Analyst Note| Karen Andersen, CFA |
BioMarin announced positive two-year data from its phase 3 study of hemophilia A gene therapy Roctavian (val-rox) that we believe will help support launches beginning in late 2022, and we're maintaining our $105 fair value estimate. The trial showed that BioMarin's one-time gene therapy was superior to standard of care (chronic factor VIII prophylaxis therapy) in its ability to reduce bleeds, with the annualized bleed rate actually declining slightly further in year two of the study (and in year three for the subset of patients with three years of data) despite declining factor VIII levels over time. The regulatory submission schedule appears to be on track, with filings expected in Europe in the first quarter and the U.S. in the second quarter. We continue to assume approval in Europe in the second half of 2022 and the U.S. in early 2023, with $1 billion in peak global sales, assuming a net global price around $1.2 million per patient ($2 million U.S.) and market share (of non-cured severe hemophilia adults and adolescents) rising as high as 4%. We think BioMarin's established rare disease portfolio, recently approved achondroplasia drug Voxzogo, and key pipeline program Roctavian all support a narrow economic moat.