HQ Team
October 13, 2024: Pfizer Inc., got a nod from the US drug regulator for its drug Hympavzi to treat two types of a rare blood disorder in haemophilia patients.
The Food and Drug Administration approved the drug for patients without antibodies that stop the production of the blood-clotting proteins called factor VIII or factor IX based on the results of a late-stage trial.
Results from the phase 3 trial in the US in 116 adults and adolescents with haemophilia A or B without inhibitors showed the injection “cut the annualized bleeding rate for treated bleeds by 35% and 92% compared to on-demand treatment or routine prophylaxis,” according to a company statement.
Hympavzi is the first and only anti-tissue factor pathway inhibitor approved in the US for the treatment of haemophilia A or B and the first haemophilia medicine approved in the country to be administered via a pre-filled, auto-injector pen, according to the statement.
Sub-cutaneous
The drug has a subcutaneous treatment option with a once-weekly dosing schedule and minimal preparation required for each administration.
“Hympavzi aims to reduce the current treatment burden by meeting an important need for these patients, including many who have required frequent, time-consuming intravenous treatment infusion regimens,” said Suchitra S. Acharya, MD, Director of Hemostasis and Thrombosis Center Northwell Health.
The approval “is a meaningful advancement for people living with haemophilia A or B without inhibitors for bleed prevention, with a generally manageable safety profile and a straightforward once-weekly subcutaneous administration,” she said.
Haemophilia is a family of rare genetic blood diseases caused by a clotting factor deficiency, which prevents normal blood clotting.
Improper blood clot
It is diagnosed in early childhood and impacts more than 800,000 people worldwide. The inability of the blood to clot properly can increase the risk of painful bleeding inside the joints, which can cause joint scarring and damage.
People living with haemophilia can suffer permanent joint damage following repeated bleeding episodes.
“Hympavzi is Pfizer’s second haemophilia treatment to receive FDA approval this year and is the latest meaningful scientific advancement in our more than 40-year commitment to improving care for people living with haemophilia,” said Aamir Malik, Chief US Commercial Officer and Executive Vice President, of Pfizer.
The Manhattan, New York City-headquartered company got FDA clearance for its haemophilia B gene therapy Beqvez in April 2024.
“The availability of this therapy represents a powerful step forward in advancing care for more individuals and families in the bleeding disorders community,” said Phil Gattone, President and CEO of the National Bleeding Disorders Foundation.
