speed (Graeme Jennings/Pool via AP Images)

Covid-19 roundup: As­traZeneca signs 400M vac­cine sup­ply deal with EU; 'No­vem­ber or De­cem­ber' Collins' best bet on a vac­cine OK

Amid talks with multiple players, the European Commission has reached its first vaccine supply deal with AstraZeneca, securing 400 million doses of the Oxford candidate for all of its member countries.

The pharma giant said in a press release that the deal builds on the existing agreement with Germany, France, Italy and the Netherlands, announced in June. It was unclear, however, whether that means simply extending the same 400 million doses to all EU countries or doubling the reservation to 800 million doses.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 87,700+ biopharma pros reading Endpoints daily — and it's free.

James Wilson, WuXi Global Forum at JPM20

FDA puts up a red light for Pas­sage Bio’s first gene ther­a­py pro­gram, de­lay­ing a pro­gram from James Wilson's group at Penn

Gene therapy pioneer James Wilson spearheaded animal studies demonstrating the potential of new treatments injected directly into the brain, looking to jumpstart a once-and-done fix for an extraordinarily rare disease called GM1 gangliosidosis in infants. His team at the University of Pennsylvania published their work on monkeys and handed it over to Passage Bio, a Wilson-inspired startup building a pipeline of gene therapies — with an IND for PBGM01 to lead the way.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 87,700+ biopharma pros reading Endpoints daily — and it's free.

Silviu Itescu, Mesoblast CEO

FDA's ODAC shrugs off ob­jec­tions to Mesoblast's GVHD drug for chil­dren, vot­ing 9-1 in fa­vor and im­prov­ing the odds of an ap­proval

The FDA’s Oncologic Drugs Advisory Committee once again waved through an investigational drug, clearing the potential final hurdle before the agency’s decision.

Thursday’s winner was Mesoblast $MESO, an Australian stem-cell company that submitted its Ryoncil drug for the treatment of steroid refractory acute graft-versus-host disease in children younger than 12. ODAC gave Ryoncil the thumbs up by a 9-1 vote, shrugging off concerns about trial design and pushing aside the need for an additional study.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 87,700+ biopharma pros reading Endpoints daily — and it's free.

Cal­lid­i­tas bets up to $102M on a biotech buy­out, snag­ging a once-failed PBC drug

After spending years developing its oral formulation of the corticosteroid budesonide, Sweden’s Calliditas now has its sights set on the primary biliary cholangitis field.

The company will buy out France-based Genkyotex, and it’s willing to bet up to €87 million ($102 million) that Genkyotex’s failed Phase II drug, GKT831, will do better in late-stage trials.

Under the current agreement, Calliditas $CALT will initially pay €20.3 million in cash for 62.7% of Genkyotex (or €2.80 a piece for 7,236,515 shares) in early October, then circle back for the rest of Genkyotex’s shares under the same terms. If nothing changes, the whole buyout will cost Calliditas €32.3 million, plus up to  €55 million in contingent rights.