Investors: Media: Traci McCarty Debra Charlesworth BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical Inc. (415) 455-7558 (415) 455-7451 BioMarin Announces First Quarter 2019 Financial Results - Total Revenues

BioMarin Announces First Quarter 2019 Financial Results

- Total Revenues Top $400 million in the Quarter

- Full-year 2019 Total BioMarin Revenue Guidance of Between $1.68 billion to $1.75 billion Reaffirmed

- As of March 31, 2019, 414 U.S. Commercial Patients Receiving Treatment with Palynziq

Financial Highlights (in millions of U.S. dollars, except per share data, unaudited)

SAN RAFAEL, Calif., April 25, 2019 - BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) (BioMarin or the Company) today announced financial results for the first quarter ended March 31, 2019.

Total Net Product Revenues for the first quarter 2019 increased to $394.5 million, compared to $369.1 million for the first quarter of 2018. Net Product Revenues Marketed by BioMarin increased to $349.2 million, compared to $303.0 million for the first quarter of 2018. The increase in Net Product Revenues Marketed by BioMarin was attributed to the following:

Aldurazyme Net Product Revenues decreased $20.8 million, or 31%, due to timing of shipments to Genzyme. Under the new revenue standards adopted in 2018, the Company records Aldurazyme Net Product Revenues when the product is released and control is transferred to Genzyme based on the estimated variable consideration payable it expects to earn when the product is sold through by Genzyme. Aldurazyme net product sales reported by Genzyme increased to $75.7 million, or up 20% in the first quarter of 2019, compared to $62.9 million the same period in 2018.

The increase in GAAP Net Loss for the first quarter of 2019, compared to the same period in 2018 was primarily due to the following:

Non-GAAP Income for the first quarter of 2019 increased $3.5 million, or 16%, to $24.8 million, compared to $21.3 million for the same period in 2018. The increase in Non-GAAP Income for the quarter was attributed to increased gross profit from sales partially offset by higher SG&A expenses as described above.

As of March 31, 2019, BioMarin had cash, cash equivalents and investments totaling approximately $1.2 billion, as compared to $1.3 billion on December 31, 2018.

Commenting on first quarter results, Jean-Jacques Bienaim , Chairman and Chief Executive Officer of BioMarin, said, "We begin 2019 well-positioned for potential significant achievements across our late-stage product pipeline, as well as record Total Revenues for the full year. Starting with valoctocogene roxaparvovec gene therapy for severe hemophilia A, our pivotal study is on track to complete enrollment in the third quarter. Based on draft guidance from the FDA for hemophilia gene therapy products published in 2018, we have the opportunity to pursue a potential accelerated approval path forward based on Factor VIII activity results from a subset of Phase 3 subjects. Based on the results from the subset of Phase 3 subjects, we will make and communicate our decision on whether or not to pursue an accelerated approval before the end of the year. We have observed a high level of interest and enthusiasm from the hemophilia community in support of valoctocogene roxaparvovec and are hopeful that it will be a potential treatment option for these patients in the very near future."

Mr. Bienaim continued, "We have a number of other exciting catalysts on the horizon including the potential approval and launch of Palynziq in Europe later this year. We have been thrilled with the pace of the U.S. launch, as we ended the first quarter with 414 patients on reimbursed Palynziq. Building on this success and as part of our strategy to increase our leadership in the PKU market, we anticipate filing an IND for BMN 307, our gene therapy product for PKU, in the second half of 2019. BMN 307 demonstrated lifetime normalization of Phe in a validated PKU mouse model, and as a result we believe it has the potential to be an important new treatment and market expander as part of our PKU franchise. Finally, we look forward with great anticipation to the results of our global Phase 3 program with vosoritide for the treatment of achondroplasia. Our ongoing Phase 2 study has so far demonstrated an average additional cumulative height gain of 5.7 centimeters over 42 months. Based on data observed to date, we are very encouraged that vosoritide could potentially be the first approved treatment option for children with achondroplasia."

2019 Full-Year Financial Guidance unchanged (in millions, except %)

*All Financial Guidance items are calculated based on U.S. GAAP with the exception of Non-GAAP Income/Loss. Refer to Non-GAAP Information beginning on page 8 of this press release for a complete discussion of the Company's Non-GAAP financial information and reconciliations to the comparable GAAP reported information.

Key Program Highlights

In 2018, the Company updated the protocol for the Phase 3 GENEr8-1 study evaluating the 6e13 vg/kg dose and has statistically powered the study results to evaluate superiority to the current standard of care, Factor VIII prophylaxis. The complete Phase 3 GENEr8-1 study will include 130 participants, and is expected to be fully enrolled in the third quarter of 2019.

BioMarin received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMA), on Palynziq on March 1, 2019. Based on this positive opinion, the Company anticipates marketing authorization in the European Union by the end of the second quarter of this year.

In 2018, BioMarin began a global Phase 2 study with vosoritide in infants and young children (less than 60 months old) with achondroplasia, to determine the impact of treatment in this age group. Three cohorts, segmented by age, are being enrolled in this study. Cohort 1 includes children ages 24 to 60 months old and will complete enrollment this year. Cohort 2 includes children ages 6 to 24 months old and is currently enrolling. Following evaluation of safety and pharmacokinetics in sentinel subjects in cohort 2, cohort 3 will begin enrolling infants up to 6 months old.

BioMarin will host a conference call and webcast to discuss first quarter 2019 financial results today, Thursday, April 25, 2019 at 4:30 p.m. ET. This event can be accessed on the investor section of the BioMarin website at www.biomarin.com.

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases. The Company's portfolio consists of several commercial products and multiple clinical and pre-clinical product candidates for the treatment of various diseases. For additional information, please visit www.biomarin.com.

Forward-Looking Statements

This press release and the associated conference call and webcast contain forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the expectations of Total Revenues, Net Product Revenues and expenses for BioMarin's commercial products, GAAP Net Loss, Non-GAAP Income and other specified income statement guidance; the financial performance of BioMarin as a whole; BioMarin's potential for growth: BioMarin being well-position for significant achievements across its late-stage product pipeline, as well as record Total Revenues for the full year; BioMarin's commercial prospects, including the timing of (i) decisions by regulators, including the European Commission's decision regarding BioMarin's Marketing Authorization Application for Palynziq, (ii) BioMarin's clinical studies and trials, (iii) completion of enrollment of those studies and trials including enrollment in BioMarin's Phase 3 program with valoctocogene roxaparvovec, and (iv) announcements of data from those studies and trials, including BioMarin's Phase 3 program and Phase 1/2 study with valoctocogene roxaparvovec; the ongoing Phase 2 and Phase 3 studies of vosoritide; the continued clinical development and commercialization of BioMarin's commercial products and product candidates, including (i) BioMarin's plans to potentially decide in the first half of 2019 whether to file an IND for BMN 290 and potentially file an IND and/or a CTA for its new gene therapy candidate for the treatment of PKU in the second half of 2019; (ii) statements regarding the timing of BioMarin's decision on whether or not to pursue an accelerated approval pathway for valoctocogene roxaparvovec and the communication of that decision; (iii) statements regarding BioMarin's encouragement that vosoritide could potentially be the first approved treatment option for children with achondroplasia; and (iv) the possible approval and commercialization of BioMarin's product candidates.

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: BioMarin's success in the commercialization of its commercial products; Genzyme Corporation's success in continuing the commercialization of Aldurazyme; 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 each of the described products and product candidates; the market for each of these products; actual sales of BioMarin's commercial products; the introduction of generic versions of BioMarin's commercial products, in particular generic versions of Kuvan; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Annual Report on Form 10-K for the year ended December 31, 2018 as such factors may be updated by any subsequent reports. Stockholders 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.

BioMarin , Brineura , Firdapse , Kuvan , Naglazyme , Palynziq and Vimizim are registered trademarks of BioMarin Pharmaceutical Inc., or its affiliates. Aldurazyme is a registered trademark of BioMarin/Genzyme LLC.

BIOMARIN PHARMACEUTICAL INC.

CONDENSED CONSOLIDATED BALANCE SHEETS

March 31, 2019 and December 31, 2018

(In thousands of U.S. dollars, except share and per share amounts)

BIOMARIN PHARMACEUTICAL INC.

CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS

Three Months Ended March 31, 2019 and 2018

(In thousands of U.S. dollars, except per share amounts)

The following table presents the Net Product Revenues by Product:

Net Product Revenues By Product

(In millions of U.S. dollars)

Non-GAAP Information

The results presented in this press release include both GAAP information and Non-GAAP information. As used in this release, Non-GAAP Income is defined by the Company as GAAP Net Loss excluding net interest expense, provision for (benefit from) income taxes, depreciation expense, amortization expense, stock-based compensation expense, contingent consideration expense and, in certain periods, certain other specified items, as detailed below when applicable. In addition, BioMarin includes in this press release the effects of these adjustments on certain components of GAAP Net Loss for each of the periods presented. In this regard, Non-GAAP Income and its components, including Non-GAAP Cost of Sales, Non-GAAP Research and Development expenses, Non-GAAP Selling, General and Administrative expense, Non-GAAP Intangible Asset Amortization and Contingent Consideration, Non-GAAP Gain on the Sale of Intangible Asset and Non-GAAP Benefit From Income Taxes are statement of operations line items prepared on the same basis as, and therefore components of, the overall Non-GAAP measures.

BioMarin regularly uses both GAAP and Non-GAAP results and expectations internally to assess its financial operating performance and evaluate key business decisions related to its principal business activities: the discovery, development, manufacture, marketing and sale of innovative biologic therapies. Because Non-GAAP Income and its components are important internal measurements for BioMarin, the Company believes that providing this information in conjunction with BioMarin's GAAP information enhances investors' and analysts' ability to meaningfully compare the Company's results from period to period and to its forward looking guidance, and to identify operating trends in the Company's principal business. BioMarin also uses Non-GAAP Income internally to understand, manage and evaluate its business and to make operating decisions, and compensation of executives is based in part on this measure.

Non-GAAP Income and its components are not meant to be considered in isolation, as a substitute for, or superior to comparable GAAP measures and should be read in conjunction with the consolidated financial information prepared in accordance with GAAP. Investors should note that the Non-GAAP information is not prepared under any comprehensive set of accounting rules or principles and does not reflect all of the amounts associated with the Company's results of operations as determined in accordance with GAAP. Investors should also note that these Non-GAAP measures have no standardized meaning prescribed by GAAP and, therefore, have limits in their usefulness to investors. In addition, from time to time in the future there may be other items that the Company may exclude for purposes of its Non-GAAP measures; likewise, the Company may in the future cease to exclude items that it has historically excluded for purposes of its Non-GAAP measures. Because of the non-standardized definitions, the Non-GAAP measure as used by BioMarin in this press release and the accompanying tables may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by other companies.

The following table presents the reconciliation of GAAP Net Loss to Non-GAAP Income:

Reconciliation of GAAP Net Loss to Non-GAAP Income

(In millions of U.S. dollars)

The following reconciliation of the GAAP reported to the Non-GAAP information provides the details of the effects of the Non-GAAP adjustments on certain components of the Company's operating results for each of the periods presented.

Reconciliation Of Certain GAAP Reported Information To Non-GAAP Information

(In millions of U.S. dollars)