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

FDA, in policy shift, publishes some drug rejection letters

The agency disclosed a tranche of more than 200 complete response letters from the past five years, but only those involving medicines that it later cleared.

By Ned Pagliarulo • July 10, 2025

Novartis gets approval of first malaria medicine for newborns

Coartem Baby, which was cleared by health authorities in Switzerland, will fill an important gap in treatment. Novartis plans to sell it “largely” on a not-for-profit basis.

By Delilah Alvarado • July 8, 2025

After delay, Kalvista wins FDA OK for drug to treat rare swelling disorder

The company had blamed FDA "resource constraints" for the delay, while a rival drug for hereditary angioedema received an on-time approval.

By Jonathan Gardner • July 7, 2025

FDA takes major step to ease access to CAR-T therapy

The agency removed some onerous requirements for the complex cancer drugs and reduced restrictions on patients’ post-treatment movement.

By Ned Pagliarulo • June 27, 2025

Top drug official at FDA reportedly set to exit next month

Jacqueline Corrigan-Curay, who serves as acting head of the powerful CDER office, will leave after nearly a decade at the agency.

By Ned Pagliarulo • June 23, 2025

Gene therapy faces fresh uncertainty as two more top FDA officials depart

The reported dismissal of high-ranking CBER officials Nicole Verdun and Rachael Anatol resurfaced lingering concerns about how gene therapies will be regulated under new FDA leadership.

By Ben Fidler • June 20, 2025

Could the FDA take an indirect approach to regulate lab developed tests?

Attorneys said many questions remain about test regulation after the FDA lost its legal fight. A recent warning letter could be a clue to future enforcement.

By Susan Kelly • June 16, 2025

FDA to miss approval deadline for Kalvista drug due to ‘resource constraints’

A “heavy workload” and “limited resources” have left the agency unable to meet a June 17 deadline to approve a therapy Kalvista has been developing for hereditary angioedema. 

By Ben Fidler • June 16, 2025

Moderna wins FDA OK to widen use of RSV vaccine

The agency’s decision to clear Moderna’s shot for use in certain younger adults is a much-needed win for a company that’s been significantly impacted by the recent changes in FDA and HHS leadership.

By Delilah Alvarado • June 13, 2025

FDA clears Nuvation lung cancer drug, setting up battle with Bristol Myers and Roche

The drug, Ibtrozi, will now vie for market share in an indication where multiple large pharmaceutical companies have already struggled to grow sales.

By Ben Fidler • June 11, 2025

Merck antibody drug for RSV approved by FDA

Known as Enflonsia, the drug will compete for market share with Sanofi and AstraZeneca’s in-demand Beyfortus, which quickly became a blockbuster medicine following its approval two years ago.

By Delilah Alvarado • June 9, 2025

FDA meeting gives window into gene therapy field’s angst

Gene therapy experts and advocates warning of the sector’s many challenges found a receptive audience in FDA leadership at a regulatory forum Thursday.

By Ned Pagliarulo • June 6, 2025

FDA’s AI tool ‘Elsa’ is here, and the industry has questions

The agency has provided only limited details about its AI pilot as it advances an “aggressive timeline” for the tool's rollout.

By Amy Baxter • June 5, 2025

FDA clears Moderna’s new COVID vaccine, but with limits

The shot, dubbed mNexspike, is approved for older adults and people whose health conditions put them at higher risk of severe disease. 

By Delilah Alvarado • May 31, 2025

Merck, Daiichi pull approval application for ADC in lung cancer

A therapy at the center of their $22 billion alliance failed to extend survival in a type of lung tumor, leading the companies to abandon a U.S. submission.

By Ben Fidler • May 29, 2025

FDA leaders seek industry input on ‘listening tour’

Commissioner Martin Makary and two of his deputies aim to meet with drugmaker CEOs to discuss how the agency can “modernize” regulations.

By Ned Pagliarulo • May 28, 2025

FDA sets COVID vaccine formula as RFK Jr. narrows guidance for shots

The agency recommends manufacturers update their vaccines to target the JN.1 strain. At the same time, HHS head Robert F. Kennedy Jr. is removing guidance that healthy children and pregnant women receive COVID shots.

By Delilah Alvarado • Updated May 27, 2025

FDA panel recommends keeping COVID shots targeted to same strain as last year

The advisory committee, which met after the agency rolled out new vaccine guidelines, said companies should tailor their booster doses to the “JN.1” coronavirus lineage.

By Delilah Alvarado • May 22, 2025

FDA panel backs Darzalex for early stage multiple myeloma

Agency drug reviewers had questioned patient risk criteria and the efficacy endpoint used in a key trial supporting J&J's drug.

By Jonathan Gardner • May 21, 2025