Novadip Biosciences, a late-stage clinical biotechnology company specializing in regenerative medicine, announces that the FDA has granted the Regenerative Medicine Advanced Therapy (RMAT) designation to its tissue regeneration product, NVD003, for the treatment of congenital pseudarthosis of the tibia (CPT).
 
The RMAT designation, issued to cell therapies, therapeutic tissue engineering products, and human cell and tissue products, demonstrates the FDA’s confidence in Novadip’s preliminary clinical evidence that NVD003 has the potential to address unmet medical needs in CPT, a rare pediatric bone condition. This designation can only be granted within the context of an IND, which Novadip received in 2021.
 
Supporting results for the FDA’s RMAT decision came from a phase 1b/2a trial (NCT05693558) which treated four patients with CPT. Combining the 12-month results from this trial together with those from four other children previously treated with NVD003 in two compassionate use programs in Belgium, 88% of patients, most of whose prior surgeries had failed, achieved healing of their fractures with NVD003.
 
“The RMAT designation is a major achievement for Novadip on our path towards getting NVD003 on the market in 2027, especially in the US,” said Denis Dufrane, MD, PhD, CEO of Novadip. “NVD003 perfectly matches the FDA requirements for this designation, as it is a tissue regeneration product intended as a treatment for CPT, a serious, rare and debilitating condition affecting children.”
 
“Given the inherent novelty of regenerative therapies, there’s often no regulatory precedence for what evidence is necessary to demonstrate efficacy in rare disease indications,” said Judy Ashworth, MD, chief medical officer at Novadip. “Receiving RMAT designation for NVD003 to treat children with CPT gives us confidence that the FDA finds our approach and preliminary results compelling and that we are on the right track as we kick off our pivotal phase 3 trial this month.”
 
In 2020, Novadip received Orphan Drug Designation and Rare Pediatric Designation from the FDA for NVD003 for the treatment of CPT, followed by Fast Track Designation in 2023.
 
Impacting less than 3.5 in 150,000 live births, CPT is a rare condition for which treatment is difficult. Once a fracture occurs, subsequent fractures are likely. 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 patients to ultimately undergo amputation of the limb.
 
Before the end of June 2025, the first clinical site will open in a pivotal phase 3 trial in CPT with recruitment in the US and Europe. Enrollment of patients will start soon after.
 
NVD003 represents potential peak sales of $1.4bn for large bone defects in pediatric and adult patients.