FDA outlines new review pathway for drugs treating ultra-rare diseases

The agency's two main review offices will work together to flexibly evaluate medicines for serious conditions that affect fewer than 1,000 people in the U.S.

By Ned Pagliarulo • Sept. 4, 2025

FDA approves updated COVID boosters, but narrows use

While the agency cleared vaccines from Pfizer, Moderna and Novavax broadly in older adults, it restricted eligibility in younger people to those with underlying health conditions.

By Delilah Alvarado • Updated Aug. 27, 2025

Why an FDA decision on Stealth’s Barth drug could ripple through the rare disease field

Stealth secured a new agency review of its experimental therapy elamipretide after a rejection this year. The result could carry broader consequences.

By Alexandra Pecci • Aug. 26, 2025

FDA suspends license for Valneva’s chikungunya shot

The agency said it had become aware of more reports of serious adverse events, leading center director Vinay Prasad to conclude the vaccine is no longer safe for its intended use.

By Delilah Alvarado • Aug. 25, 2025

FDA’s new accelerated pathway may open pharma up to risks, as well as benefits

Faster review times could leave drugmakers vulnerable to litigation, while new Trump administration priorities add more uncertainty to the approval process.

By Amy Baxter • Aug. 25, 2025

Stealth resubmits rare disease drug to FDA

The biotech, which cut jobs following the rejection in May of its Barth syndrome therapy, claimed it has addressed the agency’s concerns and now sees potential for a speedy review.

By Jonathan Gardner • Aug. 18, 2025

Novo’s Wegovy becomes first GLP-1 drug approved for MASH

The FDA clearance sets Novo’s medicine up for a market battle with Madrigal Pharmaceuticals’ fast-selling Rezdiffra.

By Ben Fidler • Aug. 18, 2025

With FDA PreCheck, drugmakers may get a manufacturing boost

The newly announced program targeting domestic manufacturing is the latest Trump administration move to ramp up drugmaking in the U.S.

By Amy Baxter • Aug. 13, 2025

Insmed gains US approval of lung disease drug forecast to be blockbuster

Brinsupri, which Insmed acquired from AstraZeneca nearly a decade ago, is the first treatment for bronchiectasis that’s not caused by cystic fibrosis. 

By Jonathan Gardner • Aug. 12, 2025

Vinay Prasad, in surprise reversal, to rejoin FDA after abrupt departure

One analyst speculated that, going forward, Prasad may be less “heavy-handed” in reviewing rare disease therapies given the public backlash to the agency’s confrontation with Sarepta.

By Ben Fidler • Updated Aug. 11, 2025

FDA to start new ‘precheck’ program to boost US drug production

The initiative is a response to a Trump administration order to speed the construction of the type of new drug factories many pharmaceutical companies have promised to build.

By Ben Fidler • Aug. 7, 2025

FDA lifts pause on Valneva’s chikungunya shot, but adds new limits

The FDA had halted use in older adults while it investigated reports of side effects. The vaccine’s label now carries new warnings and restricts vaccination to individuals at high risk of infection.

By Delilah Alvarado • Aug. 7, 2025

Vinay Prasad’s ouster leaves biotech guessing at FDA direction

The abrupt exit of the former CBER director raises questions about the FDA’s leadership. For now, newly appointed CDER head George Tidmarsh will take over Prasad’s post.

By Ben Fidler • Updated July 30, 2025

Vinay Prasad, controversial FDA official, abruptly departs agency

Prasad's exit ends a tumultuous tenure during which he led a reworking of agency guidelines on COVID vaccines and his office got embroiled in controversy over a Duchenne gene therapy.

By Ben Fidler • July 29, 2025

FDA allows Sarepta to resume some Elevidys shipments

Wall Street analysts suggested the FDA’s unexpected change in stance might reflect pressure from advocacy groups and a rebuke from higher-ups in the Trump administration.

By Ned Pagliarulo , Ben Fidler • Updated July 29, 2025

FDA delays approval decision for Bayer menopause therapy

The agency told Bayer it needs additional time to review the non-hormonal drug, called elinzanetant. Regulators in Canada and the U.K. have already cleared it for use.

By Delilah Alvarado • July 25, 2025

FDA panel elevates concerns over antidepressant use during pregnancy

Experts on the panel shared many of the same views around SSRIs, arguing the risks of the drugs during pregnancy are greater than currently accepted.

By Delilah Alvarado • July 22, 2025

Sarepta stops Elevidys shipments after standoff with FDA

Company CEO Doug Ingram said the pause was necessary for Sarepta to maintain a "productive and positive working relationship" with the regulator.

By Ned Pagliarulo • July 21, 2025

Former biotech executive appointed to lead FDA drug office

George Tidmarsh, a Stanford University physician who founded Horizon Pharma and later ran La Jolla Pharma, was named head of the Center for Drug Evaluation and Research.

By Ned Pagliarulo • July 21, 2025

5 questions on Sarepta, the FDA and a Duchenne gene therapy crisis

While Sarepta has now consented to the FDA’s request to stop selling Elevidys, the company’s brief standoff with the agency could still carry major consequences for the Duchenne community.

By Ben Fidler , Ned Pagliarulo • Updated July 22, 2025

FDA asks Sarepta to stop shipping Duchenne gene therapy

The company refused the FDA’s request and will continue shipping its therapy, Elevidys, to Duchenne patients who can still walk. A pause on shipments to older, non-ambulatory patients remains in place.

By Ned Pagliarulo • Updated July 19, 2025

Panel urges FDA to remove warnings on hormonal menopause therapy

A meeting held by the FDA Thursday spotlighted debate over black box warnings that have limited use of hormone treatment for hot flashes.

By Delilah Alvarado • July 18, 2025

Moderna’s latest approval again reveals FDA rift over COVID vaccines

Vinay Prasad overruled other reviewers for a third time in recent months in clearing only narrow use of Moderna's vaccine in young children. 

By Delilah Alvarado • July 16, 2025

FDA turns back Capricor’s Duchenne cell therapy

The company said it was “surprised” by the decision, which followed the ouster of cell and gene therapy officials from the agency during deramiocel’s review.

By Ben Fidler • July 11, 2025

Moderna COVID vaccine gets full approval for children

The approval comes amid regulatory upheaval under HHS head Robert F. Kennedy Jr., who has pushed for changes around mRNA vaccines in particular.

By Delilah Alvarado • Updated July 11, 2025