Mont Saint-Guibert, Belgium – Novadip Biosciences, a late-stage clinical biotechnology company specializing in regenerative medicine, today announces the recruitment of the first pediatric patients for the pivotal phase 3 trial of its tissue regeneration product NVD003 in congenital pseudoarthrosis of the tibia (CPT).
Phoenix Children’s Hospital, (Phoenix, AZ, USA), and Cliniques universitaires Saint-Luc, (Brussels, Belgium) have each recruited one pediatric patient, a toddler aged 3 and a teenager respectively.
The trial in Belgium is spearheaded by Professor Pierre-Louis Docquier, pediatric orthopedic surgeon at the Cliniques universitaires Saint-Luc. He is also the coordinating investigator of this trial. Taking the lead at Phoenix Children’s Hospital is Dr. Mohan Belthur, pediatric orthopedic surgeon and clinical assistant professor, Department of Child Health, University of Arizona College of Medicine in Phoenix.
Other sites recruiting patients include: Loma Linda University Children’s Health (Loma Linda, CA, USA), LifeBridge Health (Baltimore, MD, USA) the Mayo Clinic (Rochester, MN, USA), the hospital Necker-Enfants Malades (Paris, France), 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 this 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 be monitored for an additional four years to assess the durability and safety of NVD003.
Of the eight children with CPT treated to date in previous trials with NVD003, seven have shown to have achieved durable union after two to seven years of follow-up. Most had experienced multiple failed procedures prior to being treated with NVD003. For this trial, the pediatric patients will be enrolled whether it’s their first or fifth surgical procedure. They will undergo graft surgeries three months after adipose tissue procurement.
“I’m so proud of our team and all our colleagues involved in getting us to this point,” said Judy Ashworth, MD, chief medical officer of Novadip Biosciences. “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 a bowing of the tibia that progresses to a spontaneous fracture. Once a fracture occurs, subsequent fractures are likely. This often occurs between six and 18 months following 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 face amputation of the limb.
Novadip received Orphan Drug Designation and Rare Pediatric Designation from the FDA for NVD003 for the treatment of CPT in 2020, followed by Fast Track Designation in 2023 and RMAT Designation in 2025.
Limb sparing: a high and unmet need in orthopedics
“It has been a long road from discovery to finally starting this strategic phase 3 trial,” said Denis Dufrane, MD, PhD, CEO and founder of Novadip Biosciences. “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 game-changing treatment for severe and recalcitrant bone defects and saving limbs.”
Novadip is currently working with the FDA and EMA to further explore the use of NVD003 in other populations with critical size bone defects in the context of limb-sparing, with the goal of starting a phase 3 clinical trial in adults in Q3 2026. The trial will focus on limb sparing associated with bone non-union (BNU). To date, a total of nine adults with recalcitrant BNU following traumatic fractures have been treated with NVD003. NVD003 has been shown to be safe and has achieved 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 innovative therapies. The company’s goal is to achieve market availability of NVD003 for pediatric patients by the end of 2027 and for adult patients in 2029, with the aim of achieving potential peak sales of $1.6 billion (€1.4 billion).
About NVD003
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).
About Novadip Biosciences
Novadip is a late-stage clinical biotech company aiming at advancing the standard of care for patients undergoing bone and tissue regenerative treatment. Based on the scientific discoveries of founder Prof. Dr. Denis Dufrane, MD, PhD, and research from UCLouvain and St. Luc University Hospital, the company is developing its unique 3M³ tissue regeneration technology platform, designed 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 two lead products: NVD003, an autologous cell-based therapy currently in phase 3 trials for congenital pseudarthrosis of the tibia in the US and EU, 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 (Investigational New Drug) 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. For more information please visit www.novadip.com .
Contact
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