CSL flags global stockout of bleeding disorder gene therapy

Reuters
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CSL flags global stockout of bleeding disorder gene therapy

By Sahil Pandey

March 17 (Reuters) - Australia's CSL CSL.AX 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.N 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)

((Sahil.Pandey@thomsonreuters.com;))

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