Forbes

Despite Eye-Popping $3.5 Million Price Tag For Gene Therapy Hemgenix, Budget Impact For Most Payers Will Be Relatively Small

Forbes contributors publish independent expert analyses and insights. Joshua Cohen is a Boston-based writer who covers health policy. On November 22 nd, the Food and Drug Administration (FDA) approved ...
Nasdaq

CSL Behring Announces the First Patient Has Received FDA-Approved HEMGENIX® (etranacogene dezaparvovec-drlb) for Hemophilia B

HEMGENIX®, the first and only FDA approved gene therapy for hemophilia B, has been proven to elevate and sustain factor IX levels for years, significantly reduce the rate of annual bleeds versus ...
FiercePharma

ASH: CSL proves efficacy of hemophilia B gene therapy Hemgenix holds up after 5 years

Three years after introducing Hemgenix, the first gene therapy for hemophilia B, CSL Behring has released long-term data proving the treatment’s benefits remain durable after five years. The five-year ...
FiercePharma

After Hemgenix approvals, uniQure cashes out some of its royalty rights for $375M-plus

Months after the U.S. and European approvals of its CSL-partnered hemophilia A gene therapy Hemgenix, uniQure is selling some royalty rights for up to $400 million. In a deal with HealthCare Royalty ...
Benzinga.com

uniQure's Hemgenix Royalty Sales Agreement Extends Cash Runway, Relieves Near-Term Commercial Risk, Says Analyst

uniQure N.V. QURE agreed to sell a portion of the royalty rights due from CSL Behring from Hemgenix (etranacogene dezaparvovec-drbl) net sales to HealthCare Royalty and Sagard Healthcare for up to ...
Labroots

The most expensive drug in the world, Hemgenix, approved at $3.5 million per dose

On Tuesday, November 22nd, 2022, the United States Food and Drug Administration approved Hemgenix, a new drug to treat hemophilia. Hemophilia is a lifelong & rare blood disorder where the blood cannot ...
Seeking Alpha

CSL Behring's Gene Therapy HEMGENIX® (etranacogene dezaparvovec-drlb) Four Years Post-Infusion Data Continue to Show Sustained Efficacy and Safety in Adults with Hemophilia B

94 percent of patients eliminated factor IX prophylaxis and remained free of continuous prophylaxis through four years post-treatment Mean factor IX activity levels were sustained at near normal ...
Pharmabiz

CSL’s gene therapy, Hemgenix receives US FDA approval for haemophilia B

Global biotechnology leader CSL announced that the US Food and Drug Administration (FDA) approved Hemgenix (etranacogene dezaparvovec-drlb), the first and only one-time gene therapy for appropriate ...
Nasdaq

Data Published in the New England Journal of Medicine Confirm the Long-term Durability and Safety of HEMGENIX® (etranacogene dezaparvovec-drlb) Over Five Years

- 94% of patients (51 of 54) remained free from the burden of continuous prophylaxis treatment through five years following a single infusion of HEMGENIX, demonstrating sustained therapeutic benefit- ...
PharmiWeb

First Gene Therapy for Hemophilia B, CSL’s HEMGENIX®, Approved by the European Commission

MARBURG, GERMANY – February 21, 2023 – Global biotechnology leader CSL (ASX: CSL) today announced that the European Commission has granted conditional marketing authorization (CMA) for HEMGENIX ® ...
Medindia

CSL Behring's Gene Therapy HEMGENIX® (etranacogene dezaparvovec-drlb) Four Years Post-Infusion Data Continue to Show Sustained Efficacy and Safety in Adults with Hemophilia B

KING OF PRUSSIA, Pa., Feb. 7, 2025 /PRNewswire/ -- Global biotechnology leader CSL (ASX:CSL; USOTC:CSLLY) today announced the four-year results from the pivotal HOPE-B study confirming the long-term ...