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Achondroplasia

Current Clinical Trials: Achondroplasia

Study: A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients with Achondroplasia

Indication: Achondroplasia

Investigational Therapeutic: No drug is administered

Study Type: Observational

Goal: Collection of consistent baseline growth measurements on pediatric patients with Achondroplasia being considered for subsequent enrollment in Study 111-301.

Status: This study is currently recruiting participants.

Additional information: View on Clinicaltrials.gov

Study: A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children with Achondroplasia

Indication: Achondroplasia

Investigational Therapeutic: BMN 111 (Analog of CNP for Achondroplasia)

Study Type: Phase 3

Goal: To evaluate the efficacy, safety, and tolerability of BMN 111
administered in children with achondroplasia.

Status: This study is enrolling participants by invitation only.

Additional information: View on Clinicaltrials.gov

Study: A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia (ACH)

Indication: Achondroplasia

Investigational Therapeutic: BMN111 (Analog of CNP for Achondroplasia)

Study Type: Phase 2

Goal: To assess the safety and tolerability of daily BMN 111 administered to children with achondroplasia.

Status: This study is ongoing, but not recruiting participants.

Additional information: View on Clinicaltrials.gov

Study: A Phase 2 Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children With Achondroplasia

Indication: Achondroplasia

Study Type: Phase 2

Goal: This is a Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia. The primary objective is to evaluate the long-term safety and tolerability of daily SC injections of BMN 111 in children with ACH who have completed two years of treatment in the 111-202 study.

Status: This study is enrolling participants by invitation only.

Additional information: View on Clinicaltrials.gov

Completed Clinical Trials: Achondroplasia

Study: Phase 1, two-part, double-blind, placebo-controlled study to evaluate safety, tolerability, and pharmacokinetics of single and multiple doses of BMN 111 administered to healthy adult volunteers.

Indication: Achondroplasia

Investigational Therapeutic: BMN 111

Study Type: Phase 1

Goal: The purpose of this study is to measure how much of the study drug gets into the blood- stream and how long it takes the body to get rid of it when given as a single dose. Information about any side effects that may occur will also be collected.

Status: This study has been completed.

Additional information: View on Clinicaltrials.gov

Roadmap to a New Therapy

Roadmap to a new therapy

Click here to open the full-size pdf.

Clinical Trial

A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients with Achondroplasia
Read more »

Clinical Trial

A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children with Achondroplasia
Read more »

Clinical Trial

A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia (ACH)
Read more »

Clinical Trial

Phase 1, two-part, double-blind, placebo-controlled study to evaluate safety, tolerability, and pharmacokinetics of single and multiple doses of BMN 111 administered to healthy adult volunteers.
Read more »

Clinical Trial

A Phase 2 Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children With Achondroplasia.
Read more »

BioMarin

Diseases

Lysosomal Storage Disorders (MPS I, MPS IVA, MPS VI, CLN2 disease) PKU, LEMS

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