The current read
BioMarin is emerging from its reset as a more diversified and profitable rare-disease company, but the quality of the recovery is less compelling than the headline earnings multiple suggests. Amicus adds durable growth assets and meaningful cost synergies, while hypochondroplasia provides a credible second leg for VOXZOGO.
- Against that, the core achondroplasia franchise now faces a once-weekly competitor, the YUVIWEL royalty cushion expires in 2030, and the balance sheet carries substantially more leverage. We need evidence that acquired growth and deleveraging can more than offset CNP share loss and mature-portfolio erosion before taking a more constructive view.