Achondroplasia

• CLINICAL_TRIALS trial_result · 2026-07-14
  Clinical 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.

• CLINICAL_TRIALS trial_result · 2026-06-25
  Clinical 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;…

• CLINICAL_TRIALS trial_result · 2026-04-21
  Clinical 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…

• CLINICAL_TRIALS trial_result · 2026-04-03
  Clinical 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…

• CLINICAL_TRIALS trial_result · 2026-03-17
  Clinical 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 · 2026-03-13
  Clinical 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…

• CLINICAL_TRIALS trial_result · 2026-03-13
  Clinical 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.

• CLINICAL_TRIALS trial_result · 2026-01-15
  Clinical 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…

• CLINICAL_TRIALS trial_result · 2025-12-18
  Clinical 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.

• CLINICAL_TRIALS trial_result · 2025-10-22
  Clinical Trial Update: Study of Infigratinib in Children With Achondroplasia [Phase 2]

  This update notes that the Phase 2 trial of Infigratinib in children with achondroplasia has been completed.

• CLINICAL_TRIALS trial_result · 2025-02-25
  Clinical Trial Update: Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of SAR442501 in Pediatric Participants With Achondroplasia [Phase 2]

  Phase 2 study evaluating SAR442501 in pediatric achondroplasia assessing safety, tolerability, pharmacokinetics/pharmacodynamics, and effica…

• CLINICAL_TRIALS trial_result · 2024-10-17
  Clinical Trial Update: Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia.

  Pfizer-sponsored observational study on clinical and anthropometric features of children with Achondroplasia was terminated, with results po…

• CLINICAL_TRIALS trial_result · 2024-09-20
  Clinical Trial Update: A Study to Learn About Recifercept in Patients With Achondroplasia

  Pfizer completed a clinical trial evaluating Recifercept in achondroplasia. Results from the study were posted on ClinicalTrials.gov in Sept…

• CLINICAL_TRIALS trial_result · 2024-06-13
  Clinical Trial Update: A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia [Phase 2]

  Phase 2 trial results for BMN-111 (vosoritide) in infants and young children with achondroplasia have been completed and results posted.

• CLINICAL_TRIALS trial_result · 2024-03-07
  Clinical Trial Update: A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia [Phase 2]

  A Phase 2 dose-escalation study of once-weekly TransCon CNP in prepubertal children with achondroplasia has completed, and results have been…

• CLINICAL_TRIALS trial_result · 2024-02-15
  Clinical Trial Update: A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia [Phase 2]

  Phase 2 trial update evaluating safety, tolerability, and effectiveness of Recifercept in children with Achondroplasia; study terminated wit…

• CLINICAL_TRIALS trial_result · 2024-02-08
  Clinical Trial Update: Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia [Phase 2]

  A Phase 2 continuation study of Recifercept in achondroplasia has terminated; results have been posted.

• CLINICAL_TRIALS trial_result · 2023-11-02
  Clinical Trial Update: A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Multiple Subcutaneous Doses of TransCon CNP Administered Once Weekly in Children With Achondroplasia [Phase 2]

  A Phase 2 dose-escalation trial is evaluating safety, efficacy, and pharmacokinetics of weekly subcutaneous TransCon CNP in children with Ac…

• CLINICAL_TRIALS trial_result · 2023-02-28
  Clinical Trial Update: A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Achondroplasia [Phase 4]

  Phase 4 clinical trial evaluating recombinant human growth hormone in children with achondroplasia to assess efficacy and safety.

• CLINICAL_TRIALS trial_result · 2022-03-02
  Clinical Trial Update: A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia [Phase 3]

  Phase 3 trial evaluating BMN 111 in children with Achondroplasia has completed with results posted.

• CLINICAL_TRIALS trial_result · 2021-01-15
  Clinical Trial Update: A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia [Phase 2]

  Phase 2 trial evaluating BMN 111 in children with achondroplasia has completed with posted results.

• CLINICAL_TRIALS trial_result · 2017-05-05
  Clinical Trial Update: A Survey Collecting Data on Adult Height in Patients With Achondroplasia Treated With Somatropin

  A completed clinical trial update describing a survey to collect data on adult height in people with achondroplasia treated with somatropin.

• CLINICAL_TRIALS trial_result · 2015-04-14
  Clinical Trial Update: C-Type Natriuretic Peptide and Achondroplasia

  This update reports the completion of a clinical trial investigating C-type natriuretic peptide for achondroplasia and hypochondroplasia.

• CLINICAL_TRIALS trial_result · 2012-06-11
  Clinical Trial Update: A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers [Phase 1]

  Phase 1 trial evaluating safety and tolerability of BMN 111 (vosoritide) in healthy adult volunteers has completed; results summarize safety…

• CLINICAL_TRIALS trial_result · 2011-04-04
  Clinical Trial Update: Simultaneous Lengthening of Bilateral Femora and Tibiae in Achondroplastic Patients [NA]

  A clinical trial investigating simultaneous lengthening of the bilateral femora and tibiae in achondroplasia patients has completed.