Mereo setrusumab gets FDA rare pediatric disease designation for osteogenesis imperfecta

The FDA granted Mereo BioPharma’s (NASDAQ:MREO) setrusumab rare pediatric disease designation for the treatment of osteogenesis imperfecta (OI).

OI, also known as brittle bone disease, is caused by a genetic mutation in the gene responsible for producing collagen. Setrusumab is an anti-sclerostin antibody designed to encourage bone formation. 

In Mereo’s Phase 2b ASTEROID study, setrusumab demonstrated a dose-dependent bone building effect and a trend of reduction in fractures in adults with OI.

“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,” Denise Scots-Knight, Ph.D., Mereo’s CEO, said in a statement.

Dr. Scots-Knight noted that both the FDA and EMA have agreed on the principles of a design of a single Phase 3 pediatric study in OI.

“We believe there is a clear path forward for setrusumab in OI and are continuing discussions with potential partners prior to the initiation of a Phase 3 study consistent with our company strategy,” she added.