Setrusumab was granted Rare Pediatric Disease Designation by FDA
Osteogenesis Imperfecta (OI) aka Brittle Bone Disease, characterized by a group of inherited disorders with fragile bones that break easily. The prime cause for OI is the improperly working genes. Some times the underlying causes for it are unknown. The prevalence of OI is in general 1 in 20000.
In OI, the protein collagen is severely effected and leads to damage of bones, teeth, weakness of muscles, spine etc., The life expectancy is normal however the death in people suffering from OI is majorly due to dysfunction of lungs and respiratory failure. There is no permanent cure for OI, rather it can be regulated by the drugs which fortify bones, physical therapy and surgery.
Towards their endeavor in treatment of Pediatric Osteogenesis Imperfecta, the UK based MEREO BIOPHARMA GROUP PLC has developed a drug, Setrusumab and was granted approval today by FDA for Rare Pediatric Disease Designation.
Mereo Biopharma Group PLC, is a clinical stage bio pharmaceutical company focused their research on rare disease and oncology. The drug, Setrusumab is a completely humanized monoclonal antibody that inhibits sclerostin, a protein that inhibits bone-forming cells development. The mechanism of action of Setrusumab may be especially appropriate for treatment of OI. Setrusumab has the potential to become the first approved treatment choice that could minimize fractures and improve the quality of life for individuals with OI. Mereo Biopharma Group PLC has obtained orphan drug designation in OI for Setrusumab in both the United States and the EU.
“Receiving Rare Pediatric Disease designation from the FDA highlights the significant unmet medical need facing children with OI and underscores the potential of Setrusumab to become the first approved treatment option specifically for these patients,” – Dr. Denise Scots-Knight, CEO of Mereo.