According to a story from BioSpace, the biopharmaceutical company Mereo BioPharma Group recently announced that its investigational therapy setrusumab has earned Rare Pediatric Disease designation from the US Food and Drug Administration (FDA). This designation was for the treatment of osteogenesis imperfecta, a rare disorder that affects the bones. The drug has demonstrated potential benefit for patients in a phase 2 clinical trial.

About Osteogenesis Imperfecta

Osteogenesis imperfecta, which is also referred to as brittle bone disease, is a group of genetic disorders which are distinguished by abnormally weak bones that break easily. The effect can range from mild to severe. These disorders are linked to a lack of type I collagen, leading to issues with connective tissue. In the vast majority of cases, this is caused by a mutation affecting either the COL1A1 or COL1A2 genes. Aside from the weakened bones, osteogenesis imperfecta also inflicts other symptoms and signs, such as short stature, breathing issues, dental problems, loosened joints, hearing loss, a blue tinge to the whites of the eyes, and aortic dissection. Currently, there is no cure for the disease and no FDA approved treatments. Avoiding smoking and exercising regularly can help reduce the risk of fractures. Other treatments may include physiotherapy, surgery, and biphosphonates, which can help reduce fractures, particularly in children. To learn more about osteogenesis imperfecta, click here.

About Rare Pediatric Disease Designation

Rare Pediatric Disease designation is given to experimental therapies that are being developed to treat serious rare conditions that primarily affect people age 18 or younger. A rare condition is defined as any that affects less than 200,000 people in the US. The recipient company of this designation is presented with an FDA Priority Review voucher if the drug’s Biologics License Application is approved by the agency. This voucher can be used for a future product or can be sold or traded to another company. 

About Setrusumab

Setrusumab is a monoclonal antibody that inhibits a protein called sclerostin, which is known to play a role in preventing the activity of cells responsible for the growth of bones. If approved, the medicine could be the first disease-altering therapy for osteogenesis imperfecta. The drug has earned Orphan Drug designations in the EU and US.