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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 Inc.'s recent performance was negatively impacted by several factors. The company's decision to withdraw its hemophilia gene therapy, Roctavian, from the market likely raised concerns among investors. Additionally, several institutional investors, including Envestnet Asset Management Inc., trimmed their stakes in the company, with Envestnet reducing its position by 50.4%. Furthermore, BioMarin announced the discontinuation of mid-stage trials for its bone disorder treatment due to reports of hip joint complications, leading to a 4% decline in its shares.

1.c. Company Highlights

2. BioMarin Pharmaceutical's Strong Revenue Growth Continues

BioMarin Pharmaceutical reported a record $3.22 billion in total revenues for 2025, representing a 13% year-over-year growth. The company's enzyme therapies revenue increased by 9%, while Voxzogo revenues surged by 26%. In the fourth quarter, total revenues reached $875 million, a 17% year-over-year growth, driven by a 31% increase in Voxzogo sales and a 13% rise in enzyme therapies revenue. The company's actual EPS came in at $0.46, beating estimates of $0.25.

Publication Date: Feb -25

📋 Highlights
  • 2025 Revenue Growth:: Total revenues reached $3.22 billion (+13%), driven by 9% enzyme therapies growth ($2.225–$2.275B guidance for 2026) and 26% Voxzogo revenue ($927M; $975M–$1.025B guidance for 2026).
  • Q4 Performance:: Fourth-quarter revenues hit $875 million (+17% YoY), with Voxzogo and enzyme therapies growing 31% and 13% respectively.
  • BMN 333 Pipeline:: Phase II/III study aims to demonstrate 2.25 cm/year growth over placebo, targeting 90% power to detect 50% growth velocity increase vs. Voxzogo.
  • Voxzogo Safety Data:: 10,000+ patient-years of safety data, including 10 years of treatment in children, supporting full FDA approval and differentiation in the achondroplasia market.
  • Hypochondroplasia Expansion:: Voxzogo’s potential extension into hypochondroplasia, with expected approvals in 2027 and immediate revenue impact, mirroring its achondroplasia success.

Segment Performance

The strong performance was largely driven by the continued adoption of Voxzogo, which generated $927 million in revenue for the full year, a 26% increase over 2024. Enzyme therapies also demonstrated durability and reach across the company's 80-country footprint. For 2026, the company expects enzyme therapies revenue to range between $2.225 billion to $2.275 billion, and Voxzogo revenue to be between $975 million to $1.025 billion, representing high single-digit growth rates for both business units.

Pipeline Updates and Future Prospects

The company is making significant progress in its pipeline, with multiple meaningful milestones anticipated in the coming year. BioMarin is preparing to submit the full approval package for Voxzogo in achondroplasia and is set to begin enrollment in a Phase II/III study of BMN 333, a next-generation, long-acting CNP therapy. As Gregory Friberg noted, "We present a comprehensive story around final adult height and broader health and wellness outcomes for Voxzogo's patient population." Analysts estimate next year's revenue growth at 10.8%.

Valuation Metrics

With a P/E Ratio of 23.11, P/S Ratio of 3.89, and EV/EBITDA of 14.65, the market appears to be pricing in a certain level of growth for BioMarin Pharmaceutical. The company's ROE of 8.69% and ROIC of 6.9% suggest a relatively efficient use of capital. The current valuation metrics indicate that the market has high expectations for the company's future performance, but the anticipated growth in revenues and pipeline progress could potentially justify these levels.

3. NewsRoom

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BioMarin Pharmaceutical's Rare Disease Portfolio Supplemented By Amicus Acquisition Makes A Buy

Apr -15

Source: seekingalpha.com

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BioMarin stops mid-stage trials of bone disorder treatment

Mar -16

Source: reuters.com

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BioMarin Presents New Data on the Positive Impact of Early Treatment With VOXZOGO® (vosoritide) on Proportionality and Arm Span in Children with Achondroplasia at the 2026 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting

Mar -12

Source: prnewswire.com

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Dimensional Fund Advisors LP Purchases 671,566 Shares of BioMarin Pharmaceutical Inc. $BMRN

Mar -12

Source: defenseworld.net

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BioMarin Pharmaceutical Inc. (BMRN) Presents at Barclays 28th Annual Global Healthcare Conference Transcript

Mar -11

Source: seekingalpha.com

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Uniqure To Rally Around 92%? Here Are 10 Top Analyst Forecasts For Wednesday

Mar -11

Source: feeds.benzinga.com

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BioMarin Pharmaceutical Inc. (BMRN) Presents at Leerink Global Healthcare Conference 2026 Transcript

Mar -09

Source: seekingalpha.com

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Fisher Asset Management LLC Has $1.72 Million Stock Holdings in BioMarin Pharmaceutical Inc. $BMRN

Mar -07

Source: defenseworld.net

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

A-Score: 5.8/10

Value: 7.8

Growth: 5.6

Quality: 8.4

Yield: 0.0

Momentum: 9.5

Volatility: 3.3

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BioMarin Pharmaceutical

A-Score: 4.8/10

Value: 5.0

Growth: 8.4

Quality: 8.0

Yield: 0.0

Momentum: 1.5

Volatility: 6.0

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Rhythm Pharma

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Growth: 5.1

Quality: 4.1

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SpringWorks Therapeutics

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Value: 6.2

Growth: 4.6

Quality: 5.0

Yield: 0.0

Momentum: 8.0

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A-Score: 4.0/10

Value: 8.0

Growth: 0.4

Quality: 4.9

Yield: 0.0

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Agios Pharmaceuticals

A-Score: 3.2/10

Value: 7.6

Growth: 4.2

Quality: 5.1

Yield: 0.0

Momentum: 0.0

Volatility: 2.0

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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

55.27$

Current Price

55.27$

Potential

-0.00%

Expected Cash-Flows