BioMarin is pumping the brakes on yet another indication for its CNP analog, Voxzogo.
The rare disease biotech announced that it would discontinue efforts to develop Voxzogo for children with Noonan syndrome, a genetic disorder that can affect height, facial features and the heart.
BioMarin said the decision to discontinue was not based on the safety or efficacy of Voxzogo, but instead related to the “feasibility of the study and the current treatment landscape for children with Noonan syndrome,” according to a securities filing.
Voxzogo is already FDA-approved to treat achondroplasia, a form of dwarfism. And BioMarin filed for an indication expansion with the FDA earlier this month based on strong phase 3 results for the drug in children with hypochondroplasia, a milder form of dwarfism.
But Noonan syndrome is not the first indication trimmed from the Voxzogo pipeline this year. Earlier this spring, BioMarin discontinued dosing and enrollment in phase 2 trials testing Voxzogo in three other indications: Turner syndrome, SHOX deficiency and aggrecan (ACAN) deficiency.
At that time, BioMarin said the choice to stop the initial trials was based on reports of possible adverse effects, namely slipped capital femoral epiphysis (SCFE) events, but trials investigating Noonan syndrome would continue as planned.
The new decision to further refine Voxzogo’s pipeline strategy likely points to the drug’s changing competitive landscape.
In February, the FDA approved Ascendis’ Yuviwel to treat achondroplasia. The rival treatment offers logistical advantages, requiring only weekly injections compared with daily injections of Voxzogo.
The growing competitive pressures have been significant, with analysts at Leerink Partners saying that BioMarin’s success in the new hypochondroplasia indication may not be enough to offset competitive losses due to Yuviwel.
BioMarin did manage to recoup some of those losses last month. In a deal to end patent disputes in five countries where BioMarin alleged that Yuviwel infringed on its intellectual property, Ascendis agreed to pay BioMarin a 20% royalty on U.S. net sales and an 18% royalty on net sales in the European Union, Brazil and South Korea. In exchange, BioMarin agreed to dismiss pending proceedings and grant Ascendis a broad license to patents related to Yuviwel and navepegritide products.
In August, BioMarin upped its expected 2026 revenue from Voxzogo to $1 billion to $1.05 billion, citing strong patient demand and adherence. The company’s stock slipped just under 3% after it announced the discontinuation of the Noonan syndrome program.