BioMarin Pharmaceutical has discontinued development of VOXZOGO for children with Noonan syndrome, according to a Sept. 15 securities filing and reporting by Fierce Pharma.
BioMarin said its decision followed an assessment of study feasibility and the current treatment landscape for children with Noonan syndrome. The company said the decision was not related to the safety or efficacy of VOXZOGO.
Noonan syndrome is a genetic disorder that can affect height, facial features and the heart, Fierce Pharma reported.
The decision is the latest change to BioMarin’s VOXZOGO development program. Earlier this year, the company discontinued dosing and enrollment in Phase II studies of VOXZOGO in Turner syndrome, SHOX deficiency and aggrecan, or ACAN, deficiency, according to Fierce Pharma.
BioMarin said those earlier decisions were based on reports of possible adverse effects, including slipped capital femoral epiphysis events. At that time, the company said studies in Noonan syndrome would continue.
VOXZOGO is approved by the U.S. Food and Drug Administration for achondroplasia, a form of dwarfism. BioMarin also submitted an FDA application earlier this month to expand use of the treatment to children with hypochondroplasia, based on Phase III results, Fierce Pharma reported.
Ascendis Pharma’s Yuviwel was approved by the FDA in February for achondroplasia. Yuviwel is administered weekly, while VOXZOGO is administered daily, according to Fierce Pharma.
In August, BioMarin raised its 2026 VOXZOGO revenue forecast to between $1 billion and $1.05 billion, citing patient demand and adherence. Fierce Pharma reported that BioMarin shares fell by just under 3% after the company announced the Noonan syndrome development decision.
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