FGFR Aberrations May Be a Driver of Pediatric Skeletal Dysplasia

Achondroplasia is the most common form of dwarfism, affecting approximately 1 out of every 20,000 newborns, and treatment advances for children with this bone growth disorder have been limited.1,2 People with achondroplasia reach an adult height of 4 feet and 10 inches or shorter, and have normal intelligence and life expectancy. However, achondroplasia may cause serious, often debilitating health complications, such as spinal cord compression at the opening of the skull and lower spine, spinal deformity, chronic middle ear infections, and sleep apnea.1

One of the primary characteristics of achondroplasia is the failure of the long bones to fully develop. Shortened bones are a result of FGFR3 activating mutations that cause impaired bone development.1

In preclinical research, low doses of infigratinib have demonstrated a corrective effect on pathological hallmarks of achondroplasia.2

  • Based on this mouse model, infigratinib demonstrated greater than 2x improvement when compared to published data for previously tested interventions for achondroplasia.3,4 These data provide the basis for future clinical studies

FGFR=fibroblast growth factor receptor.

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References

  1. National Institutes of Health. Genetics Home Reference. Achondroplasia. https://ghr.nlm.nih.gov/condition/achondroplasia. Accessed December 3, 2018.
  2. Komla-Ebri D, Dambroise E, Kramer I, et al. Tyrosine kinase inhibitor NVP-BGJ398 functionally improves FGFR3-related dwarfism in mouse model. J Clin Invest. 2016;126(5):1871-1884.
  3. Rasmussen CE, Sprogøe K, Hersel U, et al. Pharmacokinetics and cardiovascular assessment of TransCon CNP, a sustained-release c-type natriuretic peptide prodrug, for the treatment of achondroplasia. Poster presented at: ASMBR 2017 Annual Meeting; September 8-11, 2017; Denver, CO.
  4. Lorget F, Kaci N, Peng J, et al. Evaluation of the therapeutic potential of a CNP analog in a Fgfr3 mouse model recapitulating achondroplasia. Am J Hum Genet2012;91(6):1108-1114.
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