BioMarin's Voxzogo Launches Strongly Ahead of Potential Roctavian Approval

We're maintaining our $105 fair value estimate for BioMarin following solid first-quarter results, with 7% top-line growth despite a headwind from generic competition to oral PKU drug Kuvan. Sales of newly approved achondroplasia drug Voxzogo reached $20 million, and management has raised full-year sales guidance for the drug to $100 million-$125 million (still below our $135 million forecast). BioMarin should also have data for the drug in short stature imminently, which could further expand sales of the drug beyond our assumed roughly $1.5 billion in peak sales. We think the firm is on track to reach $2.16 billion in sales in 2022, just above the $2.05 billion-$2.15 billion guidance provided by management, based on this strong Voxzogo launch and continuing growth for the firm's other rare-disease therapies, including Vimizim and Naglazyme. BioMarin's diversified rare-disease lineup supports a narrow moat.