Novadip Biosciences has recruited the first pediatric patients for a pivotal phase 3 trial evaluating its tissue regeneration product NVD003 in treating congenital pseudoarthrosis of the tibia (CPT).
 
Phoenix Children’s Hospital, (Arizona), and Cliniques universitaires Saint-Luc, (Brussels, Belgium) have each recruited one pediatric patient—a 3-year-old toddler and teenager, respectively.
 
The Belgian trial is spearheaded by Professor Pierre-Louis Docquier, pediatric orthopedic surgeon at the Cliniques universitaires Saint-Luc. He is also the trial’s coordinating investigator. Taking the lead at Phoenix Children’s Hospital is Dr. Mohan Belthur, pediatric orthopedic surgeon and clinical assistant professor in the Department of Child Health at the University of Arizona College of Medicine in Phoenix.
 
Other sites recruiting patients include Loma Linda University Children’s Health (Loma Linda, Calif.), LifeBridge Health (Baltimore) the Mayo Clinic (Rochester, Minn.), the hospital Necker-Enfants Malades (Paris), the CHU Amiens-Picardie (Amiens, France), and the hospital Sant Joan de Déu (Barcelona, Spain).
 
At least 12 children with CPT fractures will be enrolled in the phase 3 trial by end of Q1 2026. The trial will measure clinical and radiological healing at 12 months post-graft surgery with NVD003. Consenting participants will also be monitored for an additional four years to assess NVD003’s durability and safety.
 
Of the eight children with CPT treated to date in previous trials with NVD003, seven have achieved durable union after two to seven years of follow-up. Most had experienced multiple failed procedures before being treated with NVD003. For this trial, pediatric patients will be enrolled regardless of their surgical histories (or lack thereof). They will undergo graft procedures three months after adipose tissue procurement.
 
“I’m so proud of our team and all our colleagues involved in getting us to this point,” Novadip Biosciences Chief Medical Officer Judy Ashworth, M.D., stated. “Clinical development is challenging enough for more common diseases. It takes high motivation, commitment and resilience to implement a trial in a rare pediatric condition. But knowing that our work can have such a major impact on the lives of children with CPT, that’s what keeps us all focused on the endgame. We expect to complete recruitment for this trial by end of Q1 2026.”
 
Impacting less than 3.5 in 150,000 live births, CPT is a rare condition for which treatment is difficult. Children with CPT are typically born with tibial bowing that progresses to a spontaneous fracture. Once a fracture occurs, subsequent fractures are likely. This often occurs between six and 18 months after minimal trauma or from weight-bearing as these children learn to walk. They commonly undergo multiple surgical procedures before the fractured bone heals. While children with CPT can face impaired mobility and years of corrective surgeries to try to repair and stabilize the bone, it is not uncommon for them to ultimately need their limb amputated.
 
Novadip received Orphan Drug Designation and Rare Pediatric Designation from the FDA for NVD003 for CPT treatment in 2020, followed by Fast Track Designation in 2023 and RMAT Designation in 2025.
 
“It has been a long road from discovery to finally starting this strategic phase 3 trial,” Novadip Biosciences Founder/CEO Denis Dufrane, M.D., Ph.D., said. “As the inventor of NVD003, I’m so excited that our pivotal trial has now launched, moving us closer to putting this technology on the market to help restore full mobility in children with CPT and to prevent amputations. This is a huge step towards our vision: a treatment for severe and recalcitrant bone defects and saving limbs.”
 
Novadip is currently working with the U.S. Food and Drug Administration (FDA) and EMA to further explore using NVD003 in other populations with critical size bone defects; the company is hoping to start a phase 3 clinical trial in adults in Q3 2026. That trial will focus on limb sparing associated with bone non-union (BNU). To date, nine adults with recalcitrant BNU after traumatic fractures have been treated with NVD003 and the product has been safe and helped achieve bone healing in 88% of patients.
 
Novadip is preparing a Series C round to accelerate its late-stage development and drive the commercialization of its therapies. The company is striving to commercialize NVD003 for pediatric patients by the end of 2027 and for adults in 2029; peak sales are projected to be $1.6 billion (€1.4 billion).
 
NVD003 is a three-dimensional (3D) osteogenic graft derived from autologous adipose derived mesenchymal stem cells (ASCs) combined with hydroxyapatite/beta-tricalcium phosphate (HA/TCP) particles. NVD003 was specifically developed to improve bone healing in severe pathophysiological conditions (e.g. hypoxia, lack of mineralized callus formation, bone resorption and low osteogenicity) as found in congenital pseudarthosis, bone tumors (after an extensive surgical resection), osteolytic syndromes such as Gorham-Stout disease, genetic bone resorption syndromes with osteoporosis as found in Hajdu-Cheney syndrome, and following severe trauma (casualties of war).
 
Novadip is a late-stage clinical biotech company advancing the standard of care for patients undergoing bone and tissue regenerative treatment. Based on the scientific discoveries of Dr. Dufrane, and research from UCLouvain and St. Luc University Hospital, the company is developing its unique 3M³ tissue regeneration technology platform, which aims to create a new class of regenerative tissue products that accelerate the healing of large bone defects, bone non-union, and spine fusion in a single treatment, for patients with limited or no treatment options.

Novadip’s pipeline includes NVD003, an autologous cell-based therapy currently in phase 3 trials for congenital pseudarthrosis of the tibia in the United States and European Union, and about to start phase 3 clinical trials in adults with bone non-union; and NVDX3, an allogenic bone grafting material currently in phase 1b/2a trials in trauma surgery and lumbar intervertebral spine fusion. The FDA has granted approval to start a phase 2b/3 IND trial with NVDX3 in level two cervical spine fusion. Founded in 2013 in Belgium, Novadip has 53 team members. Since inception, it has raised €116 million in equity and non-dilutive funding including financing from the European Investment Bank.