~~<i id='12256B7285'><strike id='12256B7285'><tt id='12256B7285'><pre id='12256B7285'></pre></tt></strike></i>~~

00:00

00:00 00:00 LIVE

buffering

Replay

knowledge

leisure time

author：explore 2025-08-12 06:34:50 - browse：77

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： What does generative AI mean for health care? We asked experts
Next article： Correcting Robert F. Kennedy Jr.'s vaccine 'facts'

focus

explore

focus

AdobeThisarticlewasadaptedfromSTAT’slatestreport,“Subgroupanalysis:howtoevaluateposthoctestsforsigni

WSS

Over 1,300 arrested in France in 4th night of protests over fatal police shooting of teen Nahel M.

Live blog: Hearing on CRISPR

After Sarepta trial failure, Duchenne families brace for more debate

How to convince healthy people to buy insurance

Bill would allow buprenorphine prescription for addiction via telehealth

Millions may soon lose Medicaid when they don't have to

knowledge

leisure time

focus

George Santos makes 1st court appearance after pleading not guilty to 13 counts

STAT Readout: The latest on Wegovy, Mounjaro, and Covid shots

Medical records are filled with copy

Canadian wildfire dangers should prompt more proactive mitigation from government: Experts

Food as medicine: CMS rules hamper 'prescribing' of fruits, veggies

U.K. agency declines to back Eli Lilly’s Mounjaro for type 2 diabetes

ChatGPT in medicine: STAT answers readers' burning questions

Prime Medicine says its gene editing passes test in monkeys

Trump demands the U.S. pay no more for drugs than other countries … again

Canadian wildfire dangers should prompt more proactive mitigation from government: Experts

focus

Altering the blood type of lungs raises potential for universal organs for transplants

STAT Readout: The latest on Wegovy, Mounjaro, and Covid shots

Living organ donors deserve priority status for transplants

How to fix the rural physician shortage in the U.S.

Novo: Oral version of Ozempic leads to 15% weight loss

Brain injuries and the importance of a second opinion

focus

explore

explore

focus