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The FDA (Food and Drug Administration) approved the first drug to treat dwarfism genetic condition —known as achondroplasia which encodes instructions to build a protein involved in bone growth and development, stated by the Genetic and Rare Diseases information center. The new drug stops the gene from going into overdrive which slows down the growth process and cartilage tissues issues.
This condition is caused by mutations in the FGFR3 gene. It creates the development of short-shaped bones causing people with dwarfism to be short in stature. Dwarfism can also give rise to numerous complications that would require surgery to fix. The FDA stated the most common side effects of this drug were redness, itchiness, vomiting, and decreased blood pressure.
The pharmaceutical company has been developing and testing this drug since 2019. They finally got it approved to treat people ages 5 and up with achondroplasia.
FDA’s Data Shows
Clinical trials have shown that the drug Voxzogo is safe and has an effect on restoring bone growth in children with achondroplasia and boosting their heights. It is unclear whether or not this drug counteracts the multiple health issues that can arise from having dwarfism. Voxzogo also works by binding to a receptor that is found in the bone and cartilage tissues.
BioMarin Pharmaceutical stated that once attached to the receptor, the medication sets off chains of reactions that stimulate bone growth, causing it to override the effects of the FGFR3 mutations. In a most recent trial, Voxzogo included 121 participants aged between five and 18, whose growth plates were still open, which meant they have not finished growing.
The different groups of ages were randomly assigned to receive one daily Voxzogo injection or a placebo shot. As the trial went on the organization assessed all the children’s rate of height growth a whole year.
The price for the year-long treatment of this drug cost would round up to 320,000 inside the dwarfism community, Voxzogo has drawn mixed reactions some have shown concern that trials had more concern on their heights than their actual health. Others expressed the hope the treatment will help improve other abilities helping their quality of life.
Written by Travon Smith
Edited by Sheena Robertson
Source:
Live Science: 1st drug to treat genetic cause of dwarfism approved by FDA; by Nicoletta Lanese
Top and Featured Image by Stuart Isett/Fortune Courtesy Of Fortune Brainstorm Health Flickr Page – Creative Commons License
Inset Image Courtesy Of Jernej Furman Flickr Page – Creative Commons License