FDA Grants Rare Pediatric Disease Designation to Novadip’s Treatment for Rare Bone Disease

Rare Daily Staff

The U.S. Food and Drug Administration has granted Rare Pediatric Disease and Orphan Drug designations for Novadip Biosciences NVD‑003, a cell therapy for the treatment of congenital pseudarthrosis of the tibia, a rare debilitating condition that primarily affects children.

Congenital pseudarthrosis of the tibia (CPT) is a shin bone fracture that fails to heal properly on its own that is present at birth or manifests in early childhood when starting to walk. The underlying cause of CPT is not completely understood but it leads to abnormal structure of the bone tissue in the tibia, and sometimes fibula, combined with abnormal vascularization of the affected tissue. The natural history of the disease is extremely unfavorable. Despite various medical techniques to restore bone union and vasculature, the final prognosis of CPT remains poor and the risk of amputation cannot be eliminated.

Novadip’s lead product candidate, NVD-003, is a clinical-stage experimental cell-based therapy in which stem cells obtained from the patient’s fatty tissue are cultured in vitro to become a biomaterial consisting of bone forming cells embedded in their self-secreted extracellular matrix together with added hydroxyapatite particles, a mineral naturally present in bone to confer initial strength. The resulting product is in the form of moldable putty that a physician can use to fill small as well as large bone defects using minimally invasive surgery techniques. Upon implantation, the bone-forming cells protected in their self-secreted matrix from the harsh pathological environment in the bone defect continue to mature and progressively transform the putty into normal, healthy bone.

Novadip has demonstrated the potential of its technology in CPT, including a case study in a five-year old boy who had failed prior treatments. After more than two years following implantation, a sufficient bone fusion was found that allowed the patient to walk without pain and avoided amputation.

To fund further development of NVD-003, Novadip said the Walloon Region government of Belgium is providing $11.83 million (€9.4 million) of non-dilutive funding to support Novadip’s two programs: $7 million (€5.7 million) to cover a phase 1/2 trial of NVD-003, and $4.8 million (€3.7 million) for the development of NVD-X3, an allogenic version NVC-003. A clinical trial is expected to start in 2021 with all manufacturing, scientific expertise and operations to remain in Wallonia, Belgium.

“This disease has always been amongst the most difficult to treat for orthopedic specialists as even after several surgical treatments to fuse fractured bone, restoration of healthy bone tissue is hindered and the likelihood of a fracture occurring again is high, leading to severe problems with pain, mobility, and proper growth of the limb,” said Denis Dufrane, CEO at Novadip.

The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The designation makes NVD-003 eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Eiger BioPharmaceuticals sold its priority review voucher for $95 million in November 2020.

The voucher program was due to expire at the end of September but Congress provided a short-term extension of the program. According to the current statutory sunset provisions, after December 11, 2020, FDA may only award a voucher for an approved rare pediatric disease product application if the sponsor has rare pediatric disease designation for the drug, and that designation was granted by December 11, 2020. After December 11, 2022, FDA may not award any rare pediatric disease priority review vouchers.

Going forward, submitted requests for rare pediatric disease designation will be reviewed in a timely manner but the FDA’s previous 60-day response time no longer applies.

Photo: Denis Dufrane, CEO at Novadip