STAT+: Novartis’ experimental cardiovascular drug fails a pivotal study
A closely watched cardiovascular drug from Novartis failed in a pivotal study, the company said Friday, a major blow to the firm and to efforts to target a common heart disease risk factor.…
The drug, called pelacarsen, had shown in earlier trials that it could…
But Novartis said that in the Phase 3 HORIZON trial, pelacarsen failed…
STAT+: After failure of Angelman syndrome drug, experts see hope in other experimental therapies
The failure of an experimental drug for Angelman syndrome, a rare genetic disease that causes severe developmental delays, was a major blow to patients and the drug’s maker, Ultragenyx, which announced the outcome of its late-stage trial earlier this week.…
But experts cautioned that there is still reason to think similar expe…
That could potentially pave the way for genetic medicines in other neu…
“I don’t think this really says anything about the other trials that a…
Opinion: Make infectious disease data boring again
Amid soaring measles cases, the U.S.health secretary and leadership at his CDC have picked a fight with the Commonwealth of Pennsylvania about what counts as a measles death.…
The last thing public health needs is drama when the focus needs to be…
States have a standard process for notifying the CDC about notifiable …
Developing those definitions is usually a long, deliberative process i…
STAT+: Recipients of gene-edited pig kidneys successfully receive human organs
When doctors think about sticking an organ from another animal into a person, one of their many questions is about what might happen after they take that organ out.…
Would the patient produce antibodies that would complicate a human-to-…
The scientists at eGenesis didn’t think so, based on what they saw in …
STAT+: Ionis wins FDA approval for first drug for Alexander disease, a rare and deadly neurological disorder
Ionis Pharmaceuticals on Thursday received Food and Drug Administration approval for the first disease modifying therapy for Alexander disease, a rare and deadly neurological condition.…
The FDA approved the drug, Zanvastro, for use in children and adults a…
The study also contained hints that treating young children can improv…
Researchers found that the drug was generally safe, with serious adver…
Opinion: I was a doula before I was an OB-GYN. I wish the two professions understood each other more
I was standing in an operating room waiting impatiently for the abdominal prep to dry before an urgent cesarean delivery following a failed trial of labor after cesarean.…
The room was filled the quiet urgency that comes just before incision.
STAT+: FDA pilot offers generative AI medical devices a path to patients before they are authorized
As the Food and Drug Administration wrestles with how to regulate medical devices that rely on generative artificial intelligence to make decisions about care, it’s provisionally allowing some developers to launch their products on the market.…
AI products from Cadence and Limbic are among four devices recently ac…
The pilot is intended to beef up the number of technologies available …
But it also offers the agency and companies the opportunity to experim…
STAT+: Abivax has still not been acquired. What’s going on?
This story first appeared in Adam’s Biotech Scorecard, a subscriber-only newsletter.STAT+ subscribers can sign up here to get it delivered to their inbox.…
Biotech M&A activity is off the charts, yet the French drugmaker — dev…
We’re nine months into a year in which a takeout of Abivax for $20 bil…
The absence of said transaction has caused the stock’s investor fanbas…
STAT+: Ultragenyx drug to treat Angelman syndrome, a rare disease, fails late-stage trial
The biotech company Ultragenyx said Wednesday that its experimental therapy for Angelman syndrome, a rare disease that causes severe intellectual disabilities and developmental delays, showed no benefit compared to a sham treatment in a large Phase 3 trial.…
The drug, GTX-102, had shown powerful results in early trials, raising…
Many advocates for patients with other neurological conditions also ho…
The news is a significant blow for Ultragenyx’s business as well.