Pfizer’s hemophilia B gene therapy succeeds in late-stage study

US drugmaker Pfizer explained on Thursday (Dec 29) its experimental gene therapy for the remedy of hemophilia B, a scarce inherited blood problem, met its principal target in a late-stage research.

Facts from the research confirmed that a solitary dose of the treatment was superior to the present standard of care in assisting reduce the bleeding level in clients with moderately significant to serious sorts of hemophilia B.

The condition hampers the body’s capability to make a blood-clotting protein identified as component IX.

Pfizer’s remedy, fidanacogene elaparvovec, is created to enable clients produce component IX on their own right after a 1-time treatment, as opposed to current treatment options, which emphasis on regular infusions of the protein.

The drugmaker licensed its hemophilia B gene treatment from Roche’s Spark Therapeutics unit in 2014 for a US$20-million upfront payment.

Pfizer ideas to go over the late-stage information with regulatory authorities in Europe and the United States and share additional info for the experimental therapy at a scientific meeting in early 2023.

In accordance to authorities info, the estimated prevalence of hemophilia in the US is 12 circumstances per 100,000 males for hemophilia A and 3.7 instances for every 100,000 males for hemophilia B. In November, the US well being regulator accepted the first gene remedy, CSL Ltd and uniQure’s Hemgenix, to take care of hemophilia B.

Pfizer is also testing other experimental gene therapies in late-stage trials as likely treatments for the bleeding problem hemophilia A and muscular disorder Duchenne muscular dystrophy.