Drugs

BioMarin Halts Dosing in Some Voxzogo Mid-Stage Trials

BioMarin says it is discontinuing dosing and enrollment in portions of its mid-stage clinical program for Voxzogo (vosoritide) after reports of a rare orthopedic complication emerged in external studies, marking a setback to expand the drug beyond its approved use. In 2021, FDA approved Voxzogo to improve growth in children five years of age and older with achondroplasia and open epiphyses (growth plates), meaning these children still have the potential to grow. Achondroplasia is the most common form of dwarfism.

The recent decision follows several cases of slipped capital femoral epiphysis (SCFE) — a condition affecting the hip joint — in two ongoing investigator-sponsored trials involving patients with Turner syndrome, SHOX deficiency and ACAN deficiency. BioMarin said the events were not observed in its own company-sponsored Phase 2 trials in the same indications but opted to halt dosing as a precaution.

Importantly, the company emphasized that no SCFE cases have been reported in more than 5,000 pediatric patients treated with Voxzogo for achondroplasia. No such events have been seen in trials for hypochondroplasia either, it says.

BioMarin said other studies in its growth disorder pipeline will continue unchanged. These include the Phase 2 CANOPY trials evaluating Voxzogo in Noonan syndrome and in children with idiopathic short stature (ISS), excluding those with ACAN deficiency. The ISS cohort represents the vast majority of participants in that program.

The move comes as BioMarin has been actively pursuing label expansions for Voxzogo, a C-type natriuretic peptide analog designed to improve bone growth. Since its initial approval for achondroplasia, analysts have viewed additional rare growth disorders as key opportunities.

While SCFE is a known risk in children experiencing rapid growth, its appearance in investigational settings raises regulatory and clinical scrutiny, especially when targeting broader pediatric populations.

The company did not disclose whether the pause will lead to protocol amendments or additional safety monitoring requirements but indicated it is continuing to review the data.

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