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Biomarin Pharmaceutical Inc BMRN

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Maintaining BioMarin $105 FVE Following Encouraging Confirmatory Hemophilia Data

Karen Andersen, CFA Sector Strategist

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.

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Company Profile

Business Description

BioMarin's focus is on rare-disease therapies. Genzyme (now part of Sanofi) markets Aldurazyme through its joint venture with BioMarin, and BioMarin markets Naglazyme, Vimizim, and Brineura independently. BioMarin also markets Kuvan and Palynziq to treat the rare metabolic disorder PKU (in addition to long-standing U.S. rights, BioMarin has reacquired international rights for Kuvan and Palynziq from Merck KGaA). Voxzogo (vosoritide) was approved in achondroplasia in 2021. BioMarin's Roctavian (hemophilia A gene therapy) is poised to potentially launch in the 2022-23 timeframe.

770 Lindaro Street
San Rafael, CA, 94901
T +1 415 506-6700
Sector Healthcare
Industry Biotechnology
Most Recent Earnings Sep 30, 2021
Fiscal Year End Dec 31, 2021
Stock Type Speculative Growth
Employees 3,059