Voxzogo Becomes First-in-Class Treatment for Dwarfism
November 19, 2021 – The U.S. FDA has approved VoxzogoTM (vosoritide) for injection to increase linear growth in pediatric patients at least five years of age who have achondroplasia and open epiphyses (growth plates).
- Achondroplasia is a genetic disorder that affects bone growth and is the most common form of dwarfism. It can lead to serious complications such as compression of the spine, abnormal spine curvature, recurrent ear infections, interruptions of breathing, and fluid accumulation in the skull.
- Voxzogo is the first drug approved by the FDA specifically for the treatment of achondroplasia. It IS also the first FDA-approved drug belonging to the C-type natriuretic peptide (CNP) analog therapeutic class.
- Recommended dosing is a once-daily subcutaneous injection with the dosage strength ranging from 0.24mg to 0.8mg, based on the patient’s body weight. Voxzogo can be given by a healthcare provider or by a caregiver.
- Patients should drink approximately 240-300mL of fluid in the hour prior to administration and have adequate food intake to reduce the risk of low blood pressure as a side effect of treatment.The patient’s body weight, growth, and physical development must be monitored every three to six months. Voxzogo should be discontinued permanently once the patient’s growth plates have closed, as this signals there is no further growth potential.
- The patient’s’body weight, growth, and physical development must be monitored every three to six months. Voxzogo should be discontinued permanently once the patient’s growth plates have closed, as this signals there is no further growth potential.
- BioMarin, the manufacturer, has launched Voxzogo at a wholesale acquisition cost (WAC) of $8,990 per vial.
