US FDA approves Pfizer gene therapy for rare bleeding disorder
Treatment called has a price tag of $3.5m, same is that of Hemgenix produced by CSL Ltd
- It is designed to stimulate production of the protein by the patient's own body instead of intravenous infusions multiple times a week or a month
- High cost, logistical issues and prospect of potential treatment advances have so far held back adoption of the first gene therapies
WASHINGTON (Reuters) – The US Food and Drug Administration approved Pfizer's gene therapy for haemophilia B on Friday, the second such therapy for the rare bleeding disorder that typically requires regular infusions of a blood-clotting protein.
People with haemophilia 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.
Read more: US approves first gene therapy for children with rare genetic disease
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 US – 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 haemophilia.
Read more: How genetic therapies transformed the lives of sickle cell patients
Nigel Key, director of the Haemophilia 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".
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 per cent of patients versus 29pc 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.
Haemophilia B is estimated to affect nearly four in every 100,000 US males, while related disorder haemophilia A is estimated to affect 12 in every 100,000 US males, according to government data.
