Update of BioMarin Clinical Development for Achondroplasia
BioMarin investigational medicine for achondroplasia, BMN-111, known as vosoritide, is currently in phase 3 of the medicine development process and this is an update provided by the company in late July 2019.
Over the last 5 years, more than 150 children with achondroplasia have enrolled in BioMarin clinical studies across eight countries. The safety and effectiveness of vosoritide are currently being investigated in ongoing clinical studies and has not been established. Vosoritide has not been approved for use outside of a clinical study by any country. At the conclusion of the clinical studies, BioMarin can apply for approval of vosoritide and authorities will evaluate the safety and effectiveness data and determine whether it can be made available in a given country.
Recent Publication
The New England Journal of Medicine (NEJM) published a peer-reviewed article based on the most recent results from the ongoing Phase 2 dose-finding and extension study. The article, “C-type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia,” discusses the 42-month findings. Some notes about this article in Beyond Achondroplasia post here. BMN 111-901:
Observational Study
A study that observes how a condition changes over time is often called an observational study or a noninterventional study. Participants do not receive investigational medicine. This study aims to provide a comparison for effects that may be seen in studies that include treatment with the investigational medicine vosoritide.
- Opened April 2012
- Participants from birth to age 18
- Aimed to enroll an equal number of boys and girls
- Measurements include growth and health-related quality of life (e.g. major illnesses and surgeries)
- Locations in Australia, Germany, Japan, Spain, Turkey, the United Kingdom, and the United States
BMN 111-202 and BMN 111-205:
Dose finding and Extension Studies
BioMarin has completed the dose evaluation study BMN 111-202 and is currently following all participants in a long term extension study called BMN 111-205.
- 35 participants, aged 5 to 14 years
- All participants are receiving the investigational medicine vosoritide
- Locations in Australia, France, the United Kingdom, and the United States
For more information on BMN 111-205, read here.
BMN 111–206 and BMN-208:
Phase 2 Infant and Toddler Studies
To evaluate the effect of vosoritide in children between the ages of 0 to 5 years.
- Approximately 70 children split into three groups
- 2 to 5 years age group has completed enrolment
- 6 months to 2 years age group continues the global enrolment
- 0 to 6 months age group is predicted to begin enrolment later this year
- The study is designed to assess the safety of vosoritide and effect on
- Growth – measured as annualized growth velocity (AVG) or rate of growth
- In addition, measurements include effects on
- Sleep apnea
- Requirement for surgeries
- Growth of the skeleton including
- Foramen magnum (in relation to the health of the spinal cord)
- Base of the skull (in relation to causes of sleep and ear health)
- Spine (in relation to back pain and spinal cord compression)
- Proportionality of the body segments and limbs
- Including effects on leg bowing, elbow extension, arm span
- Bone health
- Hip function
- Joint pain
- Health-related quality of life
- Health-related functional assessments as they relate to day to day activities
- For one year, half of the participants are on placebo (inactive medicine) and half are on the investigational medicine, allowing for the two groups to be compared
- After one year, all participants will receive the investigational medicine in a long term extension study called BMN 111-208
- The study is blinded which means the participants and the doctors do not know whether the participant received a placebo or not
- Participants must live in the country conducting the study
- Locations in Australia, Japan, the United Kingdom, and the United States
For more information on BMN 111-208, read here
BMN 111-301 and BMN 111-302:
Phase 3 Studies
Enrolment of this study is now complete. Top line results will be available at the end of 2019. Additional study results will be available in the beginning of 2020. Phase 3 studies can be the last stage of clinical development before manufacturers submit their data to authorities for evaluation of safety and efficacy.
- Participants aged between 5 to 18 years
- The study is designed to assess the safety of vosoritide and effect on
- Growth – measured as annualized growth velocity (AVG) or rate of growth
- The study is designed to assess the safety of vosoritide and effect on
- In addition, measurements include effects on
- Sleep quality
- Major illnesses
- Number of surgeries needed (for example ear tubes)
- Health-related quality of life
- Health-related functional assessments as they relate to day to day activities
- Proportionality of the body segments and limbs
- Including effects on leg bowing, elbow extension, arm span
- For one year, half of the participants are on placebo (inactive medicine) and half are on the investigational medicine, allowing for the two groups to be compared
- After one year, all participants will receive the investigational medicine in a long term extension study called BMN 111-302
- The study is blinded which means the participants and the doctors do not know whether the participant received a placebo or not
- Participants must live in the country conducting the study
- Locations in Australia, Germany, Japan, Spain, Turkey, the United Kingdom, and the United States.
For more information on BMN 111-302, read here.
BMN 111-501 and BMN 111-502:
Observational Studies on Lifetime Impact
Identifying health trends that occur from childhood through to adulthood may result in better care. The following studies aim to better understand what it is like to live with achondroplasia. Information will be collected from past medical visits (for example number and types of surgeries).
Lifetime Impact of Achondroplasia Study in Europe (LIAISE)
- Open to 300 participants ages 5 to 70 years
- Review of at least five years of past medical information
- Locations in Germany, Italy, Spain, and Sweden
- Denmark and Austria expected to open later this year
- Open to 175 participants ages 3 years and older
- Review of at least three years of past medical information
- Locations in Argentina, Colombia, and Brazil
For more information on LIAISE, read here.
For more information on LISA, read here.