US FDA approves Pfizer's gene therapy for rare bleeding disorder
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 [April 26, 2024]
By Sriparna Roy
(Reuters) -The U.S. Food and Drug Administration approved Pfizer's gene
therapy for hemophilia B on Friday, the second such therapy for the rare
bleeding disorder that typically requires regular infusions of a
blood-clotting protein.
People with hemophilia have a fault in a gene that regulates production
of proteins called clotting factors, which can cause spontaneous as well
as severe bleeding following injuries or surgery. It predominately
affects males.
Pfizer's one-time therapy, branded as Beqvez, is designed to stimulate
production of the protein, called factor IX (FIX), by the patient's own
body instead of intravenous infusions of FIX multiple times a week or a
month.
The therapy will be available by prescription to eligible patients this
quarter, a company spokesperson said.
Pfizer has set a list price for Beqvez at $3.5 million in the U.S. - the
same price tag as Australian drugmaker CSL Ltd's rival gene therapy
Hemgenix.
Gene therapies are often pricey, and the durability of a single-dose
treatment remains an open question.
High cost, logistical issues and the prospect of potential treatment
advances have so far held back adoption of the first gene therapies for
hemophilia.
Nigel Key, director of the Hemophilia and Thrombosis Center at the
University of North Carolina said the demand for gene therapies is
limited by "how satisfied they (patients) are with their current
treatment...and whether they want to dive right in, which not many do".
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Signage is seen outside of the Food and Drug Administration
(FDA) headquarters in White Oak, Maryland, U.S., August 29, 2020.
REUTERS/Andrew Kelly/File Photo
 A single dose of Pfizer's therapy
was shown to work as well as standard-of-care protein infusions
after a year, with bleeding completely eliminated in 60% of patients
versus 29% who received infusions.
Pfizer said it will continue to monitor for long-term durability and
safety of the treatment over the course of 15 years.
Hemophilia B is estimated to affect nearly 4 in every 100,000 U.S.
males, while related disorder hemophilia A is estimated to affect 12
in every 100,000 U.S. males, according to government data.
(Reporting by Sriparna Roy in Bengaluru; Editing by Shailesh Kuber
and Krishna Chandra Eluri)
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