Beyond Achondroplasia

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Update on BioMarin´s Achondroplasia Clinical Development Program

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BioMarin Pharmaceutical sent to patient organizations an update of the clinical development program for BMN-111. The current status of the achondroplasia clinical programme is described in the document below.
BioMarin’s investigational therapy for achondroplasia, BMN 111, is currently under investigation and has not been approved for use in any country.

Study 111-301

This trial is a phase 3 placebo-controlled trial with BMN-111, includes children from 5 to 17 years of age and approximately 110 participants globally. In this trial, participants are randomly selected to be included in the placebo group or in the drug group.

Outcomes of study 111-301

The primary outcome of the trial is to evaluate a change in the rate of growth or change in height. Secondary outcomes include measurements of health through evaluating health-related quality of life scores, other
associated symptoms, sleep quality as well as major illnesses and surgeries.

Duration

This trial lasts for 52 weeks and participants will have to have to complete a minimum of 6 months in the
observational trial (111-901) before they can be selected for the Phase 3 (111-301) trial.
Participants on placebo can receive the investigational therapy, or the BMN-111, after the 1 year trial period is
complete.

Study 111-501

BioMarin is preparing a new study, the 111-501, on the Lifetime Impact of Achondroplasia Study in Europe (LIAISE) is an observational study (so, no drug will be included) looking at the impact on quality of life, healthcare resource use, clinical, socioeconomic and psychosocial state of individuals living with achondroplasia.

This study is recruiting up to 300 participants between 5 and 70 years of age and will be opening in the following countries between now and early 2018: Germany, Spain, Italy, Sweden, and Denmark. Participation in the

study will include a 5-year review of historical clinical data as well as data obtained using questionnaires.

Read the full document here:

BioMarin ACH clinical development program Nov 2017

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