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)