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

8 Comments

  1. I want know when be this approved and start working before the chideren reach age 18

    • Thank you for you message.
      About orphan drugs (like BMN-111), no one can say exactly when it will be available. Firstly, this drug (like other orphan drugs) needs to show positive and effective results on phase 3 in order no be approved by EMA and FDA (the medicines regulators) and all this processes take time. And if BMN-111 is approved by EMA and FDA, the drug can enter the medicines market, the company BioMarin needs to discuss prices and reimbursement with the European member states(which country are you from) and only when member states agree with the reimbursement (which can differ very much in cost and time processes from country to country), the drug will be sent to central hospitals and children with ACH diagnosis and with the prescription of a doctor, can pick monthly doses (most probably will be like this) in the hospital and will administer it at home or health clinical center by a daily injection.
      Overall, taking in account that many clinical centers involved in the trial will start the 52 weeks trial in early 2018 for phase 3, results will be evaluated and disclosed in 2019 and then there are the processes of drug approval (market access) and reimbursement approval by member states, which can take between a couple of months to 1-2 years. This is a long process and take in account that no clinical trial is a statement that a drug will be approved. Just 1 in each 3 drugs in phase 3 is approved.

    • Thank you for your message.
      About orphan drugs (like BMN-111), no one can say exactly when it will be available. Firstly, this drug (like other orphan drugs) needs to show positive and effective results on phase 3 in order no be approved by EMA and FDA (the medicines regulators) and all these processes take time. And if BMN-111 is approved by EMA and FDA, the drug can enter the medicines market, the company BioMarin needs to discuss prices and reimbursement with the European member states(which country are you from) and only when member states agree with the reimbursement (which can differ very much in cost and time processes from country to country), the drug will be sent to central hospitals and children with ACH diagnosis and with the prescription of a doctor, can pick monthly doses (most probably will be like this) in the hospital and will administer it at home or health clinical center by a daily injection.
      Overall, taking in account that many clinical centers involved in the trial will start the 52 weeks trial in early 2018 for phase 3, results will be evaluated and disclosed in 2019 and then there are the processes of drug approval (market access) and reimbursement approval by member states, which can take a couple of months to 1-2 years. This is a long process and please take into account that no clinical trial is a statement that a drug will be approved. Just 1 in every 3 drugs in phase 3 is approved.

  2. I would like my son to join this study

    • Before getting access to phase 3 study, all children must participate previously for at least 6 months in the 111-901 study (the natural history study) and then will be selected among all participants in the 111-901 to get in the 111-301 trial. You have here the list of clinical centers that are enrolling children for this study (at the bottom).

  3. I want to enroll my son in clinic trail.

  4. Why is hypochondroplasia excluded from this study? Don’t you expect it to work for these children too? Will there be any way for doctors to prescribe off-label for hypochondroplasia?

  5. I want enroll for my daughter

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