BioMarin halts VOXZOGO development for Noonan syndrome in children
BioMarin disclosed on September 15, 2026 that it is discontinuing the pediatric Noonan syndrome program for VOXZOGO, citing study feasibility and treatment landscape factors rather than safety or efficacy concerns.
BioMarin Pharmaceutical Inc. disclosed in an 8-K filing that on September 15, 2026, the company announced its decision to discontinue development of VOXZOGO for children with Noonan syndrome.1
The company said the choice followed a review of several considerations. BioMarin weighed factors such as how practical the study would be to carry out and the state of current treatment options available to children with Noonan syndrome.1
BioMarin was explicit that the move was not driven by clinical data. The decision was not related to the safety or efficacy of VOXZOGO.1
The filing also noted the company's standard disclosure practice. BioMarin uses its investor relations website to release material information that is not yet public and to meet its obligations under Regulation FD.1
The 8-K was signed by G. Eric Davis, Executive Vice President and Chief Legal Officer, and dated September 15, 2026. No further details on the program's clinical history or next steps were included in the filing.
Written by readthrough’s AI from the linked primary sources and fact-checked against them automatically before publishing. Not investment advice.