California Biotech Co. Discovers VOXZOGO Breakthrough, Reports Strong Q1

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1. Ownership and Share Structure Information

The information listed above was updated on the date this article was published and was compiled from information from the company and various other data providers.

Since the start of May 2026, BioMarin Pharmaceuticals Inc. (BMRN:NASDAQ) has released two significant pieces of news. On May 2, BioMarin presented at the Pediatric Endocrine Society's 2026 annual meeting, offering new data on the effects of long-term usage of its drug, VOXZOGO (vosoritide). The drug is designed to treat Achondroplasia, and the study saw a positive impact on patient arm span and bone density.

Achondroplasia is the most common form of skeletal dysplasia and leads to disproportionate short stature. It is characterized by the slowing of endochondral ossification, which causes disordered architecture in the long bones, face, spine, and skull. Over 80% of patients with this condition have no hereditary history, spontaneously developing a change in the FGFR3 gene, which regulates bone growth.

Results showed that "arm span Z-scores improved from baseline in all age groups, and the arm span-to-height ratio also remained stable over time, showing treatment resulted in proportional skeletal growth. Children who initiated treatment with VOXZOGO after age 5 also achieved a mean difference in standing height of 10.60 cm after six years of treatment (p<0.0001) and 13.59 cm after eight years of treatment (p<0.0001), compared with untreated natural history cohorts."

M.D. and Ph.D. Bradley Miller, a pediatric endocrinologist at the University of Minnesota, spoke of the study results, saying, "With VOXZOGO, we now have a depth and duration of evidence that is unmatched in the treatment of achondroplasia — providing meaningful insight not only into growth, but into the broader, sustained impact on a child's health. When you can see consistent benefits over time, it gives you greater confidence to intervene early and treat with purpose."

The company also released the results of a study focused on the impact of long-term treatment of 119 children on their bone mineral content and bone mineral density every year for six years. Results showed either improvement or consistency year over year.

In a statement, Greg Frieberg, MD., Executive Vice President and Chief Research & Development Officer at BioMarin, said, "We are committed to understanding and sharing the clear impact of long-term treatment with VOXZOGO on health measures beyond height, including arm span and bone density, that are meaningful to the thousands of children around the world receiving this medicine. With more than ten years of clinical research now, we have seen again and again a breadth of data that continues to demonstrate that long-term and early treatment are critical to ensure the greatest benefit in children with achondroplasia."

2026 Financial & Operational Results

On May 5, 2026, BioMarin released Q1 2026 financial and operating cost results. President and CEO, Alexander Hardy, opened the release by saying, "With the acquisition of Amicus Therapeutics complete, the addition of GALAFOLD and POMBILITI + OPFOLDA to our commercial portfolio allows us to reach patients with Fabry and Pompe diseases and meaningfully strengthens and accelerates our near-to-mid-term growth rates. We expect these high-growth assets to support our strongest financial performance yet in 2026. Next quarter, we look forward to updating you on the long-term outlook of the Amicus integration based on our plans to leverage our global scale to expand the potential of these transformative therapies. With a faster-growing commercial portfolio, together with two near-term Phase 3 data readouts and ongoing pipeline progress expected over the coming quarters, we are well-positioned to drive innovation, create shareholder value, and improve outcomes for patients worldwide."

BioMarin listed Q1 2026 financial highlights as follows:

• Total Revenues for the first quarter of 2026 were US$766 million, an increase of US$21 million compared to the same period in 2025, primarily driven by the timing of large government orders outside the U.S. and an increase in patient demand for Enzyme Therapies (ALDURAZYME^®, BRINEURA, NAGLAZYME, PALYNZIQ, and VIMIZIM) as well as new patients initiating VOXZOGO therapy across all regions. The increase was partially offset by lower ROCTAVIAN^® revenue attributed to the voluntary withdrawal of the product from the market announced in the first quarter of 2026.
• GAAP Net Income for the first quarter of 2026 decreased to US$106 million compared to US$186 million for the same period in 2025. The decrease in GAAP Net Income was primarily attributed to the following:
• Higher Selling, General & Administrative (SG&A) spend primarily due to incremental administrative costs related to ongoing support of corporate initiatives and pre-close costs for Amicus acquisition, and higher sales and marketing spend on VOXZOGO, PALYNZIQ and VIMIZIM;
• Higher Cost of Sales primarily due to a US$31 million charge associated with an unsuccessful process qualification campaign to expand NAGLAZYME manufacturing capabilities;
• Higher Research and Development spend to support BMN 401, a late-stage clinical program acquired in the third quarter of 2025;
• Partially offset by revenue growth as mentioned above.
• Non-GAAP Income for the first quarter of 2026 decreased to US$149 million compared to US$221 million for the same period in 2025. The decrease in Non-GAAP Income was primarily due to the factors noted above.
• GAAP Diluted Earnings per Share (EPS) and Non-GAAP Diluted EPS for the first quarter of 2026 decreased compared to the same period in 2025, primarily reflecting the discrete items and higher operating expenses described above.

BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with a portfolio of commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients.

Enzyme Replacement Therapy: A Growing Sector

"The global enzyme replacement therapy market size is calculated at USD 10.4 billion in 2024, grew to USD 11.34 billion in 2025, and is projected to reach around USD 24.71 billion by 2034," reported Towards Healthcare on April 21, 2026. The rising prevalence of genetic disorders, paired with better diagnostics and development activities, could expand the market at a CAGR of 9.04% by 2034. Enzyme Replacement Therapy (ERT) replaces a missing or damaged enzyme in a patient's body in order to treat a disease and is used to treat rare genetic disorders. The article went on to say that, "ERT is being studied to treat cancer, neurodegenerative diseases, joint problems, inflammation, and infections."

In February 2026, Iqvia discussed the global pharma market projection for 2026, noting that total drug usage is expected to surpass four trillion doses daily by 2030. They wrote, "The largest drivers of medicine spending growth through the next five years will continue to be the use in developed markets of innovative therapeutics, especially in oncology, immunology, diabetes, and obesity."

BCG talked about trends biopharma companies need to be aware of in 2026 in order to stay competitive, saying, "Near term, companies need to continue to innovate to decrease the complexity and cost of these therapies, and governments can find ways to incentivize and pay for them. The longer-term challenge for companies is to factor operational and economic considerations into R&D decision making earlier, ensuring that trial designs match real-world usage, indication sequences match opportunity, and endpoints enable market access."

Analysts Offer Mixed Views

Four analysts have covered BioMarin since its May 5, 2026, release, all with varying ratings.

On May 5, Yun Zhong of Wedbush gave the company an 'Outperform' rating, with a 12-month price target of US$94.00, nearly doubling its current price. Zhong wrote, "We believe that launch of Yuviwel in early 2Q26 by Ascendis Pharma (ASND, OP) will have an impact on Voxzogo, but the magnitude remains to be seen."

H.C. Wainwright & Co.'s Mitchell Kapoor offered the company a 'Neutral' rating on the same day, with a price target of only US$55.00. Kapoor's reasoning: "Consensus moves from US$216M in 1Q26 to US$296M in 4Q26, which fits management’s 2H/4Q weighting and VOXZOGO’s ex-U.S. order dynamics. Large tenders, country-access timing, and periodic government orders can create material Q/Q lumpiness. The harder number is US$346M in 4Q27, vs. our US$239M estimate. By then, YUVIWEL should have built U.S. access, the expected European decision could pressure VOXZOGO’s largest revenue base, and oral FGFR rivals should be closer. Europe matters because ~75% of VOXZOGO revenue is ex-U.S.; a U.S.-only hit affects a minority of sales, while ex-U.S. markets drive most of the revenue base, and much of the 4Q order-timing strength consensus is extrapolating."

Ilya Zubkov and Boris Tolkachev of Freedom Broker also weighed in on May 5, giving BioMarin a 'Buy' rating, with a price target of US$105.00. They wrote: "Our Buy rating and $105 price target for BMRN are based on a weighted valuation approach, combining a 5-year DCF model employing the Free Cash Flow to the Firm (FCFF) method, and relative valuation multiples." The pair cited potential risks such as pipeline setbacks, development delays, potential competitive pressure, or possible softening demand across the enzyme therapy portfolio.

Lastly, Leland Gershell, MD and Ph.D., and Jason Dorr, PharmD., of Oppenheimer gave their opinion on May 5. The pair gave the company an 'Outperform' rating, with a price target of US$95. Their reasoning for the outperform target was: "We see shares as trading on the base business alone and anticipate outperformance on accelerating profitability, sales growth from newly-acquired assets, potentially supportive pipeline readouts (BMN 333, BMN 401), and potential development candidate acquisition/in-licensing."

Trials and Drugs in the Pipeline

According to the press release, the company plans to share topline results from its registration-enabling Phase 3 pivotal clinical trial of VOXZOGO in children with hypochondroplasia (CANOPY-HCH-3) in the first half of 2026. If the clinical results are positive, the company plans to submit data to health authorities in the second half of 2026 to seek approval for this new indication.

BioMarin listed its 2026 full-year financial guidance as follows:

• Updated guidance reflects post-close contributions from Amicus beginning April 27, 2026.
• As previously communicated, the acquisition of Amicus is expected to be slightly dilutive to full-year 2026 Non-GAAP Diluted EPS; historical BioMarin Non-GAAP Diluted EPS guidance is unchanged.
• The Amicus acquisition will be accounted for as a business combination, which will result in intangible amortization impacting GAAP results over future periods and excluded from Non-GAAP results. BioMarin will continue to include interest expense related to the Amicus financing in both GAAP and Non-GAAP financial results. Guidance is subject to change based on various factors, including finalization of purchase accounting.
• The company expects approximately two-thirds of 2026 Non-GAAP Diluted EPS to be recognized in the second half of 2026, primarily due to the anticipated timing of revenue (more than 55% of 2026 Total Revenues is expected in 2H). Non-GAAP Diluted EPS in Q2 is expected to be modestly higher than in Q1.

With the acquisition of new drugs, companies, and advancing pipelines, the company expects a 20% growth rate in 2026.

Ownership & Share Information¹

BioMarin Pharmaceutical Inc. has a market cap of CA$10.46 billion, with 193.28 million shares outstanding. The company's 52-week range is US$50.76-US$66.28.

Institutions own 97% of shares, while Management & Insiders own 3% of shares.