BioMarin Halts Voxzogo Noonan Syndrome Trial Despite $1B Sales

BioMarin Pharmaceutical is halting clinical development of its drug Voxzogo for children with Noonan syndrome following a pipeline review, even as the medication tracks toward blockbuster sales status. The decision to discontinue the program was announced in a September 15 SEC filing, narrowing the drug's ongoing expansion trials.

"The decision to halt Noonan syndrome development was based on study feasibility and the evolving treatment landscape, and was not driven by concerns over safety or efficacy," according to company disclosures.

Prior Pipeline Cuts and Safety Context

This termination follows earlier reductions in BioMarin's clinical roadmap. In March, the company ended development of Voxzogo for three other short stature indications (Turner syndrome, SHOX-deficiency, and ACAN-deficiency) after an adverse event known as slipped capital femoral epiphysis (SCFE) appeared in investigator-sponsored studies.

Despite the narrowing label expansion plans for Noonan syndrome, the core commercial product continues to scale rapidly. Voxzogo generated $472 million in sales during the first half of 2026, marking a 9% increase compared to the same period in the previous year.

Market Valuation and Financial Performance

Driven by steady demand in its approved indications, the therapy remains on track to achieve blockbuster status. Current full-year projections place expected 2026 sales between $1.00 billion and $1.05 billion.

In the public markets, BioMarin Pharmaceutical Inc (NASDAQ: BMRN) shares traded at $65.08, up 0.54% from a previous close of $64.73, as of September 17, 2026, according to Finnhub market data.

What the Decision Means for BioMarin's Growth

Noonan syndrome is a genetic disorder that causes a wide range of developmental problems, including short stature, heart defects, and characteristic facial features. By trimming exploratory indications where the treatment environment is shifting, BioMarin is directing clinical and capital resources toward protecting and expanding the drug's core commercial footprint.