July 14, 2025

The need-to-know this morning

AstraZeneca said its experimental hypertension drug, baxdrostat, reduced treatment-resistant blood pressure in a late-stage clinical trial. The drug works by lowering blood pressure in a new way, different from currently available medicines. If approved, AstraZeneca has told investors baxdrostat could become a multibillion-dollar commercial product.

REGULATION

Makary's big promises at FDA get a closer inspection

In his first 100 days as commissioner of the Food and Drug Administration, Marty Makary has made a handful of splashy announcements — from pledging transparency on rejection letters to floating fast-track vouchers for drugs tied to national health priorities.

Some ideas, like publicly releasing complete response letters, could bring long-overdue clarity to FDA decisions, STAT's Matthew Herper writes. But others, like using the promise of two-month drug reviews to incentivize lower pricing, reveal a shaky grasp of pharma's economic incentives and the agency's operational limitations.

Makary, Matt says, is media-savvy but not always precise. That, in turn, may be stirring criticism within the FDA, where morale is low. Matt writes: "The first step to sharpening his good ideas and getting internal traction probably involves not talking, but listening."

neurology

Takeda drug succeeds in Phase 3 narcolepsy trials

Takeda said this morning that its investigational therapy helped patients with a type of narcolepsy feel more awake in two large trials, though it didn't release specific data.

The drug, called oveporexton, led to statistically significant improvements for patients with narcolepsy type 1 across all primary and secondary endpoints — including wakefulness, excessive daytime sleepiness, muscle control, ability to maintain attention, and quality of life.

The results set Takeda up to be the first company on the market with a new type of medicine that activates orexin receptors, though the lack of data make it difficult to gauge how competitive its drug will be against earlier candidates in development from Alkermes and Centessa Pharmaceuticals.

While this type of drug is expected to work in narcolepsy type 1 patients, since those patients have large deficiencies of the orexin hormone, investors are waiting to see if it can also work for patients with narcolepsy type 2 and idiopathic hypersomnia. Those patients don't have significant orexin deficiencies, but if the drugs can still work for them, then there would be a much bigger market opportunity.

glp-1 drugs

Rehab center sees semaglutide success for addiction

While GLP-1 medications have shown significant promise as addiction treatments, only a handful of clinical trials are underway to measure their ability to reduce substance use. Several are unlikely to publish results within the next two years.

But at an elite, Pennsylvania-based treatment center, doctors are prescribing the GLP-1 drug semaglutide to curb addiction, from alcohol to opioids to gambling, STAT's Lev Facher reports. Though the off-label use lacks clinical trial backing, early results have staff — all of whom are themselves in long-term recovery — touting GLP-1 drug as a breakthrough in treating cravings.

"I don't think of this as doing anything wild west," said Steven Klein, one of the staff physicians who has pioneered the practice of prescribing GLP-1s as a treatment for addiction. "We're using something off-label under the umbrella of addiction, whether that be food, sex, alcohol, or opioids."

REGULATION

FDA declines to approve Ultragenyx gene therapy

Ultragenyx announced Friday that the FDA declined to approve its gene therapy for patients with Sanfilippo syndrome type A, a rare inherited metabolic disorder.

In the complete response letter from the FDA, Ultragenyx said, the agency requested that it "provide additional information and improvements" related to manufacturing issues following facility inspections. The company added that it believes the issues "are readily addressable" and "are not directly related to the quality of the product."

Ultragenyx said that, after addressing the concerns, it expects to resubmit its application and see a review that could last up to six months longer.

TD Cowen analyst Yaron Werber said the FDA response came unexpectedly early, since the deadline for the agency to decide on the drug was in August. But he said it's encouraging that the letter does not raise any concerns around the quality of the therapy and doesn't request clinical data.

Ultragenyx is seeking accelerated approval based on a biomarker, and the FDA in its letter acknowledged that the biomarker data are supportive, the company said. The biotech industry is closely watching how the FDA under the Trump administration approaches biomarkers, especially since Vinay Prasad, the top regulator of gene therapies, had been skeptical of surrogate endpoints before joining the agency.

More around STAT