The FDA approval was based on findings from the global Phase 3 GENEr8-1 study in which the annualised bleeding rate among patients reduced 52% after receiving Roctavian
FDA Centre for Drug Evaluation and Research. (Credit: The U.S. Food and Drug Administration from Wikimedia Commons)
Biotechnology company BioMarin Pharmaceutical has secured US Food and Drug Administration (FDA) approval for its Roctavian (valoctocogene roxaparvovec-rvox) gene therapy for the treatment of adults with severe haemophilia A.
The gene therapy was approved for haemophilia A patients without pre-existing antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test.
It is said to be a one-time gene therapy product which is administered as a single dose via intravenous infusion. The therapy consists of a viral vector carrying a gene for clotting Factor VIII.
The FDA approval was based on findings from the global Phase 3 GENEr8-1 study.
In the study, 134 patients were treated with Roctavian who were previously treated with Factor VIII replacement therapy.
In the trial, BioMarin collected baseline annualised bleeding rate (ABR) data from 112 patients for at least six months on FVIII prophylaxis prior to receiving Roctavian.
The baseline ABR was collected from the remaining 22 patients retrospectively and all the patients were followed for at least 3 years.
Post-infusion, 112 patients’ cohort experienced a mean ABR reduction of 52% after receiving Roctavian compared to their baseline ABR while receiving routine FVIII prophylaxis. The rate was reduced from 5.4 bleeds per year at baseline to 2.6 bleeds annually.
According to the biotechnology company, these results were based on an FDA analysis that imputed an ABR of 35 in 13 patients when they were on prophylaxis.
The patients also showed a significant reduction in the rate of spontaneous bleeds and joint bleeds following treatment with Roctavian compared to their baseline rate.
BioMarin chairman and CEO Jean-Jacques Bienaimé said: “Today’s approval of Roctavian builds on BioMarin’s proven track record of advancing treatments that target the underlying cause of life-threatening genetic conditions, which has produced eight best- or first-in-class commercial therapies.
“We are proud to now offer adults with severe haemophilia A, a one-time, single-dose treatment option.”
The company plans to study the long-term effects of the therapy by following all trial participants for up to 15 years. It will also assess the treatment in post-approval studies to follow those dosed in a real-world setting for 15 years or more.