Authorization to start clinical trial in the US granted for rare paediatric bone disease
Mont-Saint-Guibert – Novadip Biosciences (“Novadip”), a clinical stage company developing treatments to regenerate impaired tissues in patients with significant unmet medical needs, today announced that the U.S. Food and Drug Administration (FDA) has approved the Company’s Investigational New Drug (IND) application for its autologous cell therapy product NVD‑003 for the treatment of Congenital Pseudarthrosis of the Tibia (“CPT”). CPT is an orphan disease, that primarily affects children with devastating long-term functional disabilities.
IND approval has been granted by the FDA allowing Novadip to conduct a pilot study with NVD-003 in the US. The trial will investigate the potential of the Company’s cell-based osteogenic product in young children, between two and eight years of age, suffering from CPT.
NVD-003 is a novel autologous cell-based osteogenic (bone healing) product that has been generated from Novadip’s proprietary tissue regeneration platform. Made from adipose stem cells, NVD-003 has a 3-dimensional structure and works by promoting bone formation. Novadip previously reported positive interim results from a Phase I/IIa study in adults with non-healing fracture of the lower limb. The results showed a clinically and radiologically meaningful improvement in patients with recurrent bone non-union after severe trauma and with no therapeutic alternatives, highlighting the ability of NVD-003 to restore natural bone healing processes. In the frame of compassionate use, young children suffering from CPT have also been treated with this innovative product and it was shown that the need for amputation could be avoided and normal quality of life (e.g. walking, cycling and horse riding) could be significantly improved.
The NVD-003 autologous bone program was granted Orphan Drug designation and Rare Paediatric Disease designation by the US Food and Drug Administration (FDA) in 2020. Following both designations and the successful IND approval, Novadip will be eligible to obtain a priority review voucher when the product reaches the market.
Dr. Denis Dufrane (MD, PhD), Chief Executive Officer and Founder, said: “Receiving IND approval for NVD-003 is a major milestone for Novadip and we are excited by the potential impact this novel therapy could have on patients with few alternative treatment options. The results that we previously reported demonstrate that Novadip’s technology is able to stimulate bone formation and we believe that NVD-003 has the potential to become a new standard of care for people that require critical size bone defect reconstruction. In addition, this data provides further validation of our proprietary platform, supporting the future success of our next generation off-the-shelf products.”