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1. Company Snapshot

1.a. Company Description

BioMarin Pharmaceutical Inc.develops and commercializes therapies for people with serious and life-threatening rare diseases and medical conditions.Its commercial products include Vimizim, an enzyme replacement therapy for the treatment of mucopolysaccharidosis (MPS) IV type A, a lysosomal storage disorder; Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase for patients with MPS VI; and Kuvan, a proprietary synthetic oral form of 6R-BH4 that is used to treat patients with phenylketonuria (PKU), an inherited metabolic disease.


The company's commercial products also comprise Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, which is delivered through subcutaneous injection to reduce blood Phe concentrations; Brineura, a recombinant human tripeptidyl peptidase 1 for the treatment of patients with ceroid lipofuscinosis type 2, a form of Batten disease; Voxzogo, a once daily injection analog of c-type natriuretic peptide for the treatment of achondroplasia; and Aldurazyme, a purified protein designed to be identical to a naturally occurring form of the human enzyme alpha-L-iduronidase.In addition, it develops valoctocogene roxaparvovec, an adeno associated virus vector, which is in Phase III clinical trial for the treatment of patients with severe hemophilia A; BMN 307, an AAV5 mediated gene therapy, which is in Phase 1/2 clinical trial to normalize blood Phe concentration levels in patients with PKU; and BMN 255 that is in Phase 1/2 clinical trial for treating primary hyperoxaluria.The company serves specialty pharmacies, hospitals, and non-U.S. government agencies, as well as distributors and pharmaceutical wholesalers in the United States, Europe, Latin America, and internationally.


BioMarin Pharmaceutical Inc.has license and collaboration agreements with Sarepta Therapeutics, Ares Trading S.A., Catalyst Pharmaceutical Partners, Inc., and Asubio Pharma Co., Ltd.The company was incorporated in 1996 and is headquartered in San Rafael, California.

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1.b. Last Insights on BMRN

BioMarin Pharmaceutical's recent performance was driven by strong Q2 2025 earnings, with revenue increasing 16% year-over-year to $825 million, surpassing Wall Street estimates. The company's non-GAAP diluted EPS of $1.44, a 50% year-over-year increase, also exceeded expectations. The acquisition of Inozyme in July 2025 brought late-stage ERT BMN 401 for ENPP1 deficiency, with pivotal phase 3 data expected in 1H 2026, providing a potential first-mover advantage. Additionally, Voxzogo and Palynziq continue to drive growth, with label expansions and new pipeline candidates like BMN 333 poised to further bolster the company's pipeline.

1.c. Company Highlights

2. BioMarin Pharmaceutical's Q3 2025 Earnings: A Strong Performance

BioMarin Pharmaceutical reported a robust Q3 2025, with total revenue increasing by 11% year-over-year. The company's Enzyme Therapies business unit grew 8% year-to-date, driven by a significant increase in PALYNZIQ sales, which rose by more than 20%. VOXZOGO revenue also demonstrated a strong growth trajectory, increasing by 24% year-to-date, with the company reiterating its guidance of $900-$935 million for 2025. The company's non-GAAP diluted earnings per share came out at -$1.09923, missing estimates of $0.01. The company's cash and investments balance reached approximately $2 billion at the end of Q3, with operating cash flow of $369 million in Q3 and $728 million year-to-date.

Publication Date: Oct -28

📋 Highlights
  • Strong Revenue Growth:: Total revenue rose 11% YoY in Q3 2025, with VOXZOGO up 24% year-to-date and projected 2025 revenue of $900–$935M.
  • Margin Expansion:: Non-GAAP operating margin guidance raised to 26–27% for 2025, with a reaffirmed 40% target for 2026.
  • Cash Position:: $2B in cash/investments and $728M operating cash flow YTD, with $4–5B total financial firepower for growth.
  • Pipeline Advancements:: Next-gen BMN 333 for achondroplasia to initiate Phase II/III in H1 2026, aiming for 3x efficacy over VOXZOGO.
  • 2027 Revenue Outlook:: Potential $4B total revenue (excluding ROCTAVIAN), with hypochondroplasia Phase III data and global label expansion planned.

Financial Guidance and Margin Expectations

The company raised its full-year 2025 total revenue guidance to $3.15 billion and updated its non-GAAP operating margin guidance to 26-27%. Non-GAAP diluted earnings per share guidance is now $3.50-$3.60. BioMarin's 2026 non-GAAP operating margin target remains at 40%. Analysts estimate next year's revenue growth at 8.7%. With a current P/S Ratio of 3.27 and EV/EBITDA of 13.68, the market seems to be pricing in a moderate growth trajectory.

Business Developments and Pipeline Updates

The company announced plans to divest ROCTAVIAN and focus on its core business units, including the advancement of its CANOPY clinical studies with VOXZOGO across four additional indications. VOXZOGO is available in 55 countries, with a strong global launch expected for hypochondroplasia in 2027, pending positive data readout. The company is also advancing its next-generation therapy for achondroplasia, BMN 333, with a target Phase II/III initiation in the first half of 2026.

Growth Prospects and Competitive Landscape

BioMarin outlined a range of potential revenue outcomes for 2027, with a lower end estimate in line with current consensus and a higher end estimate of $4 billion. The company's VOXZOGO remains a growth engine, with 25% of revenues coming from the U.S. and 75% from ex-U.S. The company is also expecting a potential approval of PALYNZIQ for adolescents aged 12 to 17 in 2026 and Phase III data for BMN 401.

Valuation and Financial Strength

With a robust cash position of $2 billion and a total firepower of $4 billion to $5 billion, including growing EBITDA and potential leverage, BioMarin is well-positioned to drive growth through business development and new opportunities. The company's financial strength is reflected in its Free Cash Flow Yield of 8.22%, indicating a decent return for investors. The ROE of 5.69% and ROIC of 6.92% suggest that the company is generating returns on its investments, albeit at a moderate level.

3. NewsRoom

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Critical Survey: BioMarin Pharmaceutical (NASDAQ:BMRN) & Contineum Therapeutics (NASDAQ:CTNM)

Nov -30

Source: defenseworld.net

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BioMarin Pharmaceutical Inc. (BMRN) Presents at Jefferies London Healthcare Conference 2025 Transcript

Nov -18

Source: seekingalpha.com

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BioMarin Pharmaceutical Inc. $BMRN Shares Bought by Envestnet Asset Management Inc.

Nov -17

Source: defenseworld.net

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Alberta Investment Management Corp Invests $4.02 Million in BioMarin Pharmaceutical Inc. $BMRN

Nov -17

Source: defenseworld.net

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BioMarin Pharmaceutical Inc. $BMRN Shares Bought by Acadian Asset Management LLC

Nov -14

Source: defenseworld.net

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BioMarin to Participate at the Jefferies Global Healthcare Conference on Tuesday, November 18, 2025, at 10:30 AM GMT in London, England

Nov -11

Source: prnewswire.com

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D.A. Davidson & CO. Raises Holdings in BioMarin Pharmaceutical Inc. $BMRN

Oct -31

Source: defenseworld.net

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FDA Accepts BioMarin's PALYNZIQ® (pegvaliase-pqpz) Supplemental Biologics License Application for Priority Review to Expand Use to Adolescents Aged 12-17 with Phenylketonuria

Oct -29

Source: prnewswire.com

4. Business Breakdown

4.a. Revenues by Country

4.b. Revenues by Segment

5. Expected revenues mid-term growth (11.00%)

6. Segments

Innovative Therapies

Expected Growth: 11%

BioMarin's innovative therapies drive 11% growth, fueled by increasing adoption of rare disease treatments, strong pipeline of gene therapies, and expanding commercial presence in key markets. Additionally, strategic partnerships and investments in research and development further accelerate growth.

7. Detailed Products

Vimizim

An enzyme replacement therapy for the treatment of Mucopolysaccharidosis type IVA (MPS IVA), a rare genetic disorder.

Naglazyme

An enzyme replacement therapy for the treatment of Mucopolysaccharidosis type VI (MPS VI), a rare genetic disorder.

Kuvan

A medication used to reduce the blood levels of phenylalanine in patients with phenylketonuria (PKU), a rare genetic disorder.

Firdapse

A medication used to treat Lambert-Eaton myasthenic syndrome (LEMS), a rare autoimmune disorder.

Brineura

An enzyme replacement therapy for the treatment of CLN2 disease, a rare genetic disorder.

Palynziq

A medication used to reduce the blood levels of phenylalanine in patients with phenylketonuria (PKU), a rare genetic disorder.

8. BioMarin Pharmaceutical Inc.'s Porter Forces

Forces Ranking

Threat Of Substitutes

BioMarin Pharmaceutical Inc. has a moderate threat of substitutes due to the presence of alternative treatments and therapies for rare genetic disorders. However, the company's focus on developing innovative and life-changing treatments reduces the threat of substitutes.

Bargaining Power Of Customers

BioMarin Pharmaceutical Inc. has a low bargaining power of customers due to the specialized nature of its products and the lack of alternative treatment options for rare genetic disorders.

Bargaining Power Of Suppliers

BioMarin Pharmaceutical Inc. has a moderate bargaining power of suppliers due to the presence of multiple suppliers for raw materials and manufacturing services. However, the company's dependence on a few key suppliers for certain components increases the bargaining power of suppliers.

Threat Of New Entrants

BioMarin Pharmaceutical Inc. has a low threat of new entrants due to the high barriers to entry in the biotechnology industry, including the need for significant investments in research and development, manufacturing, and regulatory approvals.

Intensity Of Rivalry

BioMarin Pharmaceutical Inc. operates in a highly competitive industry with several established players, leading to a high intensity of rivalry. The company faces competition from other biotechnology companies, as well as from pharmaceutical companies that are expanding into the rare genetic disorder space.

9. SWOT Analysis

Strength

Weaknesses

Opportunities

Threats

10. Capital Structure

10.a. Balance Sheet

10.b. Weighted Average Cost of capital

Value
Debt Weight 18.12%
Debt Cost 3.95%
Equity Weight 81.88%
Equity Cost 5.65%
WACC 5.34%
Leverage 22.13%

11. Quality Control: BioMarin Pharmaceutical Inc. passed 7 out of 9 key points

12.a Historical Valuation

12.b Price/Earnings Ratio

12.c Margin Valuation

12.d Peers Valuation

Peers Group Analysis

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PTC Therapeutics

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Growth: 5.6

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Momentum: 9.5

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BioMarin Pharmaceutical

A-Score: 4.9/10

Value: 4.3

Growth: 8.4

Quality: 8.0

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A-Score: 3.5/10

Value: 8.0

Growth: 0.4

Quality: 4.9

Yield: 0.0

Momentum: 6.0

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Peers Metrics

12.e Scoring Insights

12.f DCF BETA

Parameters

Short Term Growth

Short term Time

Long-Term Growth

WACC

Target Price

53.4$

Current Price

53.4$

Potential

-0.00%

Expected Cash-Flows