OPDP let­ter crit­i­cizes drug com­pa­ny for fail­ing to dis­close risk in­fo on­line

The FDA Of­fice of Pre­scrip­tion Drug Pro­mo­tion (OPDP) on Mon­day un­veiled a new un­ti­tled let­ter sent last week to Michi­gan-based Rock­well Med­ical for a web­page that on­ly presents in­for­ma­tion about the ben­e­fits of its drug Trifer­ic (fer­ric py­rophos­phate cit­rate).

In OPDP’s 7th un­ti­tled let­ter of the year (two warn­ing let­ters were al­so is­sued), the of­fice ex­plains how the com­pa­ny omit­ted the risks as­so­ci­at­ed with Trifer­ic on its web­site, which means it failed to pro­vide ma­te­r­i­al in­for­ma­tion about the con­se­quences that may re­sult from the use of the drug and cre­at­ed a mis­lead­ing im­pres­sion about the safe­ty of Trifer­ic.

OPDP al­so took is­sue with three claims that the com­pa­ny made that sug­gest Trifer­ic is safer and more ef­fec­tive than oth­er IV iron re­place­ment prod­ucts.

“No ref­er­ences were cit­ed to sup­port these claims, and FDA is not aware of da­ta to sup­port claims that Trifer­ic is safer or more ef­fec­tive com­pared to oth­er IV iron re­place­ment prod­ucts. While we ac­knowl­edge that Trifer­ic is the on­ly FDA-ap­proved iron re­place­ment prod­uct ad­min­is­tered via he­modialysate, once in­side the blood stream, iron de­liv­ered by Trifer­ic is used by the body in the same man­ner as oth­er cur­rent­ly ap­proved iron re­place­ment prod­ucts,” the let­ter says.

The com­pa­ny al­so did not dis­close the full in­di­ca­tion and lim­i­ta­tions of use as­so­ci­at­ed with Trifer­ic.

“This is par­tic­u­lar­ly con­cern­ing giv­en claims made on the web­page such as ‘Trifer­ic im­proves the ef­fec­tive­ness of iron de­liv­ery for the ma­jor­i­ty of dial­y­sis pa­tients. . .’ (em­pha­sis added). This broad claim sug­gests that Trifer­ic is in­di­cat­ed for pa­tients re­ceiv­ing any type of dial­y­sis, when it is not in­tend­ed for use in pa­tients re­ceiv­ing peri­toneal dial­y­sis and its safe­ty and ef­fec­tive­ness for use in pa­tients re­ceiv­ing home he­modial­y­sis have not been stud­ied,” the agency adds.

The FDA al­so notes that pa­tients tak­ing Trifer­ic may be at an in­creased risk of com­pli­ca­tions, which means the com­pa­ny’s vi­o­la­tions are even more con­cern­ing from a pub­lic health per­spec­tive.

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BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Gilead re­leas­es an­oth­er round of murky remde­sivir re­sults

A month after the NIH declared the first trial on remdesivir in Covid-19 a success, Gilead is out with new results on their antiviral. But although the study met one of its primary endpoints, the data are likely to only add to a growing debate over how effective the drug actually is.

In a Phase III trial, patients given a 5-day dose of remdesivir were 65% more likely to show “clinical improvement” compared to an arm given standard-of-care. The trial, though, gave little indication for whether the drug had an impact on key endpoints such as survival or time-to-recovery. And in a surprising twist, a 10-day dosing arm of remdesivir didn’t lead to a statistically significant improvement over standard of care.

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Fangliang Zhang (Imaginechina via AP Images)

The big mon­ey: Poised to make drug R&D his­to­ry, a Chi­na biotech un­veils uni­corn rac­ing am­bi­tions in a bid to raise $350M-plus on Nas­daq

Almost exactly three years after Shanghai-based Legend came out of nowhere to steal the show at ASCO with jaw-dropping data on their BCMA-targeted CAR-T for multiple myeloma, the little player with Big Pharma connections is taking a giant step toward making it big on Wall Street. And this time they want to seal the deal on a global rep after staking out a unicorn valuation in what’s turned out to be a bull market for biotech IPOs — in the middle of a pandemic.

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Len Schleifer (left) and George Yancopoulos, Regeneron (Vimeo)

Eyes on he­mo­phil­ia prize, Re­gen­eron adds a $100M wa­ger on joint de­vel­op­ment cam­paign with In­tel­lia

When George Yancopoulos first signed up Intellia to be its CRISPR/Cas9 partner on gene editing projects 4 years ago, the upstart smartly ramped up its IPO at the same time. Today, Regeneron $REGN is coming back in, adding $100 million in an upfront fee and equity to significantly boot up a whole roster of new development projects.

And they’re highlighting some clinical hemophilia research plans in the process.

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Iterum's fu­ture looks un­cer­tain, af­ter lead an­tibi­ot­ic fails con­sec­u­tive piv­otal stud­ies

While the market for antibiotics remains in tatters — unlike many of its bankrupt (or at the brink of bankruptcy) peers — Iterum is suffering not because its antibiotic isn’t selling, but because the compound has now failed back-to-back late-stage studies.

The experimental drug, sulopenem, was designed to tackle drug-resistant infections with an outpatient focus (in addition to hospitals), to avert those reimbursement challenges that incentivize hospitals to prescribe cheaper, generic broad-spectrum antibiotics.

Jean-Jacques Bienaimé, BioMarin chairman and CEO

Bio­Marin holds the line on bleeds with 4-year val­rox up­date on he­mo­phil­ia A — but what's this about an­oth­er de­cline in Fac­tor 8 lev­els?

BioMarin has posted some top-line results for their 4-year followup on the most advanced gene therapy for hemophilia A — extending its streak on keeping a handful of patients free of bleeds and off Factor VIII therapy, but likely stirring fresh worries over a continued drop in Factor VIII levels.

We just don’t know how big a drop.

We’ll see more data when the results are presented at the World Federation of Hemophilia in a couple of weeks. But in a statement out Sunday night, BioMarin $BMRN reported that none of the patients required Factor VIII treatment, adding:

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As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Covid-19 roundup: Did in­sid­ers cash in on pos­i­tive news re­port about Gilead be­fore pub­li­ca­tion?

A series of bullish trades on Gilead options just before the release of a favorable news story is raising questions among regulatory experts, Reuters reported.

On April 16, just hours before STAT published anecdotes from a Chicago hospital that served as one of the clinical sites to test Gilead’s remdesivir in Covid-19 patients, the California-based company’s shares were trading at around $75. Four large blocks of options were purchased for about $1.5 million each, betting that the stock would rise beyond that to as much as $87.5 by mid-August.

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Roche nabs front­line OK for Avastin/Tecen­triq in com­mon liv­er can­cer, best­ing an old Bay­er drug

For the first time in 12 years, the FDA has approved a new frontline treatment for the most common form of liver cancer.

The agency okayed a combination of Roche’s anti-VEGF antibody Avastin and their immunotherapy Tecentriq for patients with unresectable or metastatic hepatocellular carcinoma (HCC). The approval comes two weeks after Roche and their big biotech sub Genentech published Phase III results showing the combo improved both progression-free survival and, crucially, helped patients live longer than the long-running standard-of-care, Bayer’s Nexavar.