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STAT+: FDA approves Sanofi diabetes drug for children with stage 3 diabetes

WASHINGTON — The Food and Drug Administration on Friday approved teplizumab, a type 1 diabetes drug developed by Sanofi, for children aged 8 and older with stage 3 diabetes. 

The drug was selected to go through a speedy review program launched last year by former FDA Commissioner Marty Makary, but the agency missed its goal date of April 21 to deliver a decision. 

STAT previously reported that Sanofi asked to pull its drug out of the program after former top drug regulator Tracy Beth Høeg disagreed with a staff decision to approve the drug. It’s rare for a center director, and particularly a political appointee like Høeg, to get involved in individual scientific reviews. 

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WASHINGTON — The Food and Drug Administration on Friday approved teplizumab, a type 1 diabetes drug developed by Sanofi, for children aged 8 and older with stage 3 diabetes. 

The drug was selected to go through a speedy review program launched last year by former FDA Commissioner Marty Makary, but the agency missed its goal date of April 21 to deliver a decision. 

STAT previously reported that Sanofi asked to pull its drug out of the program after former top drug regulator Tracy Beth Høeg disagreed with a staff decision to approve the drug. It’s rare for a center director, and particularly a political appointee like Høeg, to get involved in individual scientific reviews. 

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Opinion: ‘I’m pretty much all in’: An interview with a woman starting medical residency at almost 73

Below is a lightly edited, AI-generated transcript of the “First Opinion Podcast” interview with Dawn Zuidgeest-Craft. Be sure to sign up for the weekly “First Opinion Podcast” on Apple PodcastsSpotify, or wherever you get your podcasts. Get alerts about each new episode by signing up for the “First Opinion Podcast” newsletter. And don’t forget to sign up for the First Opinion newsletter, delivered every Sunday.

Torie Bosch: So I get a surprising number of ideas for First Opinion by watching TikTok. It’s for work, I swear. Recently, I came across a video of a woman proudly sharing the fact that her mother, age 72, had just completed medical school and matched into residency. I had to talk to the septuagenarian to find out more about going to medical school at an age when most people have already retired. And much to my delight, she agreed.

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Below is a lightly edited, AI-generated transcript of the “First Opinion Podcast” interview with Dawn Zuidgeest-Craft. Be sure to sign up for the weekly “First Opinion Podcast” on Apple PodcastsSpotify, or wherever you get your podcasts. Get alerts about each new episode by signing up for the “First Opinion Podcast” newsletter. And don’t forget to sign up for the First Opinion newsletter, delivered every Sunday.

Torie Bosch: So I get a surprising number of ideas for First Opinion by watching TikTok. It’s for work, I swear. Recently, I came across a video of a woman proudly sharing the fact that her mother, age 72, had just completed medical school and matched into residency. I had to talk to the septuagenarian to find out more about going to medical school at an age when most people have already retired. And much to my delight, she agreed.

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STAT+: Trump administration revisits policy to close Medicare drug price negotiation loophole

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WASHINGTON — The Trump administration on Friday proposed to change a policy that is designed to prevent drugmakers from avoiding Medicare price negotiation by adding active ingredients to drugs. 

The policy is part of an annual proposed rule that establishes the process that the Centers for Medicare and Medicaid Services uses to choose the next 20 drugs and biologics for price negotiation. Those drugs will be announced by Feb. 1, 2027, and their negotiated prices will take effect in 2029. The administration also considered a similar policy last year but put off a decision to study it further.

Medicare must wait seven to 11 years after a product is approved by the Food and Drug Administration before it can negotiate its price, depending on the type of medicine. Biologics that are typically administered in doctor offices get more time than drugs taken orally. 

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Hantavirus One-Shot mRNA Vaccine Fully Protects in Syrian Hamster Model

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Last month, the Andes virus outbreak on a Dutch cruise ship departing from Argentina brought a transmission context for hantavirus, that was previously unprecedented, to the forefront. The Andes virus is the only member of the hantavirus family that is capable of efficient person-to-person spread through close contact with respiratory secretions. Other hantaviruses are typically spread through contact with infected rodents, making the Andes virus a much more significant public health threat.

While at sea, the outbreak spread among passengers and crew, infecting 13 people and killing three. The cruise passengers have since returned to their home countries, 23 in total. Because a person can carry the virus for weeks before showing any symptoms, health agencies are facing a complex challenge of identifying everyone who was exposed. There are currently no vaccines or preventive treatments approved for the virus; this travel-related outbreak brought the need for vaccine development to the forefront.

Researchers at The University of Texas Medical Branch (UTMB) had previously developed and tested two mRNA vaccines against intramuscular Andes virus challenge in golden Syrian hamsters (“1-methylpseudouridine-modified or non-modified mRNA modalities encoding the envelope glycoproteins, Gn and Gc, in a single open reading frame.”)

When tested in the Syrian hamster model, both mRNA vaccines were efficacious in hamsters using a two-dose regimen. Recognizing that a fast-moving international outbreak doesn’t allow time for patients to wait weeks between shots, the team retested the vaccines to determine whether a single dose would be effective.

Now, a new report shares the finding that the vaccine provided full protection against the Andes hantavirus after a single dose.

This work is published in The Lancet in the paper, “Single-dose mRNA vaccines against Andes hantavirus.

Alexander Bukreyev, PhD, head of the Laboratory of Viral Pathogenesis and Vaccine Development at UTMB, said that the group is working to fast-track these single-dose vaccines into human clinical trials.

The results exceeded expectations. When testing the vaccines in an animal model that mimics human disease, the scientists found that a single shot provided 100% protection against a lethal dose of the virus. Even when the researchers significantly lowered the dosage to a fraction of the original amount, the results remained definitive.

“Every vaccinated animal remained completely healthy and showed no symptoms or weight loss,” said Michelle Meyer, PhD, senior scientist in the Bukreyev Laboratory. “When we looked at the tissues from the vaccinated animals a month after infection, the virus was entirely gone. The vaccines triggered a powerful immune response, creating protective antibodies in as little as 14 days.”

Because the Andes virus can take a relatively long time to make a human severely ill, these fast-acting vaccines could serve a dual purpose, possibly functioning as an emergency tool for people who have already been exposed.

“If given quickly to high-risk contacts during an outbreak, such as the Andes virus situation on the cruise ship, the vaccines could theoretically jump-start their immune systems fast enough to intercept the virus—stopping it from replicating and preventing them from getting sick or spreading it further,” Bukreyev said.

The post Hantavirus One-Shot mRNA Vaccine Fully Protects in Syrian Hamster Model appeared first on GEN – Genetic Engineering and Biotechnology News.

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