Romosozumab arose from a genetic discovery that revealed the body’s own natural ability to increase bone strength. It is a treatment which aims to block the activity of the protein sclerostin. This diminishes bone breakdown and removal and stimulates bone formation, thereby increasing bone strength. The effectiveness and safety of romosozumab for the treatment of osteoporosis in men has been studied in a phase III clinical trial. The study showed that romosozumab given by injection monthly for a 12 month period significantly increased the formation of new bones, reducing the risk of a fracture. Romosozumab was also found to be safe with no significant adverse effects.
Vosoritide is in clinical development for the treatment of achondroplasia. Achondroplasia represents the most common form of short-limb dwarfism, a condition where the bones in the arms and legs do not form properly and are shorter than normal. Patients with achondroplasia have a short stature, an enlarged head with a prominent forehead, bowed legs, ear problems, respiratory issues, compression of the spinal cord, as well as short fingers, toes, lower legs and upper arms. No pharmacologic therapies have been approved for achondroplasia in the EU.