SAN RAFAEL - BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced the presentation of new data from an investigator-led analysis of the Phase 2 111-205 study, which demonstrate that children with achondroplasia treated with VOXZOGO experienced increases in bone length while maintaining bone strength.
The data will be presented at the 11th International Conference on Children s Bone Health (ICCBH) in Salzburg, Austria, June 22-25, 2024. BioMarin will also share the first results from a study underscoring the unmet medical needs of people living 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 Hank Fuchs, M.D., president of Worldwide Research and Development at BioMarin. CNP is emerging as a natural and holistic regulator of statural development, and safety and efficacy data presented to date illustrate VOXZOGO s positive impact on bone growth, proportionality and quality of life. Based on this extensive evidence, we are accelerating the development of VOXZOGO across a multitude of growth-related conditions with significant unmet need.
Significant Impact on Bone Length, Strength, Proportionality and Health-Related Quality of Life in Achondroplasia
Results from an investigator-led analysis of BioMarin s Phase 2 111-205 study found that children who received VOXZOGO (n=30) had significant increases in bone length and metacarpal cortical area after approximately five years of therapy, suggesting that treatment allowed the bone to remain strong as it lengthened.
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 Cathleen Raggio, M.D., a pediatric orthopedic surgeon at the Hospital for Special Surgery in New York. With this study, for the first time we have shown that treatment with VOXZOGO enabled bones to remain strong as they lengthened, which is promising as we continue to better understand the impact of this therapeutic advancement.
Additional data to be presented at ICCBH, previously shared at the 2024 Pediatric Endocrine Society Annual Meeting, showcase VOXZOGO s efficacy, safety and impact on health-related quality of life in children with achondroplasia. Data observed in the Phase 3 111-301 and 111-302 studies suggest that VOXZOGO can have the potential to improve health-related quality of life among children with achondroplasia, particularly in aspects associated with physical functioning, an outcome of significant importance for children and families impacted by achondroplasia. Phase 2 and Phase 3 data also demonstrated consistent positive effects on linear growth and improvement in proportionality in children of all ages with growth potential, with follow-up conducted up to four years after the initiation of VOXZOGO treatment. Safety results were consistent with the well-characterized safety profile of VOXZOGO.
Data Underscore Unmet Needs in Hypochondroplasia
Data from a retrospective, real-world matched cohort study using electronic medical records from England showed that comorbidity event rates (e.g., respiratory, cardiovascular, orthopedic and mental health-related events) were higher in people living with hypochondroplasia (n=610) compared to people living without the condition (n=2440). General practitioner visits (median annual visits = 11.2 vs. 5.4, risk ratio [RR] = 1.8), hospital in-patient admissions (RR=5.5) and average length of hospital stay (5.6 vs. 3.8 days) were all greater as well. Most people evaluated in the study were aged 16-65 at the start of follow up. These data suggest there is a significant burden of disease amongst people of all ages with hypochondroplasia, highlighting the need for proactive management and linkage to care.
VOXZOGO Clinical Development Program Milestones
Building on its leadership in achondroplasia, BioMarin has several clinical trials underway for hypochondroplasia, idiopathic short stature (ISS) and other growth-related conditions.
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.
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 U.S. and Japan to increase linear growth in children of all ages with achondroplasia with open epiphyses, and VOXZOGO is indicated in the EU for the treatment of achondroplasia in children 4 months of age and older whose epiphyses are not closed, as confirmed by appropriate genetic testing. In the U.S., this indication 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 trial(s). To fulfill this post-marketing requirement, BioMarin intends to use the ongoing open-label extension studies compared to available natural history.
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.
About BioMarin
Founded in 1997, BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery. The company develops and commercializes targeted therapies that address the root cause of the genetic conditions. BioMarin s unparalleled research and development capabilities have resulted in eight transformational commercial therapies for patients with rare genetic disorders. The company s distinctive approach to drug discovery has produced a diverse pipeline of commercial, clinical, and pre-clinical candidates that address a significant unmet medical need, have well-understood biology, and provide an opportunity to be first-to-market or offer a substantial benefit over existing treatment options.
Forward-Looking Statements
This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: data to be presented at the 11th International Conference on Children s Bone Health (ICCBH), including the oral and poster presentations; the development of BioMarin s VOXZOGO program generally; VOXZOGO s efficacy, safety and impact on health-related quality of life in children with achondroplasia, including impact on bone length, strength and health-related quality of life; the potential benefits of VOXZOGO for children with growth-related conditions beyond achondroplasia, including hypochondroplasia and idiopathic short stature; potential unmet needs in hypochondroplasia and the continued clinical development of VOXZOGO, including BioMarin s plans for clinical trials for hypochondroplasia, idiopathic short stature and other growth-related conditions. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: results and timing of current and planned pre-clinical studies and clinical trials of VOXZOGO; any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the content and timing of decisions by the Food and Drug Administration, the European Commission and other regulatory authorities and those factors detailed in BioMarin s filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption Risk Factors in BioMarin s Quarterly Report on Form 10-Q for the quarter ended March 31, 2024, as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.
Contact:
Traci McCarty
Tel: (415) 455-7558
BIOMARIN PHARMACEUTICAL INC. 
