Global Newsroom

KING OF PRUSSIA, PA, USA
- If approved, etranacogene dezaparvovec would be the first gene therapy option for people living with hemophilia B
- This milestone underscores CSL Behring’s promise to develop and deliver a truly unique portfolio of patient-focused therapies for people with rare and serious medical conditions

Statement attributable to Greg Boss, Executive Vice President - Legal, and CSL Limited Group General Counsel.

The 500 million international units (IUs) will include product specifically manufactured for donation allowing for a longer shelf life and enabling more people around the world to access life-saving therapy.

- Marketing Authorization Application (MAA) for etranacogene dezaparvovec will be reviewed under accelerated assessment and has the potential to be the first gene therapy for patients living with hemophilia B.
- MAA filing is supported by the phase 3 HOPE-B study which demonstrated durable and sustained therapeutic effect after a single infusion.
- Milestone underscores CSL Behring’s promise to develop and deliver a truly unique portfolio of patient-focused therapies.

KING OF PRUSSIA, Pa.

With 25,000 global employees, CSL is one of 500 Large Employers recognized across 25 industry sectors

KING OF PRUSSIA, PA

- Data from the largest gene therapy study in Hemophilia B to date shows that etranacogene dezaparvovec is statistically superior in reducing annualized bleeding rate compared to baseline FIX prophylactic therapy
- Following a single administration of etranacogene dezaparvovec, participants maintained stable Factor IX (FIX) activity through 18 months
- Therapeutic benefit observed in patients with pre-existing anti-capsid neutralizing antibody levels up to a titer of 1:678

CSL continues to provide medicines to patients around the world.

KING OF PRUSSIA, PA, USA

Lexington, MA and Amsterdam, the Netherlands, King of Prussia, PA

~ Largest gene therapy study in hemophilia B achieved primary endpoint of non-inferiority in annualized bleeding rate after stable Factor IX (FIX) expression, assessed at 18 months following a single dose of etranacogene dezaparvovec
~ Etranacogene dezaparvovec also achieved secondary endpoint demonstrating statistical superiority in reduction of annualized bleeding rate compared to baseline FIX prophylactic therapy
~ Stable and durable FIX levels with mean FIX activity of 36.9 percent of normal in full study population at 18-months, compared to a mean of 39.0 percent of normal at 6 months
~ Manufacturing operations supporting process validation of etranacogene dezaparvovec successfully completed by uniQure

KING OF PRUSSIA, PA and MELBOURNE, AU

• CSL advances novel pipeline spanning six therapeutic areas, four scientific platforms and two businesses (CSL Behring and Seqirus)
• Seqirus Awarded BARDA contract to develop aQIVc and sa-mRNA Pandemic Influenza Vaccines
• New collaboration with the Walter and Eliza Hall Institute for Medical Research in Australia to create a Centre for Biologic Therapies

Kankakee, IL

CSL introduced a scholarship program for U.S. employees and their dependents as part of delivering on its promise to diversity, equity and inclusion.

Here's how CSL is working around the world with academia, industry and governments to combat COVID-19

KING OF PRUSSIA, Pa. and PHILADELPHIA, Pa.

Global biotech leader CSL and the Urban League of Philadelphia are working to address some of the most pressing needs in the community, including efforts to strengthen Public Health, Leadership Development, Workforce Diversity and Job Creation and Training.

King of Prussia, Pa.

The updated guideline empowers physicians with the confidence to expand the treatment options prescribed for CIDP patients

KING OF PRUSSIA, PA

Approval received to co-package a convenience administration kit along with the product BERINERT.

KING OF PRUSSIA, Pa.

Hizentra will be covered under the same Medicare benefit category as intravenous immune globulin (IVIg), but with the convenience of self-infusing at home

KING OF PRUSSIA, Pa.

Findings signal need for novel treatment strategies to provide more protection during this high-risk, 90-day period

NEW YORK, NY, USA

Dr. Minnie Sarwal of NephroSant has been selected to receive the $25,000 Innovation Award

KING OF PRUSSIA, PA

• Etranacogene dezaparvovec (AMT-061) is an investigational gene therapy that may potentially provide people with hemophilia B with years of functional levels of Factor IX, a blood-clotting protein that prevents excessive bleeding.

• uniQure will complete the Phase 3 HOPE-B trial and scale up manufacture for initial commercial supply under an agreed plan with CSL Behring.

King of Prussia, Pa

The label update is based on open label extension data from the landmark PATH (Polyneuropathy And Treatment with Hizentra) study