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CSL flags global stockout of bleeding disorder gene therapy
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CSL flags global stockout of bleeding disorder gene therapy
Mar 17, 2026 10:00 AM

March 17 (Reuters) - Australia's CSL said it is

facing a temporary global stockout of its bleeding disorder gene

therapy Hemgenix, which will delay treatment for some patients

in countries where the drug is already commercially available.

Here are some details:

* CSL said the supply issue is not linked to the safety or

effectiveness of Hemgenix, but to the complexity of making gene

therapies and the company's quality standards.

* Hemgenix is a one-time intravenous gene therapy for adults

with hemophilia B, a rare inherited condition that makes it hard

for blood to clot.

* The company said it is working with regulators on steps to

secure a stable supply of Hemgenix while maintaining high

manufacturing and quality requirements.

* U.S. drugmaker Pfizer ( PFE ) had stopped global

development and commercialization of its hemophilia gene

therapy, Beqvez, last year citing soft demand from patients and

their doctors.

* Hefty price tags, logistical issues and the prospect of

potential treatment advances were holding back the adoption of

gene therapies for hemophilia, according to doctors.

* CSL said the delay could affect some eligible patients who

were planning to receive the one-time treatment in countries

with existing access.

* The company said it remains confident in Hemgenix and is

committed to making the treatment available to adults with

hemophilia B when supply allows.

(Reporting by Sahil Pandey in Bengaluru)

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