BioMarin to Present Data Highlighting Significant Impact of VOXZOGO® (vosoritide) in Bone Health and Health-Related Quality of Life in Achondroplasia at 2024 International Conference on Children's Bone Health
New Investigator-Led Study Shows VOXZOGO Significantly Increased Bone Length While Maintaining Bone Strength Through 5 Years of Observation in Children with Achondroplasia
Phase 2 and 3 Data on VOXZOGO Demonstrate Safety, Efficacy and Impact on Proportionality and Health-Related Quality of Life for Children with Achondroplasia
New Research Underscores Unmet Medical Needs of People with Hypochondroplasia
"With thousands of children treated since approval, the breadth and depth of data collected from our trials demonstrate VOXZOGO's safety and benefit in children of all ages, including those under 5 who have received VOXZOGO for up to four years," said
Significant Impact on Bone Length, Strength, Proportionality and Health-Related Quality of Life in Achondroplasia
Results from an investigator-led analysis of
"In order for children with achondroplasia to experience meaningful changes in daily functioning following treatment to enable growth, it is critical that bone robustness is maintained to preserve strength," said
Additional data to be presented at ICCBH, previously shared at the 2024
Data Underscore Unmet Needs in Hypochondroplasia
Data from a retrospective, real-world matched cohort study using electronic medical records from
VOXZOGO Clinical Development Program Milestones
Building on its leadership in achondroplasia,
A multinational observational study in children with hypochondroplasia (111-902) is currently recruiting participants, and the first patient is on track to be dosed in the treatment phase (Phase 3 trial) in June with enrollment completion expected in early 2025.
The company's observational study in children with ISS (111-903) has begun enrolling patients, and the Phase 2 treatment arm will also begin enrollment later this year (111-210). Additionally, a study in multiple other genetic short stature conditions, including Turner syndrome, SHOX deficiency, and Noonan syndrome, is anticipated to begin enrollment later this year.
Key presentations at ICCBH are listed below, with all times in Central European
Oral Presentations
Persistent Growth-Promoting Effects of Vosoritide in Children with Achondroplasia is Accompanied by Improvement in Physical Aspects of Quality of Life
Oral #OC4.3
A Promising Adjuvant Treatment for Growing Mice with Moderate-to-Severe Osteogenesis Imperfecta: A Pilot Study of a CNP Analog
Oral #OC5.3
Poster Presentations
Does Vosoritide Treatment Affect Bone Strength in Children with Achondroplasia?
Poster #P151
Persistence of Growth Promoting Effects in Infants and Toddlers with Achondroplasia: Results from a Phase 2 Extension Study with Vosoritide
Poster #P79
Design and Objectives of the Acorn Study: A Non-Interventional Study Evaluating Long-Term Safety in Achondroplasia Children Treated with Vosoritide
Poster #P91
Persistent Growth-Promoting Effects of Vosoritide in Children with Achondroplasia for up to 4 Years: Update from Phase 3 Extension Study
Poster #P78
Comorbidity and Mortality Burden Among Patients with Hypochondroplasia in
Poster #P176
About VOXZOGO
In children with achondroplasia, endochondral bone growth, an essential process by which bone tissue is created, is negatively regulated due to a gain of function mutation in FGFR3. VOXZOGO, a C-type natriuretic peptide (CNP) analog, acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth.
VOXZOGO is approved in the
To date, more than 3,000 people with achondroplasia around the world have received VOXZOGO. In total, VOXZOGO is available in 43 markets worldwide.
Patient Support Accessing VOXZOGO
To reach a BioMarin RareConnections® Case Manager, please call, toll-free, 1-833-VOXZOGO (1-833-869-9646) or e-mail VOXZOGOSupport@biomarin-rareconnections.com. For more information about VOXZOGO, please visit www.voxzogo.com. For additional information regarding this product, please contact BioMarin Medical Information at medinfo@bmrn.com.
About Achondroplasia
Achondroplasia is a rare genetic growth-related condition caused by a variation in the FGFR3 gene. It is characterized by disproportionate short stature and a potentially high burden of complications related to impaired endochondral bone growth.
Approximately 80% of children with achondroplasia are born to parents of average stature as a result of a spontaneous variation in the FGFR3 gene. The worldwide incidence of achondroplasia is around one in 25,000 live births.
VOXZOGO
What is VOXZOGO used for?
- VOXZOGO is a prescription medicine used to increase linear growth in children with achondroplasia and open growth plates (epiphyses).
- VOXZOGO is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials.
What is the most important safety information about VOXZOGO?
- VOXZOGO may cause serious side effects including a temporary decrease in blood pressure in some patients. To reduce the risk of a decrease in blood pressure and associated symptoms (dizziness, feeling tired, or nausea), patients should eat a meal and drink 8 to 10 ounces of fluid within 1 hour before receiving VOXZOGO.
What are the most common side effects of VOXZOGO?
- The most common side effects of VOXZOGO include injection site reactions (including redness, itching, swelling, bruising, rash, hives, and injection site pain), high levels of blood alkaline phosphatase shown in blood tests, vomiting, joint pain, decreased blood pressure, and stomachache. These are not all the possible side effects of VOXZOGO. Ask your healthcare provider for medical advice about side effects, and about any side effects that bother the patient or that do not go away.
How is VOXZOGO taken?
- VOXZOGO is taken daily as an injection given under the skin, administered by a caregiver after a healthcare provider determines the caregiver is able to administer VOXZOGO. Do not try to inject VOXZOGO until you have been shown the right way by your healthcare provider. VOXZOGO is supplied with Instructions for Use that describe the steps for preparing, injecting, and disposing VOXZOGO. Caregivers should review the Instructions for Use for guidance and any time they receive a refill of VOXZOGO in case any changes have been made.
- Inject VOXZOGO 1 time every day, at about the same time each day. If a dose of VOXZOGO is missed, it can be given within 12 hours from the missed dose. After 12 hours, skip the missed dose and administer the next daily dose as usual.
- The dose of VOXZOGO is based on body weight. Your healthcare provider will adjust the dose based on changes in weight following regular check-ups.
- Your healthcare provider will monitor the patient's growth and tell you when to stop taking VOXZOGO if they determine the patient is no longer able to grow. Stop administering VOXZOGO if instructed by your healthcare provider.
What should you tell the doctor before or during taking VOXZOGO?
- Tell your doctor about all of the patient's medical conditions including
- If the patient has heart disease (cardiac or vascular disease), or if the patient is on blood pressure medicine (anti-hypertensive medicine).
- If the patient has kidney problems or renal impairment.
- If the patient is pregnant or plans to become pregnant. It is not known if VOXZOGO will harm the unborn baby.
- If the patient is breastfeeding or plans to breastfeed. It is not known if VOXZOGO passes into breast milk.
- Tell your doctor about all of the medicines the patient takes, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
You may report side effects to
Please see additional safety information in the full Prescribing Information and Patient Information.
About BioMarin
Founded in 1997,
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