FDA Approves First Drug to Treat Genetic Mutation Causing Dwarfism

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Courtesy of marosh (Flickr CC0)

Food and Drug Administration (FDA) recently approved Voxzogo, also known by the generic name vosoritide, as the first injectable drug to treat dwarfism (achondroplasia). The agency approved the drug for use in children ages five and up.

Theresa Kehoe, director of FDA’s Division of General Endocrinology, said that the approval fulfills an unmet medical need for more than 10,000 children in the United States.

The Voxzogo injection will be available in the U.S. in December. BioMarin Pharmaceutical is a U.S.-based company that has been developing and testing treatment for achondroplasia, according to the 2019 STAT News report. The list price for a year-long course of treatment is $320,000.

Achondroplasia is short-limbed dwarfism caused by the FGFR3 gene mutation involved in bone growth and development that leads to the development of abnormally-shaped bones. Voxzogo works by binding to the natriuretic peptide receptor-B and stimulating bone growth in children with achondroplasia to boost their height.

Courtesy of Jernej Furman (Flickr CC0)

The FDA approval of the Voxzogo drug was based on Phase III placebo-controlled that included 121 children between the ages of 5 and 18 whose growth plates are still developing. The participants received either one daily Voxzogo injection or a placebo shot, and the trial organizers assessed the participant’s height growth rate in one year.

By the end of the year, participants who received the injection grew an average of 1.57 centimeters (0.6 inches) compared with those who received a placebo shot.

STAT News reported that according to BioMarin Pharmaceutical, the data suggested that if participants have the treatment throughout their childhoods, those with dwarfism could reach similar heights as children without achondroplasia.

Some of the common side effects of the treatment are itchiness, redness on the injection site, decreased blood pressure, and vomiting, according to the FDA.

Voxzogo has drawn ethical questions within the Little People of America community that the clinical trials only focused on correcting dwarfism rather than long-term health.

Written by Janet Grace Ortigas
Edited by Cathy Milne-Ware


Live Science: 1st drug to treat genetic cause of dwarfism approved by FDA; by Nicoletta Lanese
Outsourcing-Pharma: 1st drug to treat genetic cause of dwarfism approved by FDA; by Jenni Spinner

Featured and Top Image Courtesy of marosh’s Flickr Page – Creative Commons License
Inset Image Courtesy of Jernej Furman’s Flickr Page – Creative Commons License

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