Jan 11, 2022

BioMarin recently shared a community update on their Hemophilia A Clinical Development Program for valoctocogene roxaparvovec, the company’s investigational gene therapy.

valoctocogene roxaparvovec is being investigated for safety and efficacy in adults with severe hemophilia A via multiple clinical trials, including a phase 1/2 study with 15 enrolled participants who are now in long term follow-up after a single dose administration of the therapy.

The program also includes an ongoing phase 3 study known as GENEr8-1. This trial has 134 participants enrolled, all of whom have received a single dose of valoctocogene roxaparvovec and have been observed for at least two years post treatment. Biomarin announced further updates to GENEr8-1 in a January 9th press release. The two-year analysis showed that annualized bleeding rate was reduced by 85%. The data also pointed to a significant reduction in requisite factor VIII (FVIII) product usage amongst trial participants, with average annualized FVIII infusions reduced by 98% from the baseline post administration.

In terms of safety, BioMarin reported that no GENEr8-1 participants developed an inhibitor response to FVIII, malignancy, or experienced thromboembolic events. In addition, “no new safety signals emerged, and no treatment-related serious adverse events (SAE) were reported,” in year two of the trial.

The company referenced their plans going forward in their community update:

“In the United States, BioMarin intends to submit two-year follow-up safety and efficacy data on all study participants from the Phase 3 GENEr8-1 study to support the benefit/risk assessment of valoctocogene roxaparvovec, as previously requested by the Food and Drug Administration (FDA). Based on these results, BioMarin is planning to meet with FDA to discuss the resubmission of a Biologics License Application (BLA) targeted for the second quarter of 2022, followed by an expected six-month review by the FDA.”

Source: Biomarin, Clinical Development Program Update, January 2022, Biomarin press release dated January 9th

Stay connected with us! Subscribe to receive the latest news on treatments, clinical trials, advocacy issues, and NHF events.