May 28, 2025

The need-to-know this morning

Spero Therapeutics and GSK said their jointly developed, oral antibiotic demonstrated a statistically equivalent cure rate compared to a standard, injectable antibiotic regimen in a Phase 3 study of patients with complicated urinary tract infections. The companies intend to seek regulatory approval for the experimental antibiotic, called tebipenem HBr, later this year.

infectious disease

RFK rolls back Covid booster recommendations

HHS secretary Robert F. Kennedy Jr. announced yesterday that he removed the CDC's recommendation that healthy children and pregnant people get Covid-19 booster shots — a move that experts say is unprecedented.

It's extraordinary that a health secretary would do this before undergoing a consultation process and without asking the advice of the CDC's advisory committee on immunization, a law professor said, making the move vulnerable to be being overturned if challenged in court.

There are real consequences — pulling booster shots from the CDC's vaccination schedule may lead some insurers to remove coverage for the vaccines, the lawyer said. Read more from STAT's Helen Branswell.

The unilateral action sparked immediate concern, with our Matthew Herper arguing in a new column that it "represents a bulldozing of safeguards intended to keep public health officials honest and their decisions transparent."

But from a financial perspective, investors of vaccine makers didn't seem fazed. Shares of Pfizer, Moderna, and Novavax ended higher in trading yesterday.

That's similar to when the FDA last week unveiled a new framework for Covid vaccines, saying that it will limit access to the vaccines to elderly and high-risk people, and it will require manufacturers to conduct clinical trials to show whether the vaccines benefit healthy younger adults and children.

Analysts at the time pointed out that most of the sales for Covid-19 vaccines comes from the 65-and-older patient population.

gene therapy

Patient in Rocket's rare disease trial dies

Rocket Pharmaceuticals said yesterday that a patient had died in a Phase 2 trial studying its experimental gene therapy for a rare heart condition called Danon disease.

Rocket did not disclose the patient's age, although the study aimed to enroll boys and young men. The company said it was still investigating the cause of the death. It suspects a drug given to suppress patients' immune systems before receiving the gene therapy may have played a role.

More than a half dozen patients have died after receiving gene therapies over the past six years, often — although not always — from a dangerous immune response to viruses used to ferry new genes into their cells. The deaths have prompted researchers to reevaluate treatments, search for new and more targeted viruses, and find ways to mitigate the immune responses with other drugs.

Rocket said that it still plans to move forward with the treatment, called RP-A501.

pharma

Lilly to acquire biotech developing pain drugs

Eli Lilly said yesterday that it will acquire SiteOne Therapeutics, a biotech developing non-opioid pain drugs, for up to $1 billion, including upfront and milestone payments.

SiteOne is developing small molecule drugs that inhibit sodium channels, similar to a newly approved treatment from Vertex called Journvax. The biotech's lead candidate is called STC-004, a Phase 2 ready drug that inhibits a sodium channel called Nav1.8.

It's too early yet to assess the rollout of Vertex's drug, but doctors and analysts have debated how big the launch will be. Those who are bullish argue that there's real need for non-addictive pain treatments, and pending laws will ensure Medicare reimburses the product. But more skeptical experts view Vertex's drug as modestly effective and are wary insurers will cover it given the availability of cheap painkillers.

regulation

Lawmaker probes FDA's ability to police drug ads

Sen. Dick Durbin (D-Ill.) sent a letter to FDA Commissioner Marty Makary yesterday, raising concerns about the agency's ability to regulate pharmaceutical advertising after layoffs and departures.

Durbin noted that four key leaders of the Office of Prescription Drug Promotion recently departed the agency, and the entire Division of Promotion Policy, Research, and Operations — a unit within the office that developed guidance on pharmaceutical advertising — also was reportedly laid off.

This comes amid ongoing debate on the regulation of drug ads. The U.S. is one of only two countries that permit drugmakers to advertise medicines directly to consumers — the other is New Zealand — and the pharmaceutical industry spends approximately $6 billion annually to reach Americans.