On April 20, 2022, according to the official website of the Center for Drug Evaluation of National Medical Products Administration (NMPA), VGB-R04 injection (hereinafter referred to as "the product"), the first gene replacement therapy drug independently developed by Shanghai Vitalgen BioPharma Co., Ltd. (hereinafter referred to as "Vitalgen"), was officially granted a tacit approval for clinical trials by NMPA. The product is intended for hemophilia B caused by congenital deficiency of the coagulation factor FIX.
Photograph：CDE
Hemophilia B is an inherited, X-linked, recessive disorder that results from deficiency of the functional plasma coagulation factor IX (hFIX). VGB-R04 is a rAAV product containing a variant hFIX Padua expression cassette under the control of a liver specific promoter. The hFIX Padua variant is a natural mutant found in an HB family with increased specific activity. After intravenous injection of VGB-R04, the hFIX expression cassette will be delivered into the liver cells and become episomal to continuously express and secret hFIX Padua protein into the circulation to replace the missing hFIX protein to normalize the blood clotting function in hemophilia B patients.
Due to its expected long-term expression of hFIX, this product will be a one-time i.v. administration to provide long-term treatment for the hemophilia B patients.
The tacit approval of the clinical trial for VGB-R04 injection will open up the possibility of more treatment options for hemophilia B patients.
Early & Exploratory Phase Studies Ongoing
The "clinical study to evaluate the safety and tolerability of VGB-R04 in adult hemophilia B patients" is currently being recruited by Blood Diseases Hospital, Chinese Academy of Medical Sciences. The first patient has been enrolled in the study, and the plasma FIX activity levels have continued to improve after the VGB-R04 treatment, with no treatment related adverse events observed during follow-up visits..
ODD Granted by FDA
VGB-R04 was granted Orphan Drug Designation (ODD) by FDA on December, 2021. It is the first in vivo gene therapy product developed in China for hemophilia B that has received ODD, laying a solid foundation for the international development of this product.
