BioMarin halts development of BMN401 enzyme therapy after mixed Phase 3 results

BioMarin announced it will stop work on BMN401, an enzyme replacement therapy aimed at treating ENPP1 deficiency, a rare genetic disorder.

The decision follows a Phase 3 trial that produced mixed efficacy and safety data, prompting the company to reassess the asset's commercial potential.

By discontinuing the program, BioMarin will redirect resources to other pipeline candidates, while investors will watch how the move impacts the company's second-quarter financial outlook.

The news underscores the challenges of developing therapies for ultra-rare conditions, where trial outcomes can be uncertain.