BioMarin has discontinued dosing and enrolment in Phase II trials of Voxzogo (vosoritide) in Turner Syndrome, SHOX-deficiency and Aggrecan (ACAN)-deficiency after safety events in two investigator-led trials.
According to a US Securities and Exchange Commission (SEC) listing, filed on 16 March, several patients suffered slipped capital femoral epiphysis (SCFE) in two ongoing investigator-sponsored trials. This event is characterised by the head of the femur slipping backwards off the growth plate.
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BioMarin said that SCFE events have not been observed in its other Phase II trials in these same conditions. The company added that no cases have been observed in the more than 5,000 infants and children who have received Voxzogo for achondroplasia in clinical studies or post-marketing surveillance.
There have similarly been no observed cases in BioMarin’s clinical trials for hypochondroplasia, an indication in which BioMarin is focusing for the drugs next approval.
The Phase II CANOPY trials of Voxzogo in children with Noonan syndrome (NCT06668805), as well as those living with idiopathic short stature (ISS) (NCT06382155) without ACAN-deficiency, will continue as planned.
Voxzogo gained approval for achondroplasia, a form of dwarfism, from the US Food and Drug Administration (FDA) in November 2021 in patients aged five and older. It has since been approved for achondroplasia patients of all ages with open growth plates. The subcutaneous drug is administered daily.
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By GlobalDataBioMarin set for competition in achondroplasia
Voxzogo sales reached $927m in 2025, a 26% rise over 2024 sales. BioMarin forecasts sales of the drug between $975m and $1bn in 2026.
The drug is set for competition as, in February 2026, the FDA approved Ascendis Pharma’s Yuviwel (navepegritide) to treat achondroplasia in patients aged two and older with open growth plates. Yuviwel has an advantage, however, with once-weekly dosing as opposed to the daily dosing with BioMarin’s Voxzogo.
BridgeBio is eyeing the space too, announcing a positive readout of a Phase III trial of its achondroplasia therapy infigratinib last month. Based on the pivotal trial data, the company hopes to meet with regulators in H2 2026 to discuss next steps for the drug.
GlobalData forecasts Yuviwel to bring in the lowest sales of the three, reaching $592m in 2031, while infigratinib will generate $598m in the same year. Voxzogo is forecast to bring in $1.3bn in 2031.
GlobalData is the parent company of Clinical Trials Arena.
