USA- California-based biotechnology company BioMarin has announced that European regulators have approved its gene treatment for hemophilia and given it the go-ahead for commercialization.
The medication, often known as Roctavian, was awarded conditional marketing approval as a treatment for a select group of patients with hemophilia A, the more frequent form of unusual bleeding disorder.
The drug should be used by persons with “severe” illness characterized by abnormally low levels of the blood-clotting protein (Issue VIII), who don’t have a history of producing antibodies that attack this protein.
Patients must also test negative for antibodies that could defend against that virus in order to be eligible for treatment.
This is because Roctavian introduces a functional copy of the Issue VIII-producing gene into cells through a modified virus.
With approval in hand, BioMarin is now attempting to secure reimbursement across all of the various member states of the European Fee.
The company’s chief executive officer, Jeff Ajer, said during a conference call that the goal is to immediately launch Roctavian in Germany, followed by France.
According to him, the company anticipates that Roctavian’s listing value in Europe would be “about” 1.5 million euros, or roughly US$1.5 million, after all the deductions.
Johannes Oldenburg, director of the Hemophilia Centre at the University Clinic in Bonn, Germany commented that this approval in the EU represents a medical breakthrough in the treatment of patients with severe hemophilia A.
Hemophilia A, also known as factor VIII (8) deficiency or typical hemophilia, is a condition brought on by a deficiency or absence of the clotting protein factor VIII (FVIII).
About one-third of instances are discovered to be unrelated, despite the fact that it is passed down from parents to children.
Hemophilia affects roughly 1 in 5,617 live male births, according to the US Centers for Disease Control and Prevention (CDC).
Men with hemophilia number between 30,000 and 33,000 in the US. The severe variant is present in more than half of hemophilia A diagnoses.
The prevalence of hemophilia A is four times that of hemophilia B. Every race and ethnicity is impacted by hemophilia.
Patients with severe hemophilia, in particular, receive infused engineered Factor VIII on a regular basis to prevent or treat episodes of uncontrollable bleeding.
These procedures can be costly and difficult for some people. They are also not perfect because patients can still bleed.
Johannes Oldenburg, director of the Hemophilia Centre at the University Clinic in Bonn, Germany commented that this approval in the EU represents a medical breakthrough in the treatment of patients with severe hemophilia A.
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