The following discussion of our financial condition and results of operations
should be read in conjunction with our Condensed Consolidated Financial
Statements and the related Notes thereto included in this Quarterly Report on
Form 10-Q. This discussion contains forward-looking statements that involve
risks and uncertainties. When reviewing the discussion below, you should keep in
mind the substantial risks and uncertainties that could impact our business. In
particular, we encourage you to review the risks and uncertainties described in
"Risk Factors" in Part II, Item 1A in this Quarterly Report on Form 10-Q. These
risks and uncertainties could cause actual results to differ significantly from
those projected in forward-looking statements contained in this report or
implied by past results and trends. Forward-looking statements are statements
that attempt to forecast or anticipate future developments in our business,
financial condition or results of operations. See the section titled
"Forward-Looking Statements" that appears at the beginning of this Quarterly
Report on Form 10-Q. These statements, like all statements in this report, speak
only as of the date of this Quarterly Report on Form 10-Q (unless another date
is indicated), and, except as required by law, we undertake no obligation to
update or revise these statements in light of future developments. Our Condensed
Consolidated Financial Statements have been prepared in accordance with United
States (U.S.) generally accepted accounting principles (U.S. GAAP) and are
presented in U.S. Dollars (USD).


                                       19

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   Management's Discussion and Analysis of Financial Condition and Results of
                             Operations (continued)
                  (In millions, except as otherwise disclosed)

Overview

Founded in 1997, we are a global biotechnology company dedicated to transforming
lives through genetic discovery. We develop and commercialize targeted therapies
that address the root cause of genetic conditions. Our robust research and
development capabilities have resulted in multiple innovative commercial
therapies for patients with rare genetic disorders. Our distinctive approach to
drug discovery has produced a diverse pipeline of commercial, clinical, and
pre-clinical candidates that address a significant unmet medical need, have
well-understood biology, and provide an opportunity to be first-to-market or
offer a substantial benefit over existing treatment options. A summary of our
commercial products, as of March 31, 2023, is provided below:

Commercial Products                                  Indication
Enzyme products:
VIMIZIM (elosulfase alpha)                      MPS (1) IVA
NAGLAZYME (galsulfase)                          MPS VI (2)
PALYNZIQ (pegvaliase-pqpz)                      PKU (3)
BRINEURA (cerliponase alfa)                     CLN2 (4)
ALDURAZYME (laronidase)                         MPS I (5)
Other products:
VOXZOGO (vosoritide)                            Achondroplasia (6)

ROCTAVIAN (valoctocogene roxaparvovec) (7) Severe Hemophilia A KUVAN (sapropterin dihydrochloride)

             PKU


(1)For the treatment of Mucopolysaccharidosis IV Type A

(2)For the treatment of MPS VI

(3)For the treatment of adult patients with phenylketonuria (PKU)

(4)For the treatment of neuronal ceroid lipofuscinosis type 2

(5)For the treatment of MPS I. ALDURAZYME is marketed worldwide by Sanofi



(6)For the treatment of achondroplasia in children aged five years and older for
the United States (U.S.), aged two years and older for the European Union (EU)
and for various age ranges for other markets

(7)ROCTAVIAN (formerly known as valoctocogene roxaparvovec) for the treatment of adults with severe hemophilia A was conditionally approved by the European Commission (EC) in August 2022



A summary of our on-going clinical development programs, as of March 31, 2023,
is provided below:

                                                                     Target
Clinical Development Programs                                      Indication                                             Stage
ROCTAVIAN                                             Severe Hemophilia A                                       FDA (1) regulatory review

BMN 255                                               Hyperoxaluria                                                Clinical Phase 1/2
BMN 331                                               Hereditary Angioedema (HAE)                                  Clinical Phase 1/2

(1)U.S. Food and Drug Administration


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Management's Discussion and Analysis of Financial Condition and Results of


                             Operations (continued)
                  (In millions, except as otherwise disclosed)

Financial Highlights

Key components of our results of operations include the following:



                                                                Three Months Ended
                                                                    March 31,
                                                                               2023          2022
Total revenues                                                               $ 596.4      $  519.4
Cost of sales                                                                $ 126.5      $  117.0
Research and development (R&D) expense                                       $ 171.8      $  160.8
Selling, general and administrative (SG&A) expense                          

$ 223.0 $ 194.6



Gain on sale of nonfinancial assets, net                                     $     -      $ (108.0)

Net income                                                                   $  50.9      $  120.8

See " Results of Operations " below for discussion of our results for the periods presented.

Uncertainty Relating to Macroeconomic Environment



Conditions in the current macroeconomic environment, such as inflation, changes
in interest and foreign currency exchange rates, banking crises, natural
disasters, the continuing effects of the COVID-19 pandemic, and supply chain
disruptions, could impact our global revenue sources and our overall business
operations. The extent and duration of such effects remain uncertain and
difficult to predict. We are actively monitoring and managing our response and
assessing actual and potential impacts to our operating results and financial
condition, as well as developments in our business, which could further impact
the developments, trends and expectations described below. See the risk factor,
"Our business is affected by macroeconomic conditions." described in "  Risk
Factors  " in Part II, Item 1A of this Quarterly Report on Form 10-Q.

Business Developments



We continued to grow our commercial business and advance our product candidate
pipeline during 2023. We believe that the combination of our internal research
programs, acquisitions and partnerships will allow us to continue to develop and
commercialize innovative therapies for people with serious and life-threatening
rare diseases and medical conditions. Below is a summary of key business
developments:

Continued Emphasis on Research and Development

Product Launches, Approvals and Mid-stage Product Life Cycle Expansion Opportunities



•ROCTAVIAN: We are working to finalize reimbursement and access with German,
French and Italian health insurers. At present, people in Germany with severe
hemophilia A, who are eligible for treatment, can access treatment under either
Named Patient authorizations or previously secured Outcomes Based Agreements.

In March 2023, the FDA determined that the submission of the three-year data
analysis from the ongoing Phase 3 GENEr8-1 study, as requested by the FDA,
constituted a Major Amendment to our Biologics License Application (BLA) for
ROCTAVIAN gene therapy for adults with severe hemophilia A, due to the
substantial amount of additional data and set a new Prescription Drug User Fee
Act (PDUFA) Target Action Date of June 30, 2023.

ROCTAVIAN product expansion opportunities continue, including a clinical study
investigating ROCTAVIAN treatment in those with active or prior inhibitors and
continued exploration of methods of administering ROCTAVIAN in people with
pre-existing antibodies against AAV5.

•VOXZOGO: VOXZOGO is currently approved for the treatment of children two years
old and older in Europe, for children five years old and older in the U.S., and
for all ages from birth in Japan. During the first quarter of 2023, the global
expansion continued, with market access and reimbursement activities
progressing.

In the coming months, we expect to learn the outcome of our request to expand
VOXZOGO access to younger age groups based on favorable results from a Phase 2
study in infants and young children. If age expansions are accepted, this will
increase the number of children eligible for VOXZOGO treatment in the U.S. and
Europe. We are also engaged in discussions with health authorities concerning
the opportunity to leverage VOXZOGO, a natural regulator of bone growth, in
other conditions characterized by impaired bone growth.
                                       21

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Management's Discussion and Analysis of Financial Condition and Results of


                             Operations (continued)
                  (In millions, except as otherwise disclosed)

Select Earlier-stage Development Portfolio



•BMN 255 for hyperoxaluria in chronic liver disease: we have concluded the
multi-ascending dose study in healthy human volunteers. In January 2023, we
shared early data that demonstrated a rapid and potent increase in plasma
glycolate following treatment with BMN 255. Oral daily dosing at all tested
levels for 14 days demonstrated a favorable safety profile and showed sustained
elevations of plasma glycolate, which is predicted to have a profound reduction
in oxalate excretion in patients. We plan to initiate and enroll an expanded
study in patients with chronic liver disease and hyperoxaluria in 2023. We
believe the availability of a potent, orally bioavailable, small molecule like
BMN 255 may be able to significantly reduce disease and treatment burden in a
patient population with significant unmet need.

•BMN 331 gene therapy product candidate for Hereditary Angioedema (HAE): Dosing
continues in the Phase 1/2 HAERMONY study to evaluate BMN 331, an
investigational AAV5-mediated gene therapy for people living with HAE. In
January 2023, we shared that the first participant treated with the 6e13vg/kg
dose demonstrated C1-Inhibitor levels that were approaching the therapeutically
relevant range. In March 2023, the second sentinel participant was dosed at
6e13vg/kg.

Critical Accounting Estimates



In preparing our Condensed Consolidated Financial Statements in accordance with
U.S. GAAP and pursuant to the rules and regulations promulgated by the
Securities and Exchange Commission (the SEC), we make assumptions, judgments and
estimates that can have a significant impact on our net income/loss and affect
the reported amounts of certain assets, liabilities, revenues and expenses, and
related disclosures. On an ongoing basis, we evaluate our estimates and discuss
our critical accounting policies and estimates with the Audit Committee of our
Board of Directors. We base our estimates on historical experience and various
other assumptions that we believe to be reasonable under the circumstances.
Actual results could differ materially from these estimates under different
assumptions or conditions. Historically, our assumptions, judgments and
estimates relative to our critical accounting estimates have not differed
materially from actual results.

There have been no significant changes to our critical accounting estimates during the three months ended March 31, 2023, compared to those disclosed in "Management's Discussion and Analysis of Financial Condition and Results of Operations" included in our Annual Report on Form 10-K for the year ended December 31, 2022, filed with the SEC on February 27, 2023.

Recent Accounting Pronouncements

See Note 1 to our accompanying Condensed Consolidated Financial Statements for a description of recent accounting pronouncements, if any, and our expectation of their impact on our results of operations and financial condition.


                                       22

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Management's Discussion and Analysis of Financial Condition and Results of


                             Operations (continued)
          (In millions of U.S. dollars, except as otherwise disclosed)

Results of Operations

Net Product Revenues

Net Product Revenues consisted of the following:



                                              Three Months Ended
                                                  March 31,
                                                                2023         2022        Change
Enzyme product revenues:
VIMIZIM                                                       $ 189.2      $ 183.0      $  6.2
NAGLAZYME                                                       123.0        128.0        (5.0)
PALYNZIQ                                                         62.4         54.9         7.5
BRINEURA                                                         39.1         36.2         2.9

ALDURAZYME                                                       34.4         24.4        10.0
Total enzyme product revenues                                 $ 448.1      $ 426.5      $ 21.6

VOXZOGO                                                          87.8         19.7        68.1
KUVAN                                                            50.5         59.3        (8.8)
Total net product revenues                                    $ 586.4      $ 505.5      $ 80.9




Net Product Revenues include revenues generated from our approved products. In
the U.S., our commercial products, except for PALYNZIQ and ALDURAZYME, are
generally sold to specialty pharmacies or end-users, such as hospitals, which
act as retailers. PALYNZIQ is distributed in the U.S. through certain certified
specialty pharmacies under the PALYNZIQ Risk Evaluation and Mitigation Strategy
(REMS) program, and ALDURAZYME is marketed worldwide by Sanofi. Outside the
U.S., our commercial products are sold to authorized distributors or directly to
government purchasers or hospitals, which act as the end users.

The increase in Net Product Revenues for the three months ended March 31, 2023
as compared to the three months ended March 31, 2022 was primarily attributed to
the following:

•VOXZOGO: continued global market expansion and new patients initiating therapy following regulatory approvals, particularly in Japan and Europe;

•ALDURAZYME: the timing of order fulfillment to Sanofi;

•PALYNZIQ: new patients initiating therapy in the U.S. and Europe;

•VIMIZIM: new patients initiating therapy and the timing of orders in countries that place large government orders, particularly in Europe; partially offset by

•KUVAN: lower sales primarily attributed to continued market erosion due to generic competition.



In certain countries, governments place large periodic orders for our products.
We expect that the timing of these large government orders will continue to be
inconsistent, which may create significant period to period variation in our
revenues.

See the risk factor "The sale of generic versions of KUVAN by generic
manufacturers has adversely affected and will continue to adversely affect our
revenues and may cause a decline in KUVAN revenues faster than expected"
included in Part II, Item 1A of this Quarterly Report for additional information
on risks we face.
                                       23

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Management's Discussion and Analysis of Financial Condition and Results of


                             Operations (continued)
          (In millions of U.S. dollars, except as otherwise disclosed)

We face exposure to movements in foreign currency exchange rates, primarily the
Euro, which we expect to continue in future periods. We use foreign currency
exchange forward contracts to hedge a percentage of our foreign currency
exposure. The following table shows our Net Product Revenues denominated in USD
and foreign currencies:

                                                       Three Months Ended
                                                           March 31,
                                                                         2023         2022        Change
Sales denominated in USD                                               $ 297.6      $ 259.3      $ 38.3
Sales denominated in foreign currencies                                  288.8        246.2        42.6
Total net product revenues                                             $ 586.4      $ 505.5      $ 80.9


                                                                     Three Months Ended
                                                                         March 31,
                                                                                  2023             2022            Change

Unfavorable impact of foreign currency exchange rates on product sales denominated in currencies other than USD

$ (28.3) $ (9.0) $ (19.3)

The unfavorable impact for the three months ended March 31, 2023 as compared to the three months ended March 31, 2022 was primarily driven by weakening of Argentine Peso, Euro and Japanese Yen.

Royalty and Other Revenues



Royalty and Other Revenues include royalties earned on net sales of products
sold by third parties, up-front licensing fees, milestones achieved by licensees
or sublicensees and rental income associated with the tenants in our facilities.

                                           Three Months Ended
                                               March 31,
                                                              2023        2022       Change
Royalty and other revenues                                  $ 10.0      $ 13.8      $ (3.8)


The decrease in Royalty and Other Revenues for the three months ended March 31,
2023 as compared to the three months ended March 31, 2022 was primarily due to
lower royalty revenues earned from third parties.

We expect to continue to earn royalties from third parties in the future.

Cost of Sales and Gross Margin



Cost of Sales includes raw materials, personnel and facility and other costs
associated with manufacturing our commercial products. These costs include
production materials, production costs at our manufacturing facilities,
third-party manufacturing costs, amortization of technology transfer intangible
assets and internal and external final formulation and packaging costs. Cost of
Sales also includes royalties payable to third parties based on sales of our
products and charges for inventory valuation reserves.

The following table summarizes our Cost of Sales and gross margin:



                               Three Months Ended
                                   March 31,
                                                 2023          2022        Change
Total revenues                                $ 596.4       $ 519.4       $ 77.0
Cost of sales                                 $ 126.5       $ 117.0       $  9.5
Gross margin                                     78.8  %       77.5  %       1.3  %


Cost of Sales increased for the three months ended March 31, 2023 as compared to
the three months ended March 31, 2022 primarily due to increased sales volumes
as noted above. Gross margin increased for the three months ended March 31, 2023
as compared to the three months ended March 31, 2022 primarily due to lower
royalties owed to a third-party licensor on product sales and lower per unit
manufacturing costs for our enzyme products.
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Management's Discussion and Analysis of Financial Condition and Results of


                             Operations (continued)
          (In millions of U.S. dollars, except as otherwise disclosed)

We expect gross margin to range between approximately 77.5% and 79.0% through 2023.



Research and Development

R&D expense includes costs associated with the research and development of product candidates and post-marketing research commitments related to our approved products. R&D expense primarily includes preclinical and clinical studies, personnel and raw materials costs associated with manufacturing clinical product, quality control and assurance, other R&D activities, facilities and regulatory costs.



We manage our R&D expense by identifying the R&D activities we anticipate will
be performed during a given period and then prioritizing efforts based on
scientific data, probability of successful development, market potential,
available human and capital resources and other similar considerations. We
continually review our product pipeline and the development status of product
candidates and, as necessary, reallocate resources among the research and
development portfolio that we believe will best support the future growth of our
business.

R&D expense consisted of the following:



                                              Three Months Ended
                                                  March 31,
                                                                2023         2022        Change
Research and early development                                $  69.0      $  61.7      $  7.3
ROCTAVIAN                                                        30.7         30.7           -
Other approved products                                          29.8         28.3         1.5
VOXZOGO                                                          25.1         29.1        (4.0)
BMN 331                                                           8.6          7.0         1.6
BMN 255                                                           2.0          2.2        (0.2)
Other                                                             6.6          1.8         4.8
Total R&D expense                                             $ 171.8      $ 160.8      $ 11.0


The increase in R&D expense for the three months ended March 31, 2023 as
compared to the three months ended March 31, 2022 was primarily due to higher
spend in research and early development programs due to increased pre-clinical
activities and IND-enabling studies for planned IND filings.

We expect R&D expense to increase in future periods, primarily due to increased activities for our research and early development programs and clinical programs.

Selling, General and Administrative



Sales and marketing (S&M) expense primarily consisted of employee-related
expenses for our sales group, brand marketing, patient support groups and
pre-commercialization expenses related to our product candidates. General and
administrative (G&A) expense primarily consisted of corporate support and other
administrative expenses, including employee-related expenses.

SG&A expenses consisted of the following:



                                  Three Months Ended
                                      March 31,
                                                    2023         2022        Change
S&M expense                                       $ 117.7      $ 104.9      $ 12.8
G&A expense                                         105.3         89.7        15.6
Total SG&A expense                                $ 223.0      $ 194.6      $ 28.4



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Management's Discussion and Analysis of Financial Condition and Results of


                             Operations (continued)
          (In millions of U.S. dollars, except as otherwise disclosed)

S&M expenses consisted of the following:



                                 Three Months Ended
                                     March 31,
                                                   2023         2022        Change
Enzyme Products                                  $  59.0      $  55.9      $  3.1
VOXZOGO                                             24.7         22.1         2.6
ROCTAVIAN                                           23.5         15.7         7.8
Other                                               10.5         11.2        (0.7)
Total S&M expense                                $ 117.7      $ 104.9      $ 12.8

The increase in S&M expense for the three months ended March 31, 2023 as compared to the three months ended March 31, 2022 was primarily a result of increased activities in support of the European launch of ROCTAVIAN and an increase in ROCTAVIAN pre-launch activities in the U.S.

The increase in G&A expense for the three months ended March 31, 2023 as compared to the three months ended March 31, 2022 was primarily due to increased external costs for consulting services related to strategic initiatives and higher employee-related costs.

We expect SG&A expense to increase in future periods as we prepare to launch new products and support the expansion of our global brands.

Intangible Asset Amortization and Contingent Consideration and Gain on Sale of Nonfinancial Assets, Net



Changes during the periods presented for Intangible Asset Amortization and
Contingent Consideration and Gain on Sale of Nonfinancial Assets, Net were as
follows:

                                                                      Three Months Ended
                                                                          March 31,
                                                                                   2023             2022            Change
Amortization of intangible assets                                               $  15.7          $  15.6          $    0.1
Changes in the fair value of contingent consideration                                 -              2.0              (2.0)

Total intangible asset amortization and contingent consideration

$ 15.7 $ 17.6 $ (1.9)



Gain on sale of nonfinancial assets, net                                    

$ - $ 108.0 $ (108.0)

Amortization of intangible assets - the expense for the three months ended March 31, 2023 as compared to the three months ended March 31, 2022 was relatively flat.



Fair value of contingent consideration - the decrease in the fair value of
contingent consideration for the three months ended March 31, 2023 as compared
to the three months ended March 31, 2022 was attributable to attainment of the
final commercial milestone in the fourth quarter of 2022.

Gain on Sale of Nonfinancial Assets, Net - the decrease in the three months
ended March 31, 2023 as compared to the three months ended March 31, 2022 was
due to the sale in the first quarter of 2022 of a Priority Review Voucher (PRV)
with no similar transaction in 2023.
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Management's Discussion and Analysis of Financial Condition and Results of


                             Operations (continued)
          (In millions of U.S. dollars, except as otherwise disclosed)

Interest Income



We invest our cash equivalents and investments in U.S. government securities and
other high credit quality debt securities in order to limit default and market
risk.

                               Three Months Ended
                                   March 31,
                                                  2023       2022       Change
Interest income                                 $ 11.9      $ 1.8      $ 10.1


The increase in Interest Income for the three months ended March 31, 2023
compared to the three months ended March 31, 2022 was primarily due to higher
yields on our investment portfolio. We expect Interest Income to increase over
the next 12 months due to anticipated higher interest rates and yields on our
cash equivalents and investments.

Interest Expense

We incur interest expense primarily on our convertible debt. Interest Expense for the periods presented was as follows:



                                  Three Months Ended
                                      March 31,
                                                     2023       2022       Change
Interest expense                                    $ 3.7      $ 3.8      $ (0.1)


Interest Expense for the three months ended March 31, 2023 as compared to the
three months ended March 31, 2022 was relatively flat. We do not expect Interest
Expense to fluctuate significantly over the next 12 months as the rates on our
convertible debt are fixed. See   Note 6   to our accompanying Condensed
Consolidated Financial Statements for additional information regarding our debt.

Other Expense, Net

Other Expense, Net for the periods presented was as follows:



                                  Three Months Ended
                                      March 31,
                                                    2023         2022       Change
Other expense, net                                $ (10.8)     $ (1.2)     $ (9.6)


The increase in Other Expense, Net for the three months ended March 31, 2023
compared to the three months ended March 31, 2022 was primarily due to a
$12.6 million loss on an equity investment due to impairment in the first
quarter of 2023. See   Note     2   to our accompanying Condensed Consolidated
Financial Statements for additional information.

Provision for Income Taxes

The following table summarizes our Provision for Income Taxes:



                                           Three Months Ended
                                               March 31,
                                                              2023        2022       Change
Provision for income taxes                                   $ 5.9      $ 13.4      $ (7.5)


The decrease in Provision for Income Taxes for the three months ended March 31,
2023 as compared to the three months ended March 31, 2022 was primarily due to
taxes on lower taxable income in the first quarter of 2023. Taxable income
recognized in the first quarter of 2022 included income from the sale of the
PRV.

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Management's Discussion and Analysis of Financial Condition and Results of


                             Operations (continued)
          (In millions of U.S. dollars, except as otherwise disclosed)

Financial Condition, Liquidity and Capital Resources

Our cash, cash equivalents, and investments as of March 31, 2023 and December 31, 2022 were as follows:

December 31,


                                                     March 31, 2023              2022               Change
Cash and cash equivalents                          $         580.1          $     724.5          $   (144.4)
Short-term investments                                       572.0                567.0                 5.0
Long-term investments                                        340.6                333.9                 6.7
Cash, cash equivalents and investments             $       1,492.7

$ 1,625.4 $ (132.7)




We believe our cash generated from sales of our commercial products, in addition
to our cash, cash equivalents and investments will be sufficient to satisfy our
liquidity requirements for at least the next 12 months. We believe we will meet
longer-term expected future cash requirements and obligations through a
combination of cash flows from operating activities, available cash and
investments balances and available revolving loan balances. We will need to
raise additional funds from equity or debt securities, loans or collaborative
agreements if we are unable to satisfy our liquidity requirements. For example,
we may require additional financing to fund the repayment of our convertible
debt, future milestone payments and our future operations, including the
commercialization of our products and product candidates currently under
development, preclinical studies and clinical trials, and potential licenses and
acquisitions. The timing and mix of our funding alternatives could change
depending on many factors, including how much we elect to spend on our
development programs, potential licenses and acquisitions of complementary
technologies, products and companies or if we settle our convertible debt in
cash.

We are mindful that conditions in the current macroeconomic environment, such as
inflation, changes in interest and foreign currency exchange rates, natural
disasters, the continuing effects of the COVID-19 pandemic, and supply chain
disruptions could affect our ability to achieve our goals. In addition, we sell
our products in certain countries that face economic volatility and weakness.
Although we have historically collected receivables from customers in such
countries, sustained weakness or further deterioration of the local economies
and currencies may cause customers in those countries to be unable to pay for
our products. We will continue to monitor these conditions and will attempt to
adjust our business processes, as appropriate, to mitigate macroeconomic risks
to our business.

Our cash flows are summarized as follows:



                                                                   Three Months Ended March 31,
                                                            2023                  2022              Change

Net cash used in operating activities                $    (73.9)              $   (45.4)         $   (28.5)
Net cash provided by (used in) investing activities  $    (30.0)              $    87.8          $  (117.8)
Net cash used in financing activities                $    (40.7)

$ (25.3) $ (15.4)




The increase in net cash used in operating activities in the three months ended
March 31, 2023 compared to March 31, 2022 was primarily attributed to the timing
of cash receipts from our customers and payments to vendors.

The increase in net cash provided by investing activities in the three months
ended March 31, 2023 compared to March 31, 2022 was primarily attributable to
the absence of $110.0 million proceeds from the sale of the PRV in the first
quarter of 2022.

The increase in net cash used in financing activities in the three months ended
March 31, 2023 compared to March 31, 2022 was primarily attributed to higher
taxes paid for net settlement of shares under our equity incentive plans and the
final payment to a third party related to a PKU sales milestone achieved in
2022, partially offset by increased proceeds from stock option exercises.

Financing and Credit Facilities



Our $1.1 billion (undiscounted) of total convertible debt as of March 31, 2023
will impact our liquidity due to the semi-annual cash interest payments as well
as the repayment of the principal amount, if not converted. As of March 31,
2023, our indebtedness consisted of our 0.599% senior subordinated convertible
notes due in 2024 and our 1.25% senior subordinated convertible notes due in
2027, which, if not converted, will be required to be repaid in cash at maturity
in August 2024 and May 2027, respectively. For additional information related to
our convertible debt, see   Note 6   to our accompanying Condensed
                                       28

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Management's Discussion and Analysis of Financial Condition and Results of


                             Operations (continued)
          (In millions of U.S. dollars, except as otherwise disclosed)

Consolidated Financial Statements and Note 10 - Debt to the Consolidated Financial Statements accompanying our Annual Report on Form 10-K for the year ended December 31, 2022.



In October 2018, we entered into an unsecured revolving credit facility of up to
$200.0 million which includes a letter of credit subfacility and a swingline
loan subfacility. The credit facility is intended to finance ongoing working
capital needs and for other general corporate purposes. In May 2021, we amended
the credit facility agreement, extending the maturity date from October 19, 2021
to May 28, 2024, among other changes. The amended credit facility contains
financial covenants including a maximum leverage ratio and a minimum interest
coverage ratio. As of March 31, 2023, there were no amounts outstanding under
the credit facility and we and certain of our subsidiaries that serve as
guarantors were in compliance with all covenants.

Material Cash Requirements

Funding Commitments



Our investment in our research and early development of product candidates and
continued development of our existing commercial products has a major impact on
our operating performance. R&D expenses for our commercial products and certain
product candidates for the period since inception as of March 31, 2023 were as
follows:


                            Since Program Inception
ROCTAVIAN                  $                  974.8
VOXZOGO                    $                  826.1
BMN 331                    $                  118.5
BMN 255                    $                   36.9
Other approved products    $                2,518.8


We cannot estimate with certainty the cost to complete any of our product
development programs. We may need or elect to increase our spending above our
current long-term plans to be able to achieve our long-term goals. This may
increase our capital requirements, including: costs associated with the
commercialization of our products? additional clinical trials; investments in
the manufacturing of our commercial products? preclinical studies and clinical
trials for our product candidates? potential licenses and other acquisitions of
complementary technologies, products and companies? and general corporate
purposes. Additionally, we cannot precisely estimate the time to complete any of
our product development programs or when we expect to receive net cash inflows
from any of our product development programs. Please see "Risk Factors" included
in Part II, Item 1A of this Quarterly Report on Form 10-Q, for a discussion of
the reasons we are unable to estimate such information.

Purchase Obligations



As of March 31, 2023, we had obligations of approximately $251.7 million, which
primarily related to firm purchase commitments entered into in the normal course
of business to procure active pharmaceutical ingredients, certain
inventory-related items, certain third-party R&D services, production services
and facility construction services. Of this amount, $205.9 million is expected
to be paid in the next twelve months.

Other Obligations

As of March 31, 2023, we expect to pay $39.0 million related to the loss contingency recorded as a current liability on the Company's Condensed Consolidated Balance Sheets. The same amount was recorded as a short term receivable related to expected insurance recoveries.



Our lease, contingent obligations and unrecognized tax benefits as of March 31,
2023 have not materially changed from those discussed in "Financial Condition,
Liquidity and Capital Resources" in Part II, Item 7 of our Annual Report on Form
10-K for the year ended December 31, 2022.

See Note 11 to our accompanying Condensed Consolidated Financial Statements for additional information on our commitments.


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