FDA approves rare disease drug, kind of

The FDA on Tuesday approved a new indication for a drug that has long been generic to treat a rare brain disorder, O. Rose Broderick reports.

The FDA approved GSK’s Wellcovorin (leucovorin calcium) in 1983 for other indications, including to lessen the toxicity of chemotherapy. The new approval adds an indication for cerebral folate deficiency. GSK no longer sells the drug, but by adding that indication, makers of generic leucovorin can update their labels, too.

Why would the FDA add an indication to a drug GSK stopped selling more than 25 years ago? Well, you may recall that back in September, HHS officials touted the drug as an autism treatment. Cerebral folate deficiency is not autism, but patients with that condition are characterized by similar developmental delays.

Read more for what the FDA had to say about the autism claim and the unusual way that the agency approved the new indication.

telehealth

FDA telehealth crackdown highlights role of national doctor networks

The FDA is expanding its crackdown on telehealth companies marketing compounded weight loss drugs, warning them not to imply their products are FDA-approved or that they manufacture the medications. But a STAT analysis shows the ecosystem behind those brands is far more concentrated than it appears.

Roughly 30% of the more than 70 telehealth companies warned in the past six months rely on just four nationwide “white-label” medical groups — Beluga Health, OpenLoop, MD Integrations, and Telegra — that provide the clinicians who actually prescribe the drugs.

That structure has helped telehealth brands scale quickly, but it also creates a supply chain that may draw regulatory scrutiny as misleading marketing proliferates and lawsuits mount.

“The whole reason these companies are able to get away with these personalized compounded drugs is because some provider believes that that personalized drug can be helpful for someone, and so we’re putting a lot of trust into these provider firms in making that decision,” said Ashwin Chetty, a medical student at the Yale School of Medicine who co-authored a study of 79 telehealth companies marketing GLP-1s. “Should we be questioning that trust?”

Read more.

from AIHealth Tech:

March 12, 2026 09:00 AM EDT AIHealth Tech
‘Closed-loop’ AI clinical care is coming soon, startups predict
Lydia Ramsey Pflanzer
Deputy Editor
The startups that are building tools for tasks historically done by clinicians think AI-only healthcare is coming soon.

Sharif Vakili
In the next two years, UpDoc co-founder and CEO Sharif Vakili predicted there will be AI agents that can take on jobs like writing a prescription — without a doctor involved, outside of a study setting. Vakili made his prediction at Lux Capital’s Health x Intelligence Forum in San Francisco last week during a panel moderated by Endpoin ts News.

Closed-loop AI in healthcare delivery is a new way of using the technology. Until now, most conversations have been about using tools to help doctors with administrative and clinical tasks. Letting AI work on its own would change where and how doctors practice, and how healthcare is paid for and delivered.

Doctronic co-CEO Matt Pavelle thinks closed-loop AI is coming even sooner. The startup is running a program in Utah to renew prescriptions for chronic conditions and is quickly working toward a point where AI alone makes refill decisions.

“We’re close,” Pavelle said. “We will certainly have closed-loop for AI doing renewals soon.”

Matt Pavelle
UpDoc is also working toward closed-loop clinical AI, though Vakili argued the startup is taking a different approach by aiming to work with primary care providers, though he declined to share more. On the panel, Vakili differentiated UpDoc from Doctronic by comparing UpDoc’s enterprise focus to Anthropic and Doctronic’s consumer focus to OpenAI.

John Brownstein, chief innovation officer at Boston Children’s Hospital, said that the physicians in the health system aren’t thrilled with the idea of being taken out of the equation.

“Why are we thinking about closed loop?” Brownstein said. “Why are we not trying to arm our providers with incredible insights that they wouldn’t get otherwise?”

Instead, he’s seeing ways for AI agents to “supercharge” clinical efforts, like helping diagnose rare diseases in children. So far, Brownstein said, Boston Children’s has delivered AI-based diagnoses to 40 families. He said he sees that as a collaboration between AI and geneticists at the health system.

Doctronic’s program currently charges $4 for a prescription renewal, but Pavelle said the aim is to make clinical AI free. UpDoc aims to sell its tool to primary care providers, who can use it to get paid for the care they deliver.

John Brownstein
After hearing about the two payment approaches, Brownstein said that the idea of paying for a tool for Boston Children’s primary care facilities made sense. “We would pay for something like that. We’d pay for the Anthropic of closed-loop and the OpenAI,” he joked.

Pavelle said he anticipates that a number of companies will soon begin moving toward closed-loop AI in healthcare delivery.

“We’re essentially swarming the problem of how to fix healthcare in this country,” he said.

 

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