Novadip Biosciences and Cliniques universitaires Saint-Luc have reported successful long-term outcomes in four children treated with NVD003.

This is a regenerative bone graft, for congenital pseudarthrosis of the tibia (CPT) — a rare and debilitating pediatric condition.

CPT affects between 1 in 140,000 and 1 in 250,000 live births and can lead to chronic pain, limited mobility, and, in some cases, limb amputation. Treating the condition is notoriously difficult, often involving repeated surgeries over several years.

In this compassionate use programme, coordinated through Belgium’s medicines agency AFMPS, four children were treated with NVD003 between 2018 and 2021. After four to seven years of follow-up, all patients had fully healed fractures with no recurrence, and no safety concerns related to the therapy were reported.

Professor Pierre-Louis Docquier, the treating physician at Saint-Luc and a specialist in pediatric orthopaedic surgery, said: “Critical size and non-healing bone defects are among the most difficult conditions to treat in orthopedic surgery. The results after several years of this innovative regenerative therapy in these young patients are simply exceptional.”

NVD003 is a scaffold-free, autologous bone graft developed by Novadip. Created from a patient’s own cells, the therapy is engineered into an implantable paste tailored to the individual’s bone defect.

The therapy is also being evaluated in clinical trials. Novadip recently announced interim 12-month data from its phase 1b/2a pilot trial in four additional children with CPT. Based on the results, a phase 3 trial is scheduled to begin enrolment this month, with Prof. Docquier serving as the coordinating investigator.

Dr Denis Dufrane, Novadip’s founder and CEO, said the outcomes offered hope for other children facing CPT: “We are working hard to bring this product to the market as quickly as we can.”