Full Press Release Details
Conference January 9, 2023 Transforming Lives Through Genetic Discovery J.J. Bienaime Chairman and Chief Executive Officer
Safe Harbor This non -confidential presentation contains
forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the expected financial performance of BioMarin, including, without limitation, preliminary unaudited
total revenues and revenue from sales of VOXZOGO for fiscal year 2022, double-digit revenue growth in 2023, sustainable growth and profitability, profit margin expansion, mid-decade revenues, and other specified financial guidance; the markets for
BioMarin's products and product candidates, including, without limitation, the addressable patient populations in BioMarin territories and commercial market opportunities; and BioMarin's development of product candidates and commercialization of
products, including, without limitation, IND submissions anticipated in 2023, the outcome of BioMarin's BLA resubmission for ROCTAVIAN to the FDA, and the commercial launch of ROCTAVIAN. These forward-looking statements are predictions and involve
risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: completion of BioMarin's normal quarter and year-end accounting procedures for the quarter and the
year ended December 31, 2022 and completion of the external audit with respect to such periods by BioMarin's independent registered public accounting firm; results and timing of current and planned preclinical studies and clinical trials; BioMarin's
ability to successfully manufacture its commercial products and product candidates; the content and timing of decisions by the FDA, the European Commission and other regulatory authorities concerning products and product candidates; BioMarin's
success in the commercialization of its commercial products; and those factors detailed in the press release issued January 8, 2023, as well as BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the
factors contained under the caption Risk Factors in BioMarin's Quarterly Report on Form 10-Q for the quarter ended September 30, 2022 as such factors may be updated by any subsequent reports. Investors are urged not to place undue reliance on
forward -looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward - looking statement, whether as a result of new information, future events or
otherwise. BiOMARI N 2
Significant Future Growth Expected Driven by VOXZOGO and ROCTAVIAN
VOXZOGO & ROCTAVIAN N $46 - $56 >2x New Base $4B-$5B Products Products -50% -50% Mid-Decade $2.09B $1.85B N 2021 20221 Mid- / End of Decade Decade All other products Anticipated Revenue Growth Enablers Continued growth within Base indication
launch of Regulatory approval of U.S. Expansion of VOXZOGO & base portfolio VOXZOGO & ROCTAVIAN ROCTAVIAN ROCTAVIAN indications BIOMARIN 6 1 Unaudited preliminary 2022 revenue estimate
(ACH) VOXZOGO: Opportunity to Expand Markets in Achondroplasia 1,000+
Additional Children Eligible for Treatment upon Anticipated U.S. and E. U. Age Expansions VOXZOGO Achondroplasia Market Opportunity Launch Excellence 2022 Commercial Execution $3B+ 1 year anniversary Annual Over 1,000 children
treated at 800 18,000+ Market 300 Opportunity year-end -17,000 32 active markets to date Approved $169M1 expected 2022 revenue age ranges: U.S. 5+ 2023 Opportunities Japan all Continued uptake of patients, and
EMA 2+ expansion into additional new ROW 2+ markets ACH U.S. Total Potential EMA Initially Potential age label expansions in Indicated Expansion Indicated ACH Indicated Expansion Market Value Age <5 Patients Patients Age
<2 the U.S. and Europe (Approximately 1,000 newborns with achondroplasia born each year in BioMarin territories) BiOIMARIN 9 1 Unaudited preliminary 2022 revenue estimate