Achondroplasia
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EMA approval · 2026-08-13EMA Authorised: Voxzogo (vosoritide) — BioMarin International Limited
Voxzogo (vosoritide) has been authorised in the EU for achondroplasia by BioMarin International Limited.
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CLINICAL_TRIALS trial_result · 2026-07-14Clinical Trial Results: A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With Achondroplasia [Phase 2, Phase 3]
Clinical trial evaluating TransCon CNP versus placebo in children with achondroplasia has completed Phase 2/3 with posted results.
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CLINICAL_TRIALS trial_result · 2026-06-25Clinical Trial Results: Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of SAR442501 in Pediatric Participants With Achondroplasia [Phase 2]
Phase 2 trial results for SAR442501 in pediatric achondroplasia (osteochondrodysplasia) assessing safety, tolerability, PK/PD, and efficacy;…
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EMA approval · 2026-06-11EMA Authorised: Voxzogo (vosoritide) — BioMarin International Limited
EMA authorises Voxzogo (vosoritide) for achondroplasia, granted orphan medicine status to BioMarin International Limited.
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: A Study of TYRA-300 in Children With Achondroplasia: BEACH301 [Phase 2]
A Phase 2 study of TYRA-300 in children with achondroplasia (BEACH301) is currently recruiting.
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CLINICAL_TRIALS research · 2026-04-22Clinical Trial Update: Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia [Phase 2, Phase 3]
A Phase 2/3 trial is evaluating BMN 333 versus Vosoritide to assess efficacy and safety in children with achondroplasia; the study is curren…
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CLINICAL_TRIALS trial_result · 2026-04-21Clinical Trial Update: A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia. [Phase 2]
A phase 2 clinical trial to evaluate the efficacy and safety of Navepegritide in adolescents (12-18) with Achondroplasia is currently recrui…
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CLINICAL_TRIALS research · 2026-04-08Clinical Trial Update: A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia [Phase 2]
A Phase 2 trial in infants (0 to <2 years) with achondroplasia evaluating the efficacy and safety of TransCon CNP vs placebo; recruiting sta…
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CLINICAL_TRIALS trial_result · 2026-04-03Clinical Trial Update: Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)
This clinical trial update notes that the prospective clinical assessment study in children with achondroplasia sponsored by QED Therapeutic…
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CLINICAL_TRIALS research · 2026-04-01Clinical Trial Update: Achondroplasia Natural History Multicenter Clinical Study
This is an ongoing multicenter natural history study of achondroplasia. Participant recruitment is currently underway.
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CLINICAL_TRIALS research · 2026-04-01Clinical Trial Update: Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH) [Phase 2]
Phase 2 interventional study evaluating infigratinib for achondroplasia in children under 3 years old; currently recruiting.
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CLINICAL_TRIALS trial_result · 2026-03-17Clinical Trial Update: A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia [Phase 3]
A Phase 3 study of Infigratinib in children and adolescents with achondroplasia has completed.
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CLINICAL_TRIALS trial_result · 2026-03-13Clinical Trial Update: An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia [Phase 2]
An extension Phase 2 trial is evaluating the safety and efficacy of BMN 111 (vosoritide) in children with Achondroplasia; the study is curre…
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CLINICAL_TRIALS trial_result · 2026-03-13Clinical Trial Update: An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia [Phase 3]
An extension study (Phase 3) is being conducted to evaluate the efficacy and safety of Vosoritide (BMN 111) in children with Achondroplasia.
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CLINICAL_TRIALS research · 2026-03-13Clinical Trial Update: A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia [Phase 2]
The trial to evaluate the safety of vosoritide in at-risk infants with achondroplasia remains active but not recruiting.
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CLINICAL_TRIALS research · 2026-03-13Clinical Trial Update: A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH) [Phase 2]
Phase 2 trial update for BMN 111 (vosoritide) evaluating long-term safety, tolerability, and efficacy in children with achondroplasia. The s…
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CLINICAL_TRIALS research · 2026-02-20Clinical Trial Update: Prospective Longitudinal Monocentric Study to Measure Limb Movement in Patients With FGFR3-related Skeletal Dysplasia
Prospective, monocentric study sponsored by SYSNAV to measure limb movement in patients with FGFR3-related skeletal dysplasia. Recruitment i…
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CLINICAL_TRIALS research · 2026-02-18Clinical Trial Update: A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combination With Lonapegsomatropin in Children With Achondroplasia [Phase 2]
Phase 2 clinical trial evaluating navepegritide in combination with lonapegsomatropin for children with achondroplasia; current status is ac…
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CLINICAL_TRIALS trial_result · 2026-01-15Clinical Trial Update: A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With Achondroplasia [Phase 2, Phase 3]
A Phase 2/3 clinical trial evaluating the efficacy and safety of TransCon CNP compared with placebo in children with achondroplasia has been…
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CLINICAL_TRIALS research · 2025-12-24Clinical Trial Update: A Study in Children With Achondroplasia
Abbisko Therapeutics is recruiting for a pediatric study in children with achondroplasia.
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CLINICAL_TRIALS other · 2025-12-22Clinical Trial Update: A Study of ABSK061 to Assess Safety, Tolerability, Pharmacokinetics, and Efficacy in Children With Achondroplasia [Phase 1, Phase 2]
A Phase 1/Phase 2 trial of ABSK061 in children with achondroplasia is reported, and recruitment has not yet started.
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CLINICAL_TRIALS trial_result · 2025-12-18Clinical Trial Update: VIrtual STudy in Achondroplasia for the US (VISTA)
BioMarin reports a US-based virtual clinical trial called VISTA for achondroplasia that is currently recruiting.
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EMA approval · 2025-11-18EMA Authorised: Voxzogo (vosoritide) — BioMarin International Limited
The European Medicines Agency authorised Voxzogo (vosoritide) for the treatment of achondroplasia. The Marketing Authorisation Holder is Bio…
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CLINICAL_TRIALS research · 2025-10-31Clinical Trial Update: Extension Study of Infigratinib in Children With Achondroplasia (ACH) [Phase 2]
An extension study of Infigratinib for children with achondroplasia is in Phase 2 and currently enrolling by invitation. The study is sponso…
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CLINICAL_TRIALS research · 2025-10-27Clinical Trial Update: A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia [Phase 2, Phase 3]
This entry notes an ongoing Phase 2/Phase 3 study of TransCon CNP for Achondroplasia in children and adolescents. It is assessing long-term …