Achondroplasia is a form of short-limbed dwarfism. Achondroplasia is characterized by an average-sized trunk, short arms and legs, notably short upper arms and thighs, a limited range of motion at the elbows, and an enlarged head. Spinal stenosis, a narrowing of the spinal canal that can compress the upper section of the spinal cord, is a potentially serious complication of achondroplasia. Spinal stenosis is characterized by leg pain, tingling, and weakness, which can make walking difficult. Achondroplasia is caused by mutations in the FGFR3 gene. The FGFR3 gene is responsible for giving instructions when making a protein that is involved in the development and maintenance of bone and brain tissue.
Market Dynamics
Increasing approvals of achondroplasia treatment drugs by regulatory bodies are expected to drive the market growth over the forecast period. For instance, in November 2021, BioMarin, a global biotechnology company, announced that the U.S. Food and Drug Administration (FDA) had granted accelerated approval to VOXZOGO (vosoritide) for injection, indicated to increase linear growth in pediatric patients with achondroplasia of five years of age and older with open epiphyses (growth plates). Moreover, in August 2021, QED Therapeutics, a subsidiary of BridgeBio Pharma, Inc., announced that the European Commission (EC) had granted marketing authorization for VOXZOGO (vosoritide), a once-daily injection to treat achondroplasia in children from the age of 2 until growth plates are closed, which occurs after puberty when children reach final adult height.
Also, increasing research and development by key players for the treatment of achondroplasia is expected to drive the treatment segment growth over the forecast period. For instance, in November 2021, RIBOMIC, Inc., a clinical stage pharmaceutical company specialized in aptamer therapeutics, announced the results from its Phase 1, healthy volunteer clinical study using RBM-007 for the planned treatment of achondroplasia, which was completed in May 2021.
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