Edasalonexent is currently in clinical development for the treatment of male paediatric patients with Duchenne muscular dystrophy (DMD). DMD is a rare genetic condition caused by a mistake or abnormality in a gene called dystrophin, located on the X chromosome (one of the two sex chromosomes). Chromosomes are tiny structures inside cells made from deoxyribonucleic acid (DNA) and proteins. Males have one X chromosome, while females have two X chromosomes. As males have one X chromosome, DMD is much more common in males. DMD is fatal condition with no cure. It causes progressive muscle weakness and often leads to loss of walking ability by the age of twelve, as well as problems with the heart and lungs. Current treatment options only target a particular mistake or abnormality and focus on slowing progression of the disease.
Edasalonexent is administered orally. It works by reducing the NF-κB activity, a protein that causes inflammation leading to muscle damage and prevention of muscle regeneration seen in patients with DMD. Edasalonexent is expected to reduce the muscle damage seen in DMD and enable muscle regeneration. If licensed, edasalonexent will provide a treatment option for male paediatric patients with DMD.
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.