Gene therapy can increase FVIII levels

After the third patient, all patients were started on prophylactic corticosteroid therapy. To date, no additional patients have experienced abnormal ALT levels.

BioMarin said it plans to discuss these findings with UK regulatory authorities prior to dosing the remaining patients.

“We are encouraged by this early data on BMN 270 and the trend we are seeing in increasing FVIII levels over time,” said Hank Fuchs, MD, chief medical officer at BioMarin.

“BMN 270 could have the potential to reduce and possibly eliminate the need for infusions of FVIII.”

BMN 270 has received orphan drug designation from the European Commission and US Food and Drug Administration. Phase 3 design preparation and high-volume manufacturing plans are underway.