Hypochondroplasia is a genetic skeletal dysplasia characterized by disproportionate short stature, short limbs, and other mild-to-moderate features overlapping with achondroplasia. Clinical severity is variable, and diagnosis may involve genetic testing, especially of FGFR3. Many affected individuals have normal life expectancy with supportive management.
BioMarin reported first-quarter 2026 revenue of $766 million and raised full-year guidance to $3.825 billion-$3.925 billion. The company highlighted FDA approval of PALYNZIQ for adolescents and upcoming Phase 3 readouts for BMN 401 and VOXZOGO.
The FDA approved navepegritide, a once-weekly injectable therapy for achondroplasia, while phase 3 data showed infigratinib, an investigational oral medication, achieved the highest growth velocity reported in randomized trials for the condition.
BridgeBio Pharma announced positive Phase 3 topline results for infigratinib in children with achondroplasia, meeting primary and key secondary endpoints. The company plans regulatory submissions in the U.S. and Europe in the second half of 2026.
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.
BridgeBio Pharma announced positive Phase 3 topline results for infigratinib in children with achondroplasia, meeting primary and key secondary endpoints. The company plans regulatory submissions in the U.S. and Europe in the second half of 2026.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07393373 |
Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia |
ENROLLING_BY_INVITATION | PHASE2 |
| NCT07388966 |
Prospective Longitudinal Monocentric Study to Measure Limb Movement in Patients With FGFR3-related Skeletal Dysplasia |
RECRUITING | |
| NCT07126262 |
A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months |
RECRUITING | PHASE2 |
| NCT07073014 |
Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia |
ENROLLING_BY_INVITATION | PHASE3 |
| NCT06873035 |
An Interventional Study of Infigratinib in Children With Hypochondroplasia |
ENROLLING_BY_INVITATION | PHASE2/PHASE3 |
| NCT06455059 |
Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia |
ACTIVE_NOT_RECRUITING | PHASE3 |
| NCT06410976 |
Prospective Clinical Assessment Study in Children With Hypochondroplasia |
RECRUITING | |
| NCT06212947 |
A Multicenter Multinational Observational Study of Children With Hypochondroplasia |
RECRUITING | |
| NCT05328050 |
Registry for Patients With Achondroplasia / Hypochondroplasia (OMPR-Ach/Hy) |
RECRUITING | |
| NCT01541306 |
C-Type Natriuretic Peptide and Achondroplasia |
COMPLETED |
