Paul Hudson, Sanofi CEO (Raphael Lafargue/Abaca/Sipa USA; Sipa via AP Images)

Sanofi keeps nip­ping at J&J's heels as Sar­clisa notch­es new FDA ap­proval in mul­ti­ple myelo­ma

The mul­ti­ple myelo­ma ri­val­ry be­tween Sanofi and J&J con­tin­ued Wednes­day evening, with the French drug­mak­er’s Sar­clisa tak­ing home a new ap­proval in the in­di­ca­tion.

US reg­u­la­tors OK’d the drug in com­bi­na­tion with Am­gen’s Kypro­lis and the steroid dex­am­etha­sone for adults with re­lapsed or re­frac­to­ry mul­ti­ple myelo­ma who have re­ceived be­tween one and three pre­vi­ous lines of treat­ment. It’s the sec­ond ap­proval for Sar­clisa, hav­ing pre­vi­ous­ly won a green light in March 2020 as a third-line treat­ment for the dis­ease.

And with the ex­pand­ed la­bel now in hand, Sar­clisa is once again tak­ing aim at J&J’s mar­ket lead­ing Darza­lex.

First ap­proved in 2015 for treat­ment-re­sis­tant mul­ti­ple myelo­ma, Darza­lex quick­ly be­came a block­buster and gen­er­at­ed $1.4 bil­lion of sales in its first year. It has since steadi­ly ex­pand­ed its mar­ket, most re­cent­ly earn­ing an OK last Au­gust with the same cock­tail and same in­di­ca­tion as Sar­clisa did Wednes­day.

Last year, Darza­lex tal­lied its best year yet with near­ly $4.2 bil­lion in sales across all in­di­ca­tions, ac­cord­ing to J&J and de­vel­op­er Gen­mab.

Sar­clisa, mean­while, is still find­ing its foot­ing in the mar­ket, hav­ing gained its first ap­proval a mere nine days be­fore the WHO de­clared a glob­al pan­dem­ic for Covid-19. The drug pulled in just over $50 mil­lion in 2020 sales. But the drug still marked a mile­stone for Sanofi, as Sar­clisa was the French phar­ma’s first in-house can­cer ap­proval since Jev­tana in 2010.

Both drugs are mon­o­clon­al an­ti­bod­ies that fo­cus on CD38, which is high­ly ex­pressed on mul­ti­ple myelo­ma cells, but func­tion slight­ly dif­fer­ent­ly. Darza­lex locks on to the CD38 mol­e­cule, while Sar­clisa is en­gi­neered to tar­get a spe­cif­ic por­tion of the anti­gen found on the pro­tein.

Wednes­day’s Sar­clisa OK came on the ba­sis of a 302-pa­tient Phase III tri­al, which showed the com­bo re­duced the risk of dis­ease pro­gres­sion or death by 45% com­pared to the Kypro­lis/dex­am­etha­sone stan­dard of care. That clocked in at a p-val­ue of p=0.0032.

Though cross-tri­al com­par­isons are nev­er a per­fect sci­ence, that fig­ure came in nu­mer­i­cal­ly high­er than the foun­da­tion for Darza­lex’s ap­proval in the same in­di­ca­tion. The Darza­lex com­bo with Kypro­lis and dex­am­etha­sone on­ly re­duced that risk by 37%.

Sar­clisa did miss on a sec­ondary end­point, how­ev­er, fail­ing to reg­is­ter a sta­tis­ti­cal­ly sig­nif­i­cant dif­fer­ence in over­all re­sponse rate. Those num­bers re­mained sim­i­lar for each arm: 86.6% for the Sar­clisa com­bo ver­sus 82.9% for stan­dard of care.

Go­ing for­ward, Sanofi is ex­pect­ing a de­ci­sion in this in­di­ca­tion from the EU “in the com­ing months,” and re­ceived a pos­i­tive CHMP opin­ion in Feb­ru­ary. Re­searchers con­tin­ue to look at Sar­clisa across the mul­ti­ple myelo­ma land­scape as well, as the phar­ma says it’s en­gaged in sev­er­al on­go­ing Phase III tri­als. Sanofi is al­so in­ves­ti­gat­ing the drug for the treat­ment of oth­er hema­to­log­ic ma­lig­nan­cies and sol­id tu­mors.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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Sijmen de Vries, Pharming CEO

FDA ap­proves Pharm­ing drug for ul­tra-rare im­mun­od­e­fi­cien­cy dis­ease

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.

No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

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Ly­me vac­cine test com­ple­tion is pushed back by a year as Pfiz­er, Val­ne­va say they'll ad­just tri­al

Valneva and Pfizer have adjusted the end date for the Phase III study of their investigational Lyme disease vaccine, pushing it back by a year after issues at a contract researcher led to thousands of US patients being dropped from the test.

In a March 20 update to, Valneva and Pfizer moved the primary completion date on the trial, called VALOR, from the end of 2024 to the end of 2025.

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Stuart Peltz, former PTC Therapeutics CEO

Stu­art Peltz re­signs as PTC Ther­a­peu­tics CEO af­ter 25 years

Stuart Peltz, the longtime CEO of PTC Therapeutics who’s led the rare disease drug developer since its founding 25 years ago, is stepping down.

Succeeding him in the top job is Matthew Klein, who joined PTC in 2019 and was promoted to chief operating officer in 2022. In a call with analysts, he said the CEO transition has been planned for “quite some time” — in fact, as part of it, he gave the company’s presentation at the JP Morgan healthcare conference earlier this year.

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Bet­ter Ther­a­peu­tics cuts 35% of staff while await­ing dig­i­tal ther­a­peu­tic ap­proval

Digital therapeutics company Better Therapeutics announced on Thursday that it’s cutting 35% of its staff as it awaits FDA clearance for its first product.

The company, which launched eight years ago, is one of a growing group of companies seeking a digital alternative to traditional medicine. The space saw a record $7.5 billion in investments in 2021, according to Chris Dokomajilar at DealForma, with uses spanning ADHD, PTSD and other indications. However, private insurers have been slow to hop on board.

FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.