(Reuters) -The U.S. Food and Drug Administration (FDA) on Friday approved the expanded use of BioMarin Pharmaceutical’s once-daily injection to treat children under the age of 5 with the most common form of short-limbed dwarfism.
Shares of the San Rafael, California-headquartered company rose 1.1% in aftermarket trading.
BioMarin’s drug, branded as Voxzogo, in 2021 was the first therapy to be greenlighted in the United States for treating the genetic disorder, known as achondroplasia, in children aged five and older under the FDA’s accelerated approval pathway.
The company is betting on Voxzogo and its recently approved gene therapy, Roctavian, for treating an inherited bleeding disorder to drive the company’s growth in the next few years.
Achondroplasia, a form of disproportionate short stature caused by a mutation in a gene that regulates bone growth, occurs in about one in every 25,000 newborns.
(Reporting by Pratik Jain and Khushi Mandowara in Bengaluru; Editing by Shailesh Kuber)
