BioMarin Announces Third Quarter 2013 Financial Results

BioMarin Announces Third Quarter 2013 Financial Results

 Total Revenue Grows 6.9% in the Quarter and 8.9% for First Three Quarters of
                                     2013

    Advisory Committee Meeting for Vimizim Scheduled for November 19, 2013

         Conference Call and Webcast to Be Held Today at 5:00 p.m. ET

Financial Highlights ($ in millions, except per share data, unaudited)

                                                  Q3 2013   Q3 2012   Percent
                                                                       Change
Total BioMarin Revenue                             $136.9  $128.1  6.9%
Total BioMarin Revenue (excluding Aldurazyme Net   134.0    124.0    8.1%
Product Transfer Revenue) - non-GAAP
Naglazyme Net Product Revenue                      63.2     62.5     1.1%
Aldurazyme BioMarin Net Product Revenue           23.4     23.8     -1.7%
Aldurazyme Royalty Revenue (excluding Net Product  20.5     19.7     4.1%
Transfer Revenue) - non-GAAP
Kuvan Net Product Revenue                          43.6     36.4     19.8%
Firdapse Net Product Revenue                       4.1      3.6      13.9%
Net Loss                                          (53.0)   (5.4)    
Net Loss per Share (basic and diluted)             $(0.38) $(0.04) 
Adjusted EBITDA Income (Loss) - non-GAAP           $(16.7) $11.1   
                                                                    
Cash, cash equivalents and short and long-term     $527.5  $533.2  -1.1%
investments

SAN RAFAEL, Calif., Oct. 24, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical
Inc. (Nasdaq:BMRN) today announced financial results for the third quarter
ended September 30, 2013. GAAP net loss was $53.0 million ($0.38 per diluted
share) for the third quarter of 2013, compared to GAAP net loss of $5.4
million ($0.04 per share) for the third quarter of 2012. GAAP net loss for the
nine months ended September 30, 2013 was $114.4 million ($0.84 per diluted
share), as compared to GAAP net loss of $61.3 million ($0.52 per diluted
share) for the nine months ended September 30, 2012.Non-GAAP adjusted EBITDA
loss was $16.7 million for the third quarter of 2013, compared to non-GAAP
adjusted EBITDA income of $11.1 million for the third quarter of
2012.Non-GAAP adjusted EBITDA was a loss of $24.6 million for the nine months
ended September 30, 2013, as compared to income of $3.9 million for the nine
months ended September 30, 2012.

The increased GAAP and non-GAAP adjusted EBITDA loss for the third quarter of
2013 compared to the third quarter of 2012 was primarily due to increased R&D
expenses from advancing our late stage clinical programs (PEG-PAL, BMN 673 and
BMN 701), from increased SG&A expenses, primarily due to increased
pre-commercialization expenses for Vimizim and commercialization expenses for
Naglazyme and Kuvan, and from increased contingent consideration expense also
due to progress on some of our acquired clinical programs (BMN 673 and BMN
701), partially offset by increased gross profit from total revenues. As of
September 30, 2013, BioMarin had cash, cash equivalents and short and
long-term investments totaling $527.5 million, as compared to $524.4 million
on June 30, 2013.

"During the third quarter we saw continued steady growth of our development
pipeline. We announced the advancement of two important programs, the decision
to advance BMN 673 for the treatment of gBRCA breast cancer into Phase 3 and
the initiation of a Phase 1/2 trial of BMN 190 for the treatment of Batten
Disease," said Jean-Jacques Bienaimé, Chief Executive Officer of
BioMarin."The next major inflection point for the company is the potential
market approval of Vimizim in the U.S. and Europe. We have been working
diligently to prepare for the launch of this drug and look forward to the
Advisory Committee meeting November 19 and potential CHMP opinion, which we
expect near the end of the year or early 2014."

Net Product Revenue



Total Revenue Growth, excluding Aldurazyme Net Product Transfer Revenues (in
millions)
                                                                   
             Three Months Ended September 30,  Nine Months Ended September 30,
             2013     2012     $ Change %       2013     2012     $       %
                                         Change                   Change  Change
                                                                   
Naglazyme ^   $63.2  $62.5  $0.7   1.1%    $202.5 $193.9 $8.6  4.4%
(1)
Kuvan         43.6     36.4     7.2     19.8%   122.1    103.1    19.0   18.4%
Firdapse      4.1      3.6      0.5     13.9%   11.8     10.8     1.0    9.3%
Aldurazyme    23.4     23.8     (0.4)   -1.7%   57.7     57.7     --    0.0%
Net product   134.3    126.3    8.0     6.3%    394.1    365.5    28.6   7.8%
revenue
                                                                   
Collaborative
agreement     1.8      1.2      0.6            2.8      1.7      1.1    
revenue
Royalty and
license       0.8      0.6      0.2            4.7      1.5      3.2    
revenue
Total
BioMarin      136.9    128.1    8.8     6.9%    401.6    368.7    32.9   8.9%
revenue -
GAAP
                                                                   
Less:
Aldurazyme
net product   2.9      4.1      (1.2)          -3.3     1.1      (4.4)  
transfer
revenue
Total
BioMarin
revenues
(excluding
Aldurazyme    $134.0 $124.0 10.0    8.1%    $404.9 $367.6 37.3   10.1%
net product
transfer
revenue) -
Non-GAAP ^
(2)
                                                                   
                                                                   
Reconciliation of Aldurazyme Revenues (in millions)
                                                                   
             Three Months Ended September 30,  Nine Months Ended September 30,
             2013     2012     $ Change %       2013     2012     $       %
                                         Change                   Change  Change
Aldurazyme
revenue       $50.8  $48.3  $2.5   5.2%    $152.9 $140.0 $12.9 9.2%
reported by
Genzyme
                                                                   
                                                                   
Aldurazyme
Royalties due
from Genzyme  $20.5  $19.7  $0.8          $61.0  $56.6  $4.4  
- Non-GAAP
^(2)
Incremental
net product   2.9     4.1     (1.2)          (3.3)   1.1     (4.4)  
transfer
revenue ^(3)
Total
Aldurazyme
net product   $23.4  $23.8  $(0.4)        $57.7  $57.7  --    
revenue -
GAAP
                                                                   
(1) Naglazyme revenues experience quarterly fluctuations due to the timing of
government ordering patterns in certain countries.For example, quarterly sales to
Brazil have been between $11M--$17M for Q1 2012 through Q2 2013.In each of these
quarters there has been a centralized Brazil Ministry of Health (MOH) order for
more than 50% of the Brazil ordering. However, in Q3 2013 there was no large,
centralized order from the Brazilian MOH.As a result, sales in Brazil were less
than $6M in Q3 2013.The Company does not believe this reflects a change in
underlying demand, simply the timing of the Brazil MOH order. In Q4 2013, the
Company received a centralized order from the Brazilian MOH.
(2) BioMarin believes that this non-GAAP information is useful to investors, taken
in conjunction with BioMarin's GAAP information because it provides additional
information regarding the performance of BioMarin's core business. By providing
information about both the GAAP and non-GAAP revenue measures, the company
believes that the additional information enhances investors' overall understanding
of the company's business and in particular allows for more consistent period to
period evaluation of the revenue.
(3) To the extent units shipped to third party customers by Genzyme exceed
BioMarin inventory transfers to Genzyme, BioMarin will record a decrease in net
product revenue from the royalty payable to BioMarin for the amount of previously
recognized product transfer revenue.If BioMarin inventory transfers exceed units
shipped to third party customers by Genzyme, BioMarin will record incremental net
product transfer revenue for the period.

2013 Full Year Financial Guidance

Unchanged with the exception of cash balance which reflects lower cash burn in
2013 and net proceeds of $696.6 million from the Convertible Debt offering
that closed on October 15, 2013.

Revenue Guidance ($ in millions)                   
Item                                               2013 Guidance
Total BioMarin Revenues                            $530 to $555
Naglazyme Net Product Revenue                      $265 to $285
Kuvan Net Product Revenue                          $155 to $170
                                                  
Selected Income Statement Guidance ($ in millions) 
Item                                               2013 Guidance
Cost of Sales (% of Total Revenue)                 17% to 18%
Selling, General and Admin. Expense                $220 to $240
Research and Development Expense                   $340 to $380
GAAP Net Loss                                      $(185) to $(160)
Non-GAAP Adjusted EBITDA (loss)                    $(65) to $(40)
                                                  
Cash Balance*                                      Over $1,180

* Cash balance includes cash, cash equivalents and short and long term
investments

Anticipated Upcoming Milestones

4Q 2013: First patient in Phase 3 with BMN 673 for the treatment of gBRCA
breast cancer
4Q 2013/1Q 2014: Initiation of Phase 2/3 switching trial for BMN 701 for Pompe
disease
4Q 2013/1Q 2014: Potential CHMP opinion for Vimizim for Morquio A
4Q 2013/ 1Q 2014: Initiation of Phase 1/2 trial for BMN 111 for achondroplasia
1Q 2014: PDUFA date for Vimizim for Morquio A
1Q 2014: Potential launch of Vimizim for Morquio A
4Q 2014: Top-line results for Phase 3 trial for PEG-PAL for PKU

Research and Development Programs

BioMarin continues to make significant investments in research and development
to ensure a strong and profitable pipeline for the company.The current
pipeline includes programs in various stages of development that focus on
treating a range of rare and serious unmet medical needs.

Programs Under Regulatory Review for Approval

  *Vimizim for Morquio A: Regulatory applications for marketing authorization
    for Vimizim are under active review.The company has an FDA Advisory
    Committee meeting scheduled for November 19, 2013.The FDA has granted
    Vimizim priority review designation and assigned a PDUFA date of February
    28, 2014.The EMA recently changed the review status for Vimizim from
    accelerated to standard assessment, which implies a possible CHMP opinion
    in either late 2013 or early 2014. Both the FDA and EMA have conducted
    and completed all pre-approval inspections associated with the review of
    the marketing applications.

Advanced Clinical Programs

  *PEG-PAL for PKU:Enrollment in the pivotal Phase 3 program, which opened
    in the second quarter of 2013, is progressing.The Phase 3 program
    includes: (1) an open-label Phase 3 study to evaluate safety and blood Phe
    levels in naïve patients; and, (2) a randomized controlled study of the
    Phase 2 extension study patients and patients from the open label trial to
    evaluate blood Phe levels and neurocognitive endpoints. The company
    expects top-line results for the Phase 3 study in the fourth quarter of
    2014.
    
  *BMN 701 for Pompe Disease:During the third quarter, regulatory
    authorities indicated that Maximal Inspiratory Pressure ("MIP") is a
    potentially approvable primary endpoint and that a single arm switchstudy
    could support registration if the magnitude of benefit is meaningful and
    if the data can satisfy health authorities that patient outcomes can be
    reliably predicted.A phase 2/3 trial of patients previously treated with
    alglucosidase alfa and switched to atreatment of BMN 701 at 20 mg/kg
    administered every other week for 24 weeks is being prepared to start in
    Q4 2013/Q1 2014. The primary endpoint of the study will be the respiratory
    parameter MIP.The company has completed a full scale production campaign
    using its new cell line and has verified the production
    yields.Characterization testing is currently ongoing, and it is expected
    that regulatory filings to support the introduction of this material into
    clinical studies will be submitted in the fourth quarter of 2013.
    
  *BMN 673 (PARP inhibitor):The Phase 3 trial to study its poly ADP-ribose
    polymerase (PARP) inhibitor, BMN 673, in the treatment of deleterious
    germline BRCA mutation metastatic breast cancer is open for
    enrollment.The Phase 3 trial is an open-label, 2:1 randomized, parallel,
    two-arm study of BMN 673 as compared to monotherapy physicians' choice
    (capecitabine, eribulin, gemcitabine or vinorelbine) in germline BRCA
    mutation subjects with locally advanced and/or metastatic breast cancer
    who have received no more than two prior chemotherapy regimens for
    metastatic disease.The study will enroll approximately 429 subjects and
    will be conducted globally.The primary objective of the study is to
    compare progression-free survival (PFS) of subjects treated with BMN 673
    as a monotherapy relative to those treated with protocol-specific
    physicians' choice.The company also announced that it will use Myriad
    Genetics' BRCAnalysis® test as a diagnostic in the pivotal trial.

Early-Stage Clinical Programs

  *BMN 111 for Achondroplasia: The company announced that U.S. and European
    Regulatory Authorities have agreed that a Phase 2 study in Achondroplasia
    can start without additional data.BioMarin previously completed a Phase 1
    study in adult healthy volunteers.The company expects to initiate its
    first study in pediatric patients in the fourth quarter of 2013 or the
    first quarter of 2014.
    
  *BMN 190 for LINCL (Batten disease): The company announced that the first
    patient had been dosed in the Phase 1/2 trial for BMN 190, a recombinant
    human tripeptidyl peptidase 1 (rhTPP1) for the treatment of patients with
    neuronal ceroid lipofuscinosis type 2 (NCL-2), a form of Batten
    disease.This is the first time that a patient with Batten Disease has
    been treated with an enzyme replacement therapy in a clinical trial
    setting.The Phase 1/2 study is an open-label, dose-escalation study in
    patients with NCL-2.The primary objectives are to evaluate the safety and
    tolerability of BMN 190 and to evaluate effectiveness using an
    NCL-2-specific rating scale score in comparison with natural history data
    after 48 weeks of treatment.Secondary objectives are to evaluate the
    impact of treatment on brain atrophy in comparison with NCL-2 natural
    history after 48 weeks of treatment and to characterize pharmacokinetics
    and immunogenicity.The study will enroll approximately 22 subjects for a
    treatment duration of 48 weeks.

Preclinical Programs

  *Other early stage programs: BioMarin is working on multiple additional
    early development opportunities, including two new lead optimization
    programs gained through the acquisition of Zacharon Pharmaceuticals:
    inhibition of heparan sulfate synthesis for MPS III and inhibition of
    ganglioside synthesis for diseases such as Tay Sachs and Sandhoff.The
    company also has an ongoing Factor VIII gene therapy research program for
    Hemophilia A from University College London and St. Jude Children's
    Research Hospital.

Financing Update

  *Convertible Debt Offering: On October 15, 2013, the company completed a
    Convertible Debt Offering of $750.0 million of its senior subordinated
    convertible notes consisting of $375.0 million 0.75% Senior Subordinated
    Convertible Notes due 2018 (the "2018 Notes") and $375.0 million 1.50%
    Senior Subordinated Convertible Notes due 2020 (the "2020 Notes" and
    together with the 2018 Notes, the "Notes").

  The Notes will be convertible, under certain circumstances, into cash,
  shares of BioMarin's common stock or a combination of cash and common stock
  at BioMarin's election. The initial conversion rate will be 10.6213 shares
  of common stock per $1,000 principal amount of Notes (representing an
  initial conversion price of approximately $94.15 per common share), subject
  to customary adjustments. The initial conversion rate represents
  approximately a 40% premium to the last reported sale price of the common
  stock on the NASDAQ Global Select Market on October 8, 2013.

  The company entered into privately-negotiated capped call transactions with
  respect to 50% of the principal amount of the Notes with three of the
  underwriters or their affiliates (the "hedge counterparties"). The capped
  call transactions are generally expected to reduce potential dilution to
  BioMarin's common stock upon conversion of the relevant Notes in excess of
  the principal amount of such converted Notes. The cap price of the capped
  call transactions entered into with respect to 50% of the Notes will
  initially be, in each case, approximately $121.05, which represents a
  premium of approximately 80% over the NASDAQ closing price of a share of
  BioMarin's common stock on October 8, 2013 and is subject to certain
  adjustments under the terms of such capped call transactions.

  The company received net proceeds after fees, transaction costs and the
  purchase of the capped call of approximately $696.6 million, which the
  company intends to use for general corporate purposes.

  In order to preserve flexibility and to potentially further minimize future
  dilution, on conversion, the Notes may be settled in cash, shares of
  BioMarin's common stock or a combination of cash and common stock at
  BioMarin's election.Under GAAP, convertible debt instruments that may be
  settled in cash are required to be accounted for by separating the
  instrument into separate debt and equity components based on our
  non-convertible debt borrowing rate.For GAAP purposes the equity component
  is treated as a discount to the notes, and this discount is amortized to
  interest expense using the effective interest method over the life of the
  notes as the they accrete to face value at maturity.This estimated
  additional GAAP interest expense does not have a current, past or future
  impact on cash flows.The table below shows the estimated future interest
  expense and associated GAAP tax benefits for the Notes due to the cash
  interest coupon payments and the cash issuance costs as well as the non cash
  debt discount accretion.

Estimated Convertible Note Interest Expense and Estimated Tax Benefits ($ in millions)
                                                                           
              For the Year Ended
              2013     2014      2015      2016      2017      2018      2019     2020
                                                                           
Coupon
Interest       $1.4   $8.4    $8.4    $8.4    $8.4    $8.0    $5.6   $4.7
(Cash)
Amortization
of Issuance    0.5     3.2      3.2      3.2      3.2      2.9      1.3     1.1
Costs (Cash)
Amortization
of Debt        3.9     23.8     25.1     26.5     28.0     26.8     14.5    12.7
Discount
(Non-Cash)
Total
Estimated GAAP $5.8   $35.5   $36.8   $38.1   $39.6   $37.6   $21.4  $18.5
Interest
Expense
                                                                           
Total
Estimated GAAP $(2.1) $(12.8) $(13.3) $(13.8) $(14.3) $(13.6) $(7.7) $(6.7)
Tax Benefit
                                                                           
Estimated
After Tax
Interest
Expense of     $1.2   7.4      7.4      7.4      7.4      6.9      4.4     3.7
Cash
Items(Coupon
+ Issuance
Costs)
Estimated
After Tax
Interest
Expense of     2.5     15.2     16.1     16.9     17.9     17.1     9.2     8.1
Non-CashItems
(Discount
Amortization)
Total
Estimated
After Tax GAAP $3.7   $22.7   $23.5   $24.4   $25.3   $24.1   $13.7  $11.8
Net Interest
Expense

Non-GAAP Financial Information and Reconciliation

The results for the three and nine months ended September 30, 2013 and
September 30, 2012 and financial guidance for the year ending December 31,
2013 are all determined in accordance with GAAP except for non-GAAP adjusted
EBITDA which is determined on a non-GAAP basis. As used in this release,
non-GAAP adjusted EBITDA income is based on GAAP earnings before interest,
taxes, depreciation and amortization (EBITDA) and further adjusted to also
exclude certain non-cash stock compensation expense, non-cash contingent
consideration expense and certain other nonrecurring material items (non-GAAP
adjusted EBITDA).

Reconciliation of GAAP Net Loss to Non-GAAP Adjusted EBITDA Income (Loss)
(in millions)
(unaudited)

                    ThreeMonths            NineMonths            Year Ending
                   EndedSeptember 30,    EndedSeptember 30,    December
                                                                   31, 2013
                   2013         2012       2013        2012       Guidance
                                                              
GAAP Net Loss       $(53.0)    $(5.4)   $(114.4)  $(61.3)  $(185.0) -
                                                                   $(160.0)
                                                              
Interest expense,   --         1.1       1.0        3.9       6.2
net
Provision for
(benefit from)      0.7         (6.4)     (2.8)      (6.8)     (7.2)
income taxes
Depreciation        6.3         6.4       19.0       21.0      25.0
expense
Amortization        2.6         2.7       8.9        14.5      10.5
expense
EBITDA Loss         (43.4)      (1.6)     (88.3)     (28.7)    (150.5) -
                                                                   (125.5)
                                                              
Stock-based
compensation        16.2        12.1      41.7       35.9      59.5
expense
Contingent
consideration       8.8         0.6       9.8        (3.3)     13.8
expense ^ (1)
Material                                                       
non-recurring:
Debt conversion     1.7         --       12.2       --       12.2
expense ^(2)
                                                              
Non-GAAP Adjusted                                                  $(65.0) -
EBITDA Income       $(16.7)    $11.1    $(24.6)   $3.9     $(40.0)
(Loss)
                                                              
^(1) Represents the expense associated with the change in the fair value of
contingent acquisition consideration payable for the period. The change in the
current quarter reflects changes in estimated probabilities and timing of
achieving certain developmental milestones.
^(2) Represents debt conversion expense associated with the early conversion
of a portion of our 2017 convertible notes in March and August 2013

BioMarin believes that this non-GAAP information is useful to investors, taken
in conjunction with BioMarin's GAAP information because it provides additional
information regarding the performance of BioMarin's core ongoing business,
Naglazyme, Kuvan, Aldurazyme and Firdapse and development of its pipeline.By
providing information about both the overall GAAP financial performance and
the non-GAAP measures that focus on continuing operations, the company
believes that the additional information enhances investors' overall
understanding of the company's business and prospects for the future.Further,
the company uses both the GAAP and the non-GAAP results and expectations
internally for its operating, budgeting and financial planning purposes and
uses the non-GAAP adjusted EBITDA methodology in establishing corporate goals
for internal compensation programs.

Conference Call Details

BioMarin will host a conference call and webcast to discuss third quarter 2013
financial results today, Thursday, October 24, at 5:00 p.m. ET. This event can
be accessed on the investor section of the BioMarin website at www.BMRN.com.

U.S. / Canada Dial-in Number: 877.303.6313
International Dial-in Number: 631.813.4734
Conference ID: 32118292

Replay Dial-in Number: 855.859.2056
Replay International Dial-in Number: 404.537.3406
Conference ID: 32118292

About BioMarin

BioMarin develops and commercializes innovative biopharmaceuticals for serious
diseases and medical conditions. The company's product portfolio comprises
four approved products and multiple clinical and pre-clinical product
candidates. Approved products include Naglazyme^® (galsulfase) for
mucopolysaccharidosis VI (MPS VI), a product wholly developed and
commercialized by BioMarin; Aldurazyme^® (laronidase) for
mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a
50/50 joint venture with Genzyme Corporation; Kuvan^® (sapropterin
dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership
with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; and
Firdapse™ (amifampridine), which has been approved by the European Commission
for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Product
candidates include GALNS (N-acetylgalactosamine 6-sulfatase), which is
currently in Phase III clinical development for the treatment of MPS IVA,
amifampridine phosphate (3,4-diaminopyridine phosphate), which is currently in
Phase III clinical development for the treatment of LEMS in the U.S., PEG-PAL
(PEGylated recombinant phenylalanine ammonia lyase), which is currently in
Phase II clinical development for the treatment of PKU, BMN-701, a novel
fusion protein of insulin-like growth factor 2 and acid alpha glucosidase
(IGF2-GAA), which is currently in Phase I/II clinical development for the
treatment of Pompe disease, BMN-673, a poly ADP-ribose polymerase (PARP)
inhibitor, which is currently in Phase I/II clinical development for the
treatment of genetically-defined cancers, and BMN-111, a modified
C-nutriuretic peptide, which is currently in Phase I clinical development for
the treatment of achondroplasia. For additional information, please visit
www.BMRN.com. Information on BioMarin's website is not incorporated by
reference into this press release.

Forward-Looking Statement

This press release contains forward-looking statements about the business
prospects of BioMarin Pharmaceutical Inc., including, without limitation,
statements about: the expectations of revenue and sales related to Naglazyme,
Kuvan, Firdapse, and Aldurazyme; the financial performance of the BioMarin as
a whole; the timing of BioMarin's clinical trials of GALNS, PEG-PAL, BMN-673,
BMN-701, BMN-111, BMN-190 and other product candidates; the continued clinical
development and commercialization of Aldurazyme, Naglazyme, Kuvan, Firdapse,
and its product candidates; and actions by regulatory authorities. 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: our success in the continued
commercialization of Naglazyme, Kuvan, and Firdapse; Genzyme Corporation's
success in continuing the commercialization of Aldurazyme; results and timing
of current and planned preclinical studies and clinical trials, particularly
with respect to GALNS, PEG-PAL, BMN-673, BMN-701, BMN-111 and BMN-190; our
ability to successfully manufacture our products and product candidates; the
content and timing of decisions by the U.S. Food and Drug Administration, the
European Commission and other regulatory authorities concerning each of the
described products and product candidates; the market for each of these
products and particularly Aldurazyme, Naglazyme, Kuvan and Firdapse; actual
sales of Aldurazyme, Naglazyme Kuvan and Firdapse; Merck Serono's activities
related to Kuvan; and those factors detailed in BioMarin's filings with the
Securities and Exchange Commission, including, without limitation, the factors
contained under the caption "Risk Factors" in BioMarin's 2012 Annual Report on
Form 10-K, and the factors contained in BioMarin's reports on Form 10-Q.
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^®, Naglazyme^®, Kuvan^® and Firdapse™ 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
September 30, 2013 and December 31, 2012
(In thousands of U.S. dollars, except share and per share amounts)
                                                                
                                                  September 30, December 31,
                                                  2013           2012^(1)
ASSETS                                             (unaudited)   
Current assets:                                                  
Cash and cash equivalents                          $181,565     $180,527
Short-term investments                             198,086       270,211
Accounts receivable, net (allowance for doubtful   124,745       109,066
accounts: $376 and $348, respectively)
Inventory                                          148,684       128,695
Current deferred tax assets                        32,238        29,454
Other current assets                               28,161        25,509
Total current assets                              713,479       743,462
Noncurrent assets:                                               
Investment in BioMarin/Genzyme LLC                 854           1,080
Long-term investments                              147,771       115,993
Property, plant and equipment, net                 285,664       284,473
Intangible assets, net                             165,791       162,980
Goodwill                                           54,258        51,543
Long-term deferred tax assets                      238,703       225,501
Other assets                                       $14,010      16,611
Total assets                                      $1,620,530   $1,601,643
LIABILITIES AND STOCKHOLDERS' EQUITY                             
Current liabilities:                                             
Accounts payable and accrued liabilities           $167,633     $147,068
Convertible debt                                   --           23,365
Total current liabilities                          167,633       170,433
Noncurrent liabilities:                                          
Long-term convertible debt                         78,310        324,859
Long-term contingent acquisition consideration     26,500        30,618
payable
Long-term deferred tax liabilities                 37,190        33,296
Other long-term liabilities                        34,411        26,674
Total liabilities                                  344,044       585,880
Stockholders' equity:                                            
Common stock, $0.001 par value: 250,000,000 shares
authorized at September 30, 2013 and December 31,
2012; 142,200,995 and 125,809,162 shares issued    142           126
and outstanding at September 30, 2013 and December
31, 2012, respectively.
Additional paid-in capital                         1,926,133     1,561,890
Company common stock held by Nonqualified Deferred (7,451)       (6,603)
Compensation Plan
Accumulated other comprehensive income (loss)      11,473        (202)
Accumulated deficit                                (653,811)     (539,448)
Total stockholders' equity                         1,276,486     1,015,763
Total liabilities and stockholders' equity         $1,620,530   $1,601,643
                                                                
^(1) December 31, 2012 balances were derived from the audited consolidated
financial statements.

BIOMARIN PHARMACEUTICAL INC.
                                                              
CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS
Three and Nine Months Ended September 30, 2013 and 2012
(In thousands of U.S. dollars, except per share amounts)
(Unaudited)
                                                              
                         Three Months Ended        Nine Months Ended
                          September 30,             September 30,
                         2013          2012        2013          2012
REVENUES:                                                      
Net product revenues      $134,330    $126,310  $394,074    $365,540
Collaborative agreement   1,754        1,210      2,778        1,729
revenues
Royalty and license       790          597        4,760        1,516
revenues
Total revenues            136,874      128,117    401,612      368,785
OPERATING EXPENSES:                                            
Cost of sales (excludes
amortization of certain   28,054       24,619     71,121       65,298
acquired intangible
assets)
Research and development  88,064       66,209     257,468      217,855
Selling, general and      61,841       46,337     163,547      143,124
administrative
Intangible asset
amortization and          9,639        1,443      13,173       5,819
contingent consideration
Total operating expenses  187,598      138,608    505,309      432,096
LOSS FROM OPERATIONS      (50,724)     (10,491)   (103,697)    (63,311)
Equity in the loss of     (147)        (336)      (711)        (968)
BioMarin/Genzyme LLC
Interest income           574          778        1,942        1,819
Interest expense          (526)        (1,837)    (2,854)      (5,709)
Debt conversion expense   (1,732)      --        (12,152)     --
Other income (expense)    239          125        344          (15)
LOSS BEFORE INCOME TAXES  (52,316)     (11,761)   (117,128)    (68,184)
Provision for (benefit    704          (6,404)    (2,765)      (6,849)
from) income taxes
NET LOSS                  $(53,020)   $(5,357)  $(114,363)  $(61,335)
NET LOSS PER SHARE, BASIC $(0.38)     $(0.04)   $(0.84)     $(0.52)
AND DILUTED
                                                              
Weighted average common
shares outstanding, basic 140,796       123,434     136,102       118,810
and diluted
                                                              
COMPREHENSIVE LOSS       $(43,988)   $(7,674)  $(102,688)  $(63,889)
                                                              
                                                              
STOCK-BASED COMPENSATION EXPENSE
Total stock-based compensation expense included in the Condensed Consolidated
Statements of Comprehensive Loss is as follows (unaudited):
                                                              
                         Three Months Ended        Nine Months Ended
                          September 30,             September 30,
                         2013          2012        2013          2012
                                                              
Cost of Sales             $1,489      $1,327    $3,663      $3,535
Research and development  7,116         5,060      18,821       15,351
Selling, general and      7,600         5,752      19,214       17,021
administrative
                         $16,205     $12,139   $41,698     $35,907

CONTACT: Investors
         Traci McCarty
         BioMarin Pharmaceutical Inc.
         (415) 455-7558
        
         Media
         Debra Charlesworth
         BioMarin Pharmaceutical Inc.
         (415) 455-7451

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