The FDA has granted accelerated approval for YUVIWEL (navepegritide), the first once-weekly treatment for children with achondroplasia. Commercial availability is expected in early Q2 2026, with a Rare Pediatric Disease Priority Review Voucher granted.
BridgeBio Pharma reported positive Phase 3 results for oral infigratinib in children with achondroplasia, meeting primary and secondary endpoints with superior growth velocity and the first statistically significant improvement in body proportionality. The company plans regulatory submissions in the second half of 2026.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT06002373 |
Assessment of Artificial Intelligence for Treatment Decision Recommendation of Adult Skeletal Class III Patients |
UNKNOWN | |
| NCT05991609 |
Extreme Morphology and Metabolic Health |
ACTIVE_NOT_RECRUITING | |
| NCT05876416 |
Decoding the Genetic Landscape of Skeletal Diseases |
RECRUITING | |
| NCT05247645 |
Data Collection of Patients With Rare Bone Diseases |
RECRUITING | |
| NCT03548779 |
North Carolina Genomic Evaluation by Next-generation Exome Sequencing, 2 |
COMPLETED | NA |
| NCT02762318 |
Identification and Characterization of Bone-related Genetic Variants in Families |
TERMINATED |
