Research into hypochondroplasia and related FGFR3 skeletal dysplasias is moving quickly. There is currently no approved drug that targets the underlying cause of hypochondroplasia. However, several companies and research groups are studying treatments that aim to improve growth, body proportions, function, mobility, or quality of life. Some treatments are already approved for achondroplasia but not hypochondroplasia.
Others remain investigational and are being studied in clinical trials. This page is intended to help families understand the current research landscape. It does not recommend or endorse any specific treatment, company, or clinical trial. Families considering participation in research should speak with their healthcare team and carefully review the purpose, eligibility criteria, risks, benefits, and practical commitments of any study.
List of Current HCH Studies
Clinical trial information can change frequently. The table below shows hypochondroplasia studies registered on ClinicalTrials.gov, including their current recruitment status and study locations.
View all registered hypochondroplasia studies in a table
Families should always check the official study record for the latest information. A study may be recruiting overall but may not yet be open at every location.
In September 2026, full results from the Phase 3 CANOPY-HCH-3 study were published in NEJM Evidence and presented at the European Society for Paediatric Endocrinology Annual Meeting.
After 52 weeks, children receiving vosoritide had an average annualised growth velocity 2.33 cm per year greater than children receiving placebo. The study also reported improvements in standing height, height Z-score and arm span. The overall safety findings were consistent with the established safety profile of vosoritide in achondroplasia. Longer-term follow-up is continuing.
BioMarin has submitted an application to the US Food and Drug Administration to expand the approval of vosoritide to include children with hypochondroplasia. The company has also stated that submissions to the European Medicines Agency and other regional health authorities are progressing.
Vosoritide is not currently approved for hypochondroplasia. Regulatory authorities will review the evidence before deciding whether it should be approved, for which children and under what conditions.
Read BioMarin’s September 2026 announcement
ACCEL is a global observational study for children and adolescents with hypochondroplasia. No study medication is given during this study.
The study is collecting information about growth, body proportions, physical function, cognitive development, quality of life, medical history and health concerns associated with hypochondroplasia.
Potential participants must be aged 2.5 to under 17 years when entering the study and have a diagnosis of hypochondroplasia confirmed through genetic testing. Other eligibility criteria also apply.
Participants must complete at least six months in ACCEL before they may be considered for ACCEL 2/3, the interventional study of infigratinib.
ACCEL study record: NCT06410976
BridgeBio information about the ACCEL programme
ACCEL 2/3 is an international Phase 2/3 study evaluating infigratinib in children and adolescents with hypochondroplasia. Infigratinib is an investigational oral medicine designed to reduce overactive FGFR3 signalling.
This study is listed as enrolling by invitation. Children must first participate in the ACCEL observational study for at least six months and meet the additional eligibility requirements before they may be considered for ACCEL 2/3.
View the ACCEL 2/3 study record: NCT06873035
This Phase 2 study is evaluating the safety and efficacy of daily vosoritide compared with placebo in infants and young children with hypochondroplasia.
The study is recruiting children aged 0 to under 36 months. Eligibility requirements and recruitment status vary by study location.
View the study record NCT07126262
This observational study is collecting information about growth and the clinical course of hypochondroplasia. Participants do not receive an investigational treatment as part of the study.
View the study record NCT06212947
This Phase 3 extension study is evaluating the long-term safety and efficacy of daily vosoritide in children with hypochondroplasia who participated in an eligible earlier study.
The study is enrolling by invitation and is not open to children who did not participate in one of the qualifying studies.
View the study record NCT07073014
This Phase 3 study evaluated daily vosoritide in children with hypochondroplasia aged 3 to 17 years. It is active but no longer recruiting new participants.
View the study record NCT06455059
Some therapies aim to increase activity in the C-type natriuretic peptide, or CNP, pathway. This pathway can help counterbalance overactive FGFR3 signalling, which is involved in hypochondroplasia and achondroplasia.
Vosoritide. A daily injection approved for achondroplasia in some countries and studied in children with hypochondroplasia.
Navepegritide, previously called TransCon CNP,. A once-weekly CNP therapy approved in the United States for certain children with achondroplasia, but not approved for hypochondroplasia.
Other investigational treatments aim to act more directly on FGFR signalling.
Infigratinib. An oral FGFR inhibitor currently being studied in children with hypochondroplasia.
Dabogratinib, formerly called TYRA-300. An oral FGFR3-selective inhibitor being studied clinically in achondroplasia. Research in models of hypochondroplasia has also been published.
These programmes are at different stages of research. A treatment being studied in achondroplasia, laboratory models or another skeletal dysplasia should not be assumed to be safe or effective for people with hypochondroplasia.
Some investigational treatments have been discontinued. These programmes remain part of the research history, but they are not current treatment options or actively enrolling hypochondroplasia studies.
Recifercept. Previously developed by Pfizer for achondroplasia.
SAR-442501. Previously developed by Sanofi for achondroplasia and related skeletal dysplasias.
The following treatments are not currently being studied in clinical trials specifically for hypochondroplasia. They are included because they target pathways involved in bone growth and may be relevant to the wider skeletal dysplasia research landscape.
Abbisko Therapeutics is developing ABSK061, an investigational oral FGFR2/3 inhibitor for skeletal dysplasias. The programme is currently focused on achondroplasia and is not an active hypochondroplasia clinical trial.
Learn more about the ABSK061 programme
RIBOMIC is developing umedaptanib pegol, also known as RBM-007, for achondroplasia. It is an investigational therapy designed to inhibit fibroblast growth factor 2, or FGF2, which is involved in the regulation of bone growth. It works through a different pathway from treatments that target FGFR3 directly.
RBM-007 is not currently being studied in a clinical trial for hypochondroplasia.
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