BioMarin Pharmaceutical Inc. announced it is discontinuing the development of its drug, VOXZOGO, for the treatment of children with Noonan syndrome. The company attributed the decision to the feasibility of the required study and the current treatment landscape for the condition.
Key Details
- Program Halted: The development program for VOXZOGO in the Noonan syndrome indication has been stopped as of September 15, 2026.
- Stated Rationale: The decision was explicitly not related to any safety or efficacy concerns with the drug.
- Strategic Focus: This move represents a strategic shift in the company's development pipeline for this specific indication.