BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) has long been a key player in the biotechnology sector, specializing in rare genetic diseases and innovative therapies. With a strong pipeline of treatments for conditions such as hemophilia, phenylketonuria (PKU), and dwarfism, the company has attracted significant attention from investors and analysts alike. However, as with any biotech stock, BioMarin’s potential hinges on regulatory approvals, clinical trial outcomes, and market adoption of its therapies.
Wall Street analysts have been closely monitoring BioMarin’s progress, particularly with its flagship gene therapy, Roctavian (valoctocogene roxaparvovec), for severe hemophilia A, which received FDA approval in 2023. While some analysts remain bullish on the company’s long-term growth prospects, others express caution due to pricing challenges, competition, and the inherent risks of biotech investing.
In this analysis, we examine whether BioMarin Pharmaceutical is a promising stock according to Wall Street’s top analysts, exploring their ratings, price targets, and key factors influencing their outlook.
We recently compiled a list of the 12 Most Promising Biotech Stocks According to Wall Street Analysts. In this article, we are going to look at where BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) stands against other biotech stocks.
Biotech Sector Set for Growth Amid Innovation and Investor Interest
The biotechnology sector is gaining new momentum with better market conditions, cutting-edge innovations, and growing investor attention. The industry has recovered from a challenging 2024 and is poised for significant expansion led by advances in personalized medicine, AI-driven drug discovery, and growing demand for biologics. MarketsandMarkets predicted that the global biotech market would expand by a strong 13%, from $483.0 billion in 2024 to $546.0 billion in 2025. This expansion demonstrates the sector’s tenacity and promise for growth.
The anticipated shift in the Federal Reserve’s interest rate policies is a key driver of this upturn. Biotech is sensitive to changing rate patterns since it requires a large amount of money for costly R&D and clinical testing. Lower rates make more funding available, which helps biotech companies expand, draw in venture capital, and expedite drug development, according to Genetic Engineering and Biotechnology News. A rate drop, according to analysts, may release billions of dollars in investment funds that have been put aside for fledgling biotech firms looking for steady funding.
Investor interest in biotech equities is growing. Biotech is still a high-growth industry with many chances for risk-takers, despite brief ups and downs. The comeback of biotech has been noted by leading investment banks. Citing solid fundamentals, improved clinical outcomes, and a supportive regulatory environment, Goldman Sachs referred to it as an “undervalued opportunity.” According to the firm, biotech equities have tremendous upside potential and an “option-like structure,” particularly when interest rates decline. Analysts at JPMorgan anticipate a recovery in biotech funding at the same time, pointing to indications of stability in manufacturing and research sectors that were negatively impacted by earlier funding shortages. Industry analysts predict that declining interest rates and advantageous investment circumstances may reopen the IPO window for businesses looking for institutional support, even though biotech IPOs have been quiet since 2021.
As gene editing, AI-powered drug discovery, and precision medicine transform how we treat uncommon genetic illnesses, autoimmune diseases, and cancer, scientific advances are also propelling the expansion of biotech. Biotech firms are meeting medical demands in ways that were unthinkable a decade ago because of developments in CRISPR gene editing and cell treatments. Given this, we will take a look at some of the best promising stocks from the biotech sector.
Our Methodology
For this article, we shortlisted stocks that met the following criteria: a market capitalization of over $2 billion, coverage by at least 15 analysts, and a price target upside of more than 20%. We then ranked the selected stocks based on their price target upside, as determined by Wall Street analysts, as of March 21, 2025.
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A pharmaceutical plant manufacturing a proprietary synthetic oral form of a C-type natriuretic peptide.
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN)
Analyst Upside: 39.51%
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) develops and markets specialized therapies for rare genetic diseases, focusing on enzyme replacement treatments and other biopharmaceuticals. The business is notable for its commitment to rare diseases; it has successfully introduced eight innovative medications to the market, including therapies for lysosomal storage diseases, severe hemophilia A, and achondroplasia.
The Q4 2024 financial results of BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) demonstrated robust development in several important areas. Q4 revenue surged by 16% to $747 million, and its overall revenue grew by 18% to $2.85 billion for the year. Increased acceptance for achondroplasia led to a 56% increase in VOXZOGO sales to $735 million, while enzyme treatments contributed over $1.9 billion, a 12% increase from 2023.
The non-GAAP operating margin increased to 28.6% for the year and 31.1% in Q4, indicating a considerable improvement in the company’s profitability. Revenue growth and operational efficiency were the main drivers of the 69% increase in earnings per share (EPS) to $3.52 and the 88% increase in Q4 EPS to $0.92. Additionally, operating cash flow increased by a staggering 260% to $573 million, giving investors financial flexibility for upcoming projects.
With a 10% growth projection, BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) projects revenue in 2025 to reach between $3.1 billion and $3.2 billion. With non-GAAP operating margins between 32% and 33% and EPS between $4.20 and $4.40, margins are expected to continue to expand which suggests sustained profitability and efficiency. Given its strong financial performance and future growth potential, many analysts consider BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) one of the most promising stocks in the biotech sector.
In addition to its financials, the company is developing its therapeutic pipeline; later this year, early clinical data for BMN 333 and BMN 351 (Duchenne Muscular Dystrophy) are anticipated. These changes may spur additional expansion and have a favorable effect on the stock.
Overall, BMRN ranks 10th among the 12 most promising biotech stocks according to wall street analysts. While we acknowledge the potential of biotech companies, our conviction lies in the belief that AI stocks hold greater promise for delivering higher returns, and doing so within a shorter timeframe. If you are looking for an AI stock that is more promising than BMRN but that trades at less than 5 times its earnings, check out our report about the cheapest AI stock.
Ramin Sarajari’s Comment:
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) is a leading biotechnology company focused on developing treatments for rare genetic diseases. With an established portfolio of approved therapies—including Voxzogo (for achondroplasia) and Roctavian (for hemophilia A)—and a robust pipeline, BioMarin has garnered significant attention from investors and analysts. However, its stock performance has been volatile, reflecting both optimism about its innovative therapies and concerns about commercialization challenges.
Wall Street’s Take on BioMarin
As of mid-2024, analyst sentiment on BMRN is mixed but leaning positive:
- Bullish Case: Many analysts highlight BioMarin’s leadership in rare disease treatments, strong revenue growth from Voxzogo, and the long-term potential of Roctavian (despite slow initial uptake due to pricing and reimbursement hurdles). The company’s pipeline, including gene therapies for PKU and other genetic disorders, adds further upside potential.
- Bearish Concerns: Some analysts remain cautious due to competition in hemophilia, high development costs, and the risks associated with rare disease drug launches. Roctavian’s slower-than-expected adoption has been a key concern, though some believe it could gain traction over time.
Key Analyst Ratings & Price Targets (as of 2024)
- Consensus Rating: Mostly “Buy” or “Hold”
- Average Price Target: ~100−100−110 (suggesting ~15-25% upside from recent levels)
- Notable Upgrades/Downgrades: Some firms have raised targets on strong Voxzogo sales, while others remain neutral until Roctavian’s commercial performance improves.
My View: Cautiously Optimistic
BioMarin has strong science and a solid rare disease franchise, making it a compelling long-term biotech play. However, its near-term performance depends on:
- Roctavian’s adoption (whether payers and patients embrace this high-cost gene therapy).
- Pipeline success (upcoming data for PKU and other gene therapies).
- Profitability improvements (the company is working toward sustained earnings growth).
Final Verdict
- For long-term investors: BioMarin could be a promising hold, given its leadership in rare diseases and potential pipeline catalysts.
- For short-term traders: The stock may remain volatile until Roctavian gains more commercial momentum.