Wikipedia -WSS

Wikipedia

author：knowledge 2025-10-02 20:52:48 - browse：981

FDA's Peter Marks talks with STAT's Adam Feuerstein — Peter Marks, the FDA's top regulator of gene therapies (right), speaks with STAT's Adam Feuerstein in San Francisco. Sarah Gonzalez/Peopletography for STAT

SAN FRANCISCO — Peter Marks, the Food and Drug Administration’s top regulator of gene therapies, seems very comfortable with granting some form of full approval to Elevidys, the Sarepta Therapeutics gene therapy for Duchenne muscular dystrophy, based on his comments to me on Monday night during our “fireside chat” at a STAT event.

To be clear, Marks declined to comment specifically about the ongoing FDA review of Elevidys. I tried. However, Marks described a “hypothetical case” in the context of my Sarepta questions that clearly suggested the failure of an Elevidys confirmatory study in October didn’t bother him all that much.

“Hypothetically, if you had a product that used an aggregate scale and all of the components of that aggregate scale looked pretty good, or nearly all of them look pretty good, but the aggregate, for various reasons, didn’t come out perfectly. We approved products based on that,” Marks told me.

Get unlimited access to award-winning journalism and exclusive events.

Subscribe Log In

Previous article： At least 13 dead in Texas as scorching temps continue
Next article： Redefining sustainability for the pharmaceutical industry

focus

WSS

Laying a path for careers in science

Color Health wants to make cancer screenings convenient

Mifepristone protocols being wielded by pill's opponents in Supreme Court case

Suspected Gilgo Beach killer appeared 'traumatized' after arrest: Defense attorney

CDC panel: People 65 and older should get a Covid spring booster shot

Medicare Advantage isn’t working — for anyone

Wikipedia

Wikipedia

knowledge

Bright Health sells Medicare Advantage business to Molina

Russia strikes Ukraine's critical port facilities in Odesa after halting grain deal

Duchenne breakthrough therapy leaves behind pioneering families

Actors' union, Hollywood studios squabble over which side to blame as strike enters 5th day

Malaria cases in Florida and Texas: Here’s what you need to know

India sets sights on home

Malaria cases in Florida and Texas: Here’s what you need to know

Stanford updates investigation of president’s past research

ChatGPT in medicine: STAT answers readers' burning questions

The kidney, in all its complexity, is captured in a new atlas

focus

'Vaccine

Doctor of U.S. President Joe Biden says he is fit for duty

Elisabeth Bik tackles the widespread issue of research misconduct

Change Healthcare cyberattack shows risk of industry consolidation

Resistance to cancer drugs: A new genetic explanation

STAT's 2023 biotech VC rankings: ARCH Venture Partners tops list

focus

leisure time

focus

Wikipedia