~~<i id='A6B6B7B219'><strike id='A6B6B7B219'><tt id='A6B6B7B219'><pre id='A6B6B7B219'></pre></tt></strike></i>~~

00:00

00:00 00:00 LIVE

buffering

Replay

explore

leisure time

author：knowledge 2025-10-02 11:09:08 - browse：11

The Food and Drug Administration on Thursday approved a gene therapy to treat people with hemophilia A, an inherited and rare bleeding disorder.

The treatment, called Roctavian, is made by BioMarin Pharmaceutical. It was first approved in Europe in August 2022.

Roctavian is a one-time therapy that, in clinical trials, dramatically reduced bleeding episodes and helped patients live without the blood transfusions used to treat the disease. The FDA first rejected Roctavian in 2020, asking BioMarin to amass more data on its safety and long-term durability, and then delayed its decision again to account for additional evidence.

Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!

GET STARTED Log In

Previous article： Drug repurposing or repositioning? The language matters
Next article： Syphilis numbers in the United States reach record heights

leisure time

focus

leisure time

Wikipedia

entertainment

focus

JUSTINTALLIS/AFPviaGettyImagesWanttostayontopofthescienceandpoliticsdrivingbiotechtoday? Signuptoget

WSS

Venture capital firm's plans to take over Akron hospital face hurdles

STAT series 'Denied by AI' wins major journalism prizes

Merck CEO won’t testify in Senate, citing no expertise in drug prices

Wegovy leads to weight loss. Can it treat depression, too?

Reconstructing family trees of blood cells may help predict disease

Boston competes with Raleigh

explore

leisure time

leisure time

Anesthesiologist group: stop taking Ozempic before surgery

Readout Newsletter: Raw milk, Wegovy tests, Eisai and Biogen

After affirmative action ruling, medical educators look to 'holistic review'

Why Medicare shouldn’t cover every FDA

Cancer drug shortages should be causing more outrage

Investment in vaccine development is lagging, report finds

Medical records are filled with copy

Health Care Service Corp to buy Cigna Medicare Advantage plans

Indiana Supreme Court upholds abortion ban, says state constitution gives only limited protections

Hottest summer in 2,000 years puts focus on extreme heat, health

focus

Boston hospital trains restaurant workers on naloxone for overdoses

Abbott's new stent may help patients with peripheral artery disease

What a koala virus tells us about the human genome

Raw milk easy to obtain despite bird flu warning, FDA interstate ban

Readout Newsletter: CG Oncology IPO, Eli Lilly, Tesla, Merck

The latest on Bluebird gene therapies, Vertex's pain drug, and ADC FOMO

leisure time

Wikipedia

entertainment

focus