BioMarin Pharmaceutical scored a first on Friday and provided an option for patients with a rare disease for which there is little recourse. The U.S. Food and Drug Administration approved Voxzogo (vosoritide) to improve growth in children five years of age and older with achondroplasia, a rare genetic disorder that causes the most common form of dwarfism.

The drug, a once-daily injection, has been approved specifically for children who have open epiphyses, or growth plates, which gives them the potential for growth. For those children with achondroplasia, a mutation prevents the growth of that bone. Voxzogo, a C-type natriuretic peptide (CNP) analog, corrects that by acting as a positive regulator of the signaling pathway downstream of the fibroblast growth factor receptor 3 gene. With the FDA nod, Voxzogo becomes the first drug approved in the U.S to treat the condition. Prior to the approval, there were some drastic surgical options available to patients, but no therapeutics.

Achondroplasia is a genetic condition that causes severely short stature and disproportionate growth. The average height of an adult with achondroplasia is approximately four feet.

Voxzogo was greenlit under accelerated approval based on clinical data demonstrating an improvement in annualized growth velocity (AGV). Under the framework of accelerated approval, California-based BioMarin will be required to continue to provide follow-up data to the FDA. The company said it intends to use ongoing open-label extension studies compared to available natural history to fulfill the requirement.

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