New Data for BioMarin Pharmaceutical's Drug Voxzogo Shows Promise in Treating Growth-Related Conditions
BioMarin Pharmaceutical has announced new data for its drug Voxzogo (vosoritide). The treatment, currently approved for a type of short-limbed dwarfism, has shown promise in improving growth velocity in children with idiopathic short stature and Noonan syndrome. In a study of eight children, mean annualized growth velocity increased from 3.7 cm/year to 8.5 cm/year after 12 months of treatment. Additionally, a phase 3 extension study of Voxzogo in children with achondroplasia showed improvements in growth velocity compared to children not on the drug. BioMarin has updated its 2024 guidance, expecting an EPS raise but no change to revenue.
Key Takeaways
- BioMarin Pharmaceutical's treatment Voxzogo shows effectiveness for other growth-related conditions, including ISS and Noonan syndrome.
- Study of Voxzogo in children with ISS or Noonan syndrome aged 3-11 found improvement in annualized growth velocity (AGV) and height standard deviation.
- Mean AGV increased from a baseline of 3.7 cm/year to 8.5 cm/year, and mean height SD changed from -3.6 SD to -2.9 SD in children who finished 12 months of treatment.
- Phase 3 extension study of Voxzogo in children with achondroplasia showed improvements in AGV compared to children not on the drug.
- Mean difference in AGV between treated and untreated children was 1.47 cm/year in girls and 1.71 cm/year in boys for those continuing treatment for more than three years.
Analysis
BioMarin Pharmaceutical's Voxzogo drug shows promise in treating growth-related conditions beyond achondroplasia, including ISS and Noonan syndrome. This development may prompt further research and investment in gene therapy for rare diseases. The improved growth velocity in treated children could lead to better quality of life and reduced healthcare costs. Positive results from the phase 3 extension study may bolster BioMarin's market position in growth disorders, potentially increasing shareholder value. However, the impact on revenue remains uncertain, as BioMarin's 2024 guidance shows no change. Regulatory agencies and competitors will closely monitor these developments, possibly influencing their strategies in gene therapy and orphan drugs.
Did You Know?
- Voxzogo (vosoritide): This is a drug developed by BioMarin Pharmaceutical for the treatment of short-limbed dwarfism. The new data announced by BioMarin suggests that the drug may also be effective in improving growth velocity in children with idiopathic short stature (ISS) and Noonan syndrome.
- Idiopathic short stature (ISS): This is a condition in which a child's height is significantly below the average for their age and sex, but there is no known underlying medical cause.
- Noonan syndrome: This is a genetic disorder that affects the normal development of various parts of the body, including the heart, blood vessels, and bones.