Sanofi grap­ples with re­jec­tion of po­ten­tial first-in-class or­phan drug, faults third-par­ty man­u­fac­tur­ing

A snap re­view didn’t lead to a quick OK for Sanofi’s su­tim­limab. A day af­ter the FDA’s sup­posed de­ci­sion date on the or­phan drug, the French phar­ma gi­ant said it was hand­ed a re­jec­tion.

The com­plete re­sponse let­ter cites “cer­tain de­fi­cien­cies iden­ti­fied by the agency dur­ing a pre-li­cense in­spec­tion of a third-par­ty fa­cil­i­ty re­spon­si­ble for man­u­fac­tur­ing,” Sanofi wrote. Reg­u­la­tors ap­par­ent­ly didn’t take any is­sue with the clin­i­cal da­ta or safe­ty.

Picked up in the $11.6 bil­lion Biover­a­tiv ac­qui­si­tion, the drug is a C1s in­hibitor tar­get­ing cold ag­glu­tinin dis­ease — a rare dis­or­der where the im­mune sys­tem runs amok at­tack­ing healthy red blood cells. Pa­tients can suf­fer from se­vere ane­mia and life-threat­en­ing throm­bot­ic events.

Just months be­fore it was ac­quired Biover­a­tiv, a Bio­gen spin­out orig­i­nal­ly fo­cused on he­mo­phil­ia, had dished out $825 mil­lion to buy out the orig­i­nal de­vel­op­er of the drug. True North Ther­a­peu­tics had al­ready scored break­through ther­a­py des­ig­na­tion through the back­ing of Or­biMed Ad­vi­sors, Klein­er Perkins Caulfield & By­ers, MPM Cap­i­tal, SR One and Bax­ter Ven­tures.

Sit­ting at the in­ter­sec­tion of im­munol­o­gy, rare dis­eases and rare blood dis­or­ders, su­tim­limab fits nice­ly with­in the kind of spe­cial­ty care fo­cus that CEO Paul Hud­son laid out last year as he chopped the lega­cy pipeline in di­a­betes and car­dio. R&D chief John Reed has al­so tout­ed it as a first-in-class tar­get­ed ther­a­py that “has the po­ten­tial to change the treat­ment par­a­digm for CAD.”

It cleared a small piv­otal study in­volv­ing 24 pa­tients, meet­ing the com­pos­ite end­point mea­sur­ing he­mo­glo­bin and need for trans­fu­sion while im­prov­ing oth­er met­rics of he­mol­y­sis, ane­mia and fa­tigue.

The dura­bil­i­ty por­tion of the study is on­go­ing — al­though Sanofi said it won’t fac­tor in­to the cur­rent BLA.

“Sat­is­fac­to­ry res­o­lu­tion of the ob­ser­va­tions by the third-par­ty man­u­fac­tur­er is re­quired be­fore the BLA can be ap­proved and Sanofi re­mains in close con­tact with the FDA and the third-par­ty man­u­fac­tur­er to reach a res­o­lu­tion in a time­ly man­ner,” it wrote.

Biogen CEO Michel Vounatsos (via Getty Images)

With ad­u­canum­ab caught on a cliff, Bio­gen’s Michel Vounatsos bets bil­lions on an­oth­er high-risk neu­ro play

With its FDA pitch on the Alzheimer’s drug aducanumab hanging perilously close to disaster, Biogen is rolling the dice on a $3.1 billion deal that brings in commercial rights to one of the other spotlight neuro drugs in late-stage development — after it already failed its first Phase III.

The big biotech has turned to Sage Therapeutics for its latest deal, close to a year after the crushing failure of Sage-217, now dubbed zuranolone, in the MOUNTAIN study.

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Pascal Soriot (AP Images)

As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

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John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

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Leonard Schleifer, Regeneron CEO (Andrew Harnik/AP)

Trail­ing Eli Lil­ly by 12 days, Re­gen­eron gets the FDA OK for their Covid-19 an­ti­body cock­tail

A month and a half after becoming the experimental treatment of choice for a newly diagnosed president, Regeneron’s antibody cocktail has received emergency use authorization from the FDA. It will be used to treat non-hospitalized Covid-19 patients who are at high-risk of progressing.

Although the Rgeneron drug is not the first antibody treatment authorized by the FDA, the news comes as a significant milestone for a company and a treatment scientists have watched closely since the outbreak began.

Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

Af­ter Ko­dak de­ba­cle, US lends $1.1B to a syn­thet­ic bi­ol­o­gy com­pa­ny and their big Covid-19, mR­NA plans

In mid-August, as Kodak’s $765 million government-backed push into drug manufacturing slowly fell apart in national headlines, Ginkgo Bioworks CEO Jason Kelly got a message from his company’s government liaison: HHS wanted to know if they, too, might want a loan.

The government’s decision to lend Kodak three quarters of a billion dollars raised eyebrows because Kodak had never made drugs before. But Ginkgo, while not a manufacturing company, had spent the last decade refining new ways to produce materials inside cells and building automated facilities across Boston.

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FDA hands Liq­uidia and Re­vance a CRL and de­fer­ral, re­spec­tive­ly, as Covid-19 cre­ates in­spec­tion chal­lenge

Two biotechs said they got turned away by the FDA on Wednesday, in part due to pandemic-related travel restrictions.

North Carolina-based Liquidia Technologies was handed a CRL for its lead pulmonary arterial hypertension drug, citing the need for more CMC data and on-site pre-approval inspections, which the FDA hasn’t been able to conduct due to travel restrictions. The agency also deferred its decision on Revance Therapeutics’ BLA for its frown line treatment, because it needs to inspect the company’s northern California manufacturing facility. The action, Revance emphasized, was not a CRL.

The flu virus (CDC)

Roche tacks on an­oth­er Xofluza in­di­ca­tion as flu sea­son meets pan­dem­ic

Xofluza was heralded as the first new flu drug in 20 years when it got the FDA OK back in 2018. But even so, Roche saw tough competition from cheaper Tamiflu generics that appeared to be nearly as — if not just as — effective.

Now, the pharma says the drug also can be used to prevent influenza after exposure, snagging a new approval and adding to Xofluza’s appeal as flu season meets the pandemic.

Bob Nelsen (Photo by Michael Kovac/Getty Images)

Bob Nelsen rais­es $800M and re­cruits a star-stud­ded board to build the 'Fox­con­n' of biotech

Bob Nelsen spent his pandemic spring in his Seattle home, talking on the phone with Luciana Borio, the scientist who used to run pandemic preparedness on the National Security Council, and fuming with her about the dire state of American manufacturing.

Companies were rushing to develop vaccines and antibodies for the new virus, but even if they succeeded, there was no immediate supply chain or infrastructure to mass-produce them in a way that could make a dent in the outbreak.

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