BioMarin to Present Data Underscoring Sustained Positive Impact of VOXZOGO® (vosoritide) on Health-Related Quality of Life, Growth and Maintenance of Bone Strength in Children with Achondroplasia at 2024 International Skeletal Dysplasia Society Meeting
Results from
New Data Highlight Meaningful Improvements in Health-Related Quality of Life for Children with Achondroplasia
Investigator-Led Study Shows Significant Increases in Bone Length While Maintaining Bone Strength in Children with Achondroplasia
Investigator-Led Study Observes Sustained Growth Gains in Children with Hypochondroplasia, Noonan Syndrome and Genetic Variants Associated with Idiopathic Short Stature
"VOXZOGO is now becoming the standard of care in achondroplasia, based on its proven effects on growth velocity, its safety profile and the clinical data demonstrating positive impact on proportionality and quality of life in treated children with achondroplasia," said
Positive Impacts in Achondroplasia on Health Outcomes Including HRQoL and Maintenance of Bone Strength
Qualitative insights elucidating caregiver perspectives of children with achondroplasia revealed that VOXZOGO had positive impacts on HRQoL across several physical, emotional and social functioning domains. Notable physical functioning improvements reported included improved motor skills (e.g., higher reach, walking, running, balance and cycling) and self-care, which are particularly meaningful outcomes for children and families impacted by the condition. Psychosocial functioning outcomes included improved confidence and social interactions.
Additional results from an investigator-led analysis of
"VOXZOGO is the first and only approved treatment for children with achondroplasia, providing families with an option that can be initiated in infants, and we are excited to continue investigating its possibilities in other genetic skeletal conditions through our CANOPY clinical program," said
Investigator-Led Studies of VOXZOGO Show Promising Efficacy and Safety Data in Hypochondroplasia and Other Genetic Skeletal Conditions
Additional presentations at ISDS include efficacy data for VOXZOGO in ongoing research across genetic skeletal conditions beyond achondroplasia. Safety results were shown to be consistent with the well-characterized safety profile of VOXZOGO in achondroplasia.
Results from an investigator-sponsored study showed sustained improvement in mean annualized growth velocity (AGV) and height standard deviation (SD) in 24 children with various genetic skeletal conditions, including Noonan syndrome and those with genetic variants associated with idiopathic short stature (ACAN deficiency and heterozygous
Updated investigator-sponsored results from the first clinical study of VOXZOGO for children with hypochondroplasia showed sustained improvement in AGV and hypochondroplasia-specific height SDs in 26 participants over one year. Treatment efficacy was similar to what had been previously seen in achondroplasia, and there were no new safety signals observed.
Key presentations at ISDS are listed below, with all times in Central European
Oral Presentations
Adjuvant Therapeutic Enhances Bone Growth and Quality in Growing Mice with Moderate-to-Severe Osteogenesis Imperfecta: Exploration of a CNP Analog
Oral #C-0012
Vosoritide Increases Growth in Children with Hypochondroplasia: Phase 2 Trial Results
Oral #C-0017
Vosoritide Improves Growth in RASopathies, ACAN and
Oral #C-0022
Body Composition by Bioelectrical Impedance Analysis (BIA) in Adults with Achondroplasia in CLARITY (the Achondroplasia Natural History Study)
Oral #C-0027
Poster Presentations
Caregiver Perspectives on Vosoritide Treatment of Children with Achondroplasia
Poster #C-0090
Examining the Effect of Vosoritide Treatment on Bone Strength in Children with Achondroplasia
Poster #C-0033
The Achondroplasia Roadmap
Poster #C-0066
Design and Objectives of the Acorn Study: A Non-Interventional Study Evaluating Long-Term Safety in Achondroplasia Children Treated with Vosoritide
Poster #C-0110
About the VOXZOGO CANOPY Clinical Program
The CANOPY clinical program was designed to evaluate the potential of VOXZOGO (vosoritide) in children with various genetic skeletal conditions, including achondroplasia, hypochondroplasia, Noonan syndrome, SHOX deficiency, Turner syndrome and idiopathic short stature, with the goal of addressing the unmet needs of and expanding treatment options for children and families impacted by these conditions.
Studies underway as part of the CANOPY program beyond achondroplasia include:
- CANOPY HCH-OS, a multinational observational study in children with hypochondroplasia.
- CANOPY HCH-3, a Phase 3 randomized, placebo-controlled, double-blind multicenter study in children with hypochondroplasia.
- CANOPY ISS-OS, a multinational observational study in children with idiopathic short stature.
- CANOPY
ISS-2 , a Phase 2 randomized, controlled, multicenter study in children with idiopathic short stature. - CANOPY NS, TS, SHOX-D-2, a Phase 2 study in multiple genetic skeletal conditions including Noonan syndrome, Turner syndrome and SHOX deficiency.
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, approximately 3,500 people with achondroplasia around the world have received VOXZOGO. In total, VOXZOGO is available in 44 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 skeletal 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,
Forward-Looking Statements
This press release contains forward-looking statements about the business prospects of
Contacts: |
|
|
|
Investors |
Media |
Traci McCarty |
|
|
|
(415) 455-7558 |
(628) 269-7393 |
View original content to download multimedia:https://www.prnewswire.com/news-releases/biomarin-to-present-data-underscoring-sustained-positive-impact-of-voxzogo-vosoritide-on-health-related-quality-of-life-growth-and-maintenance-of-bone-strength-in-children-with-achondroplasia-at-2024-international-skeletal-dys-302251087.html
SOURCE