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Updated: 1 hour 11 min ago

STAT Plus: Novartis CEO coy about pricing its gene therapy for spinal muscular atrophy

2 hours 32 min ago

CAMBRIDGE, Mass. — Days before his company is expected to win approval of the most expensive drug ever, the head of Swiss drug giant Novartis said Wednesday he could justify charging up to $5 million for the revolutionary treatment for a rare genetic disease, but it will end up being priced at a fraction of that.

Speaking at the company’s biomedical research institute, Novartis CEO Vas Narasimhan wouldn’t disclose the price of the drug, called Zolgensma, until the government approves it, probably by the end of the month. Zolgensma treats spinal muscular atrophy, or SMA, a deadly neurodegenerative disease.

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STAT Plus: Pharmalittle: Novartis to price gene therapy ‘far lower’ than $4m; advocates press WHA on transparency resolution

3 hours 4 min ago

Good morning, everyone, and how are you today? We are doing just fine, thank you, especially now that rain clouds have departed and the sun is trying mightily to warm the otherwise damp and chilly Pharmalot campus. Such circumstances call, not surprisingly, for a hot cup or three of stimulation. And so we are firing up the coffee kettle. Our choice remains mocha nut fudge, since we have an unusually large stash. We generally keep a year’s supply of each flavor, in case you were wondering. Anyway, time to get cracking. Here are your tidbits. Hope you conquer the world today and do keep in touch. …

Novartis (NVS) chief executive Vas Narasimhan says the company plans to price a forthcoming gene therapy for spinal muscular atrophy “far lower” than the $4 million to $5 million figure the drug maker has said it could be worth, Reuters writes. U.S. regulators are expected to make a decision this month on whether to approve Zolgensma, a one-time therapy seen as a potential long-term solution for the rare disease that is the leading genetic cause of death in infants. The company has previously said it could price the treatment in a range of $1.5 million to $5 million.

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Opinion: Prejudice and patient choice: an insidious confluence in medicine

8 hours 6 min ago

“Idon’t want your kind taking care of me!”

To an outsider, it might have resembled a standoff from an old Western. But it was taking place in a most modern hospital: the patient, an older white man hunched over with a snarl distorting his face; the physician, a young woman with a patterned hijab wrapped around her head.

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STAT Plus: Did FDA unfairly treat Catalyst by approving a similar drug from another company?

8 hours 16 min ago

Reeling from an unexpected move by regulators, Catalyst Pharmaceuticals (CPRX) is threatening to hit back.

Until recently, the drug maker had been riding high. Last December, the Food and Drug Administration approved its medicine for treating adults with a rare neuromuscular disorder called LEMS, which meant Catalyst has seven years of marketing exclusivity. And despite controversy over its $375,000 price tag, the company appeared to have silenced some critics with assistance programs that kept patient out-of-pocket costs low. Meanwhile, its soaring stock price pleased investors.

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STAT Plus: Microbiome therapies could be the next frontier in medicine. But how exactly do you make them?

8 hours 16 min ago

Before any company can begin a clinical trial, the Food and Drug Administration has to know how they’re going to make their “drug.” For some microbiome companies, that’s not really a problem; to put it delicately, the active ingredient for their drug comes from the guts of willing volunteers. All that’s left to do is process it.

But as more attention and hope is placed on bacteria specifically selected or designed to be therapeutic, the companies testing them may hit a roadblock that could cost them millions or slow their progress.

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California names former Google scientist as the state’s ‘mental health czar’

Wed, 05/22/2019 - 11:29

Noted psychiatrist and former Verily leader Dr. Tom Insel is going to be the “mental health czar” for the state of California, Democratic Gov. Gavin Newsom announced Tuesday.

Insel, the former National Institute of Mental Health director, will also continue his work with Mindstrong, a startup that is working on a mental health app, a company spokesperson confirmed. Insel joined the company in 2017 after leaving Verily, Google’s life sciences arm.

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STAT Plus: Lawmakers contend WHO pain treatment guidelines are really Purdue ‘marketing materials’

Wed, 05/22/2019 - 08:19

Two  lawmakers are urging the World Health Organization to rescind guidelines issued nearly a decade ago for treating pain because they contain “dangerously misleading” and sometimes “outright false claims” about the safety and effectiveness that were orchestrated by Purdue Pharma.

In a new report, the lawmakers contend that the WHO guidelines, which were released in 2011 and 2012, are “serving as marketing materials for Purdue.” And they pointed to efforts by the company to create and fund front groups that participated in research that shaped WHO decision making – and dovetailed with corporate goals to boost use of opioids, such as its own OxyContin pill.

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STAT Plus: Pharmalittle: Lawmakers push bill to punish opioid execs; Novartis will have ‘enormous leverage’ pricing its SMA drug

Wed, 05/22/2019 - 06:26

Good morning, everyone, and how are you today? We are doing just fine, thank you, especially now that the middle of the week is here. After all, this means just a few days are left until the weekend arrives. So keep plugging away. After all, what are the alternatives? While you ponder the possibilities, we will keep busy brewing another cup of stimulation — mocha nut fudge is our choice today, for no particular reason — and forage for interesting items. Meanwhile, here are some tidbits to get you going. Have a splendid day and we hope you conquer the world. …

Four lawmakers from the Senate and House introduced a bill to prohibit illegal marketing and distribution of opioids; create criminal liability for top company executives; penalize drug makers who illegally advertise, market or distribute an opioid product; and require drug makers to reimburse the country for the negative economic impact of their products. The move comes after former Insys Therapeutics (INSY) executives were convicted of bribing doctors to prescribe an addictive opioid and Rochester Distributor executives were indicted for failing to monitor opioid shipments.

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Behind the scenes of Google’s effort to test AI for lung-cancer screening

Wed, 05/22/2019 - 06:05

A version of this story first appeared in STAT Health Tech, our weekly newsletter about how tech is transforming health care and the life sciences. Sign up here to receive it in your inbox.

Behind the scenes of Google’s effort to build an AI system to screen patients for lung cancer is a long and tangled tale. A study the tech giant published Monday reported positive findings: The system outperformed radiologists in analyzing CT scans, significantly reducing false positives and finding more actual cancers (though more research is needed to show whether it would help patients live longer).

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Opinion: Vaccinations will take a hit if the Trump administration topples the Affordable Care Act

Wed, 05/22/2019 - 01:40

In the midst of the country’s worst measles outbreak in 25 years, President Trump made an abrupt about-face on vaccinations.

Before becoming president, he had spread vaccination fears by peddling anti-vaccine tropes on Twitter and meeting with anti-vaccine conspiracists. When faced with the growing measles outbreak, the president seems to have changed his tune and now urges Americans to “get their shots.” Behind the scenes, though, his administration’s efforts could undermine public health efforts to ensure that children and adults get the vaccinations they need to prevent illness.

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The Ebola response effort is struggling. Experts say these steps could help

Wed, 05/22/2019 - 01:35

With Ebola response teams struggling to contain the outbreak in the Democratic Republic of the Congo, the World Health Organization and its partners can make changes to shore up their effort and try to prevent the crisis from escalating further, according to a handful of experts surveyed by STAT.

The experts are wary of criticizing WHO officials and others trying desperately to stop the virus from spreading. Violence aimed at Ebola response workers and a refusal to cooperate with control measures in some communities has rendered this outbreak, the second largest on record, unlike anything the world of Ebola responders has seen before. There have been repeated attacked on Ebola treatment centers, and on many days response workers have been unable to move about in outbreak hotspots.

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‘What’s my real identity?’: As DNA ancestry sites gather more data, the answer for consumers often changes

Wed, 05/22/2019 - 01:30

NEW YORK — 23andMe caused Leonard Kim not one identity crisis but two.

The first came in 2016, when Kim, who was raised believing he was 100% Korean, took the company’s DNA ancestry test and learned he was almost half Japanese. Then, earlier this month, he was out for drinks with his wife and some friends near his Los Angeles home when someone questioned his Japanese ancestry.

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MIT professor is accused of claiming others’ scientific discoveries as his own

Tue, 05/21/2019 - 15:21

MIT professor Ram Sasisekharan made his name on the idea that algorithms and computer models could lead to better and more potent therapies, a promise that launched three biotech companies and attracted hundreds of millions of dollars.

But two treatments purportedly discovered with Sasisekharan’s computational approach are almost identical to compounds that had previously been described by other labs, according to a new paper by outside researchers. The finding casts serious doubts on the integrity of Sasisekharan’s research, the authors claim.

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STAT Plus: Tocagen continues a brain cancer gene therapy clinical trial. Investors fear the worst

Tue, 05/21/2019 - 13:33

Tocagen (TOCA) said Tuesday that a Phase 3 clinical trial involving a novel gene therapy for aggressive brain tumors will continue to a final analysis later this year, following an interim look at patient survival data conducted by independent monitors. Tocagen’s stock price dropped sharply.

The San Diego-based biotech pitched the study’s continuation as an encouraging sign for its gene therapy called Toca 511/Toca FC, which is designed to deliver a localized chemotherapy directly to the site of brain tumors.

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STAT Plus: Mallinckrodt sues CMS over Medicaid rebates for its pricey Acthar Gel medicine

Tue, 05/21/2019 - 11:18

In an unexpected development, Mallinckrodt (MNK) filed a lawsuit accusing the federal government of illegally resetting a benchmark used for calculating rebates the company must pay to Medicaid for its most important drug, a move that could cost it hundreds of millions of dollars and hobble its R&D efforts going forward.

At issue is the amount of money the company owes the Centers for Medicare and Medicaid Services for its controversial Acthar Gel treatment, which is used primarily to treat infantile spasms and has been a poster child for the high cost of medicines. Over the past five years, the wholesale price rose 20% and a vial now costs around $39,000, making the drug an expensive proposition for public and private payers.

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STAT Plus: Four of the world’s largest drug companies are teaming with Verily. Here is what they get

Tue, 05/21/2019 - 11:10

On Tuesday morning, Verily, Alphabet’s unit focused on life sciences, announced that it had formed alliances with Novartis, Sanofi, Otsuka, and Pfizer to work on clinical trials. What are those drug giants getting out of the deal? STAT sat down with Scarlet Shore, who leads Verily’s project Baseline, to learn about the company’s vision for the clinical trial of the future. The conversation took place at CNBC’s “Healthy Returns” conference, where the partnerships were unveiled.

Clinical trials have always been an obvious target for Verily as it tries to find a way to use technology to change health care. New devices can take years to develop. But right now, the process of conducting clinical trials is pockmarked with inefficiencies, requiring pharmaceutical companies and medical researchers to work with dozens of vendors while often keeping records in efficient ways — sometimes even using that least digital of formats, pen and paper. And Verily brings to the table the massive capacity to deal with large datasets. Its chief medical officer, Dr. Jessica Mega, previously ran large clinical trials that were published in the New England Journal of Medicine and the Lancet.

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STAT Plus: Baltimore sues a J&J unit over ‘sham’ patent litigation to extend its monopoly on a cancer drug

Tue, 05/21/2019 - 08:34

Angry over the high price of a prostate cancer drug, Baltimore officials have filed a lawsuit accusing a Johnson & Johnson (JNJ) unit of using “sham” patent litigation to delay the availability of lower-cost generics, forcing the city to spend millions of dollars unnecessarily.

The lawsuit alleges that Janssen Biotech and its partner, British Technology Group, undertook a “ruse” when seeking a patent for its Xtandi medicine by failing to disclose certain information to the U.S. Patent and Trademark Office. And the move made it possible for the drug maker to deflect generic rivals longer than it might have otherwise.

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Inspired by breath strips, scientists break away from needing a ‘cold chain’ to deliver vaccines

Tue, 05/21/2019 - 08:20

A version of this story appeared in STAT’s Morning Rounds newsletter. Sign up here to receive it in your inbox.

One of the many challenges in developing and delivering vaccines where they are needed is keeping them at the right temperature at the right price. Most vaccines, especially ones made from weakened forms of living viruses, must be kept cold to be effective.

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STAT Plus: Pharmalittle: Merck’s Keytruda fails breast cancer trial; a pair of Sacklers flee New York

Tue, 05/21/2019 - 06:41

Top of the morning to you and a fine one it is. A delicious breeze and shiny warm sun are enveloping the Pharmalot campus, where the short people have left for their productive destinations and our official mascot is happily snoozing under our feet. This means we are free to brew cups of stimulation and, most important, forage for interesting tidbits. Speaking of which, here are a few items of interest to help you on your own journey. Hope all goes well and feel free to send along hints and allegations. We enjoy a mysterious hunt. …

Former Manhattan society staples David and Joss Sackler are fleeing town and heading to Florida in a bid to escape the imperishable stain of their scandal-soaked family’s OxyContin business, The New York Post dishes. David — whose family’s company, Purdue Pharma, is accused of igniting the opioid crisis by aggressively marketing the painkiller, then directing efforts to mislead the public about its addictive risks — and wife Joss lived it up in a $6.5 million Upper East Side apartment. The couple were mainstays on the New York social scene, as well as big art patrons and donors to major museums.

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Scott Gottlieb is skeptical of the science behind CBD supplements

Tue, 05/21/2019 - 06:01

D.C. Diagnosis is STAT’s weekly newsletter about the politics and policy of health and medicine. Sign up here to receive it in your inbox.

Scott Gottlieb has a lot of thoughts on CBD

When I chatted with the former FDA commissioner about the agency’s efforts to regulate CBD, one thing was crystal clear: Gottlieb probably isn’t going to be offered a job as the head of the CBD lobby any time soon.

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