On March 11, the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease (RPD) designation to ABSK061, a drug developed by Abbisko Therapeutics for the treatment of achondroplasia. Achondroplasia is a rare genetic condition that affects bone growth and is associated with mutations in the FGFR3 gene.

Recently in 2026, the U.S. Food and Drug Administration (FDA) approved Navepegritide, marketed as Yuviwe by Ascendis Pharma, for the treatment of children with achondroplasia aged 2 years and older who still have open growth plates. Achondroplasia is a genetic condition that affects bone growth and is associated with a range of lifelong medical complications.

QED Therapeutics (an affiliate of BridgeBio) has announced the results of a clinical trial group from the PROPEL 2 Study, a Phase 2 trial of infigratinib for children with achondroplasia.

BioMarin presented results of annualized growth velocity in children with achondroplasia who started treatment during adolescence with VOXZOGO. 

What do you think about Limb lengthening in achondroplasia? If you have not considered it, not done it, or you have, please share your opinion.

The investigational drug TYRA-300 demonstrated significant increases in growth and bone length in a mouse model of FGFR3-Related Skeletal Dysplasia on recent study and is now closer to a Phase Clinical Trial.