Analyst Note| Karen Andersen, CFA |
We're maintaining our BioMarin fair value estimate at $101 per share following first-quarter results that were in line with our estimates. BioMarin's portfolio of rare-disease therapeutics drove a 3% decline in total revenue for the quarter, but excluding Kuvan (BioMarin's only small-molecule therapy, which is facing generic competition), sales for the remaining portfolio grew 9%. BioMarin's two key pipeline programs continue to generate long-term data that we think supports approval, and we model probability-adjusted peak sales for Voxzogo (vosoritide) and Roctavian at more than $1 billion each. We think BioMarin's growing rare-disease portfolio supports a narrow moat.