China-based Vcanbio Cell & Gene Engineering Corp., Ltd (SHA: 600645) has announced that it has received orphan drug designation (ODD) from the US FDA for its VUM02 (human umbilical cord-derived mesenchymal stem cells), a Category 1 therapeutic biologic product for the treatment of idiopathic pulmonary fibrosis (IPF).
VUM02: An Innovative Stem-Cell Preparation
VUM02 is an in-house developed, cryopreserved stem-cell preparation derived from the umbilical cord tissue of healthy fetuses. The product undergoes isolation, screening, and amplification in vitro before being used as a therapy. VUM02 is being developed to treat patients with IPF, chronic and acute (subacute) liver failure, acute respiratory distress syndrome (ARDS), and other conditions. The IPF indication for VUM02 was approved for clinical study in China in April 2023.
Implications of Orphan Drug Designation
The orphan drug designation from the US FDA is a significant milestone for VUM02, as it recognizes the product’s potential to treat a rare disease with a high unmet medical need. This designation can provide Vcanbio with certain development incentives, including tax benefits, market exclusivity, and potential funding support, which are crucial for advancing therapies like VUM02 that target conditions with smaller patient populations.-Fineline Info & Tech
