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Biomarin Pharmaceutical Inc BMRN

Rating as of

Morningstar’s Analysis

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1-Star Price

PREMIUM

5-Star Price

PREMIUM

Economic Moat

PREMIUM

Capital Allocation

PREMIUM

Maintaining Our $101 FVE for BioMarin Following Clinical Hold on BMN 307

Karen Andersen, CFA Sector Strategist

Analyst Note

| Karen Andersen, CFA |

The Food and Drug Administration has placed a clinical hold on BioMarin's phase 1/2 clinical trial of phenylketonuria gene therapy BMN 307 in the United States, following 52-week data from a preclinical study in mice, and BioMarin has opted to pause global enrollment. We're not making any changes to our $101 fair value estimate, as we had not yet included BMN 307 explicitly in our model. We also don't see evidence that any potential BMN 307 cancer risk in humans could be extrapolated to BioMarin's Roctavian, its gene therapy in hemophilia A that is poised to gain regulatory approval in 2022. We continue to see BioMarin's rare-disease portfolio and late-stage pipeline supporting a narrow moat. That said, if these safety concerns are found to reflect a safety issue with BioMarin's entire gene therapy platform or adeno-associated virus-based gene therapies more broadly, we might reconsider our long-term earnings growth assumptions and positive moat trend rating; this could also affect development of gene therapy BMN 331 in hereditary angioedema (to enter clinical development this year).

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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). BioMarin's Roctavian (hemophilia A gene therapy) and vosoritide (treatment for achondroplasia) are poised to potentially launch in the 2021-22 timeframe.

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

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