Biopharma Co. Reports First Quarterly Revenue Above US$1 Billion as Acquisition Expands Portfolio

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1. Ownership and Share Structure Information

Ownership percentages, named holders, share count, market capitalization, and the 52-week range for BioMarin were compiled from company public filings and investor materials, exchange notices, insider-trade reports (SEDI / equivalent), and third-party data providers. Figures are as of 2026-11-03 unless a source was dated earlier. Pie slices are normalized to 100% of basic shares outstanding; retail is the residual after disclosed institutions, management, insiders, and strategic holders. Warrant terms are current outstanding series only. Restructures are historical consolidations, splits, and symbol or name changes. Insider buying and selling covers open-market trades from the prior six months through 2026-11-03 and excludes option exercises. These are public-source estimates and can differ from a later official register.

BioMarin Pharmaceutical Inc. (BMRN:NASDAQ) reported Q3 2026 revenue of US$1.025 billion, a 32% increase year-over-year, as contributions from its Amicus Therapeutics Inc. acquisition and growth across several existing therapies expanded its commercial portfolio.

In the release, CEO Alexander Hardy said the quarter marked the first time the company had generated more than US$1 billion in quarterly revenue. BioMarin also updated its full-year financial guidance, announced a new investigational Fabry disease treatment, and reported regulatory progress for VOXZOGO.

Key Investor Takeaways

• BioMarin reported Q3 revenue of US$1.025 billion, a 32% year-over-year increase, with growth supported by the Amicus acquisition and higher revenue from several existing therapies.
• GALAFOLD and POMBILITI + OPFOLDA contributed US$201 million in combined revenue during their first full quarter as part of BioMarin.
• The company added BMN 450, an investigational long-acting subcutaneous ERT for Fabry disease, through a license agreement that includes a US$70 million upfront payment.
• VOXZOGO has a May 30, 2027, FDA action date for hypochondroplasia, while a separate decision on full approval for achondroplasia is expected by February 28, 2027.
• BioMarin lowered its 2026 revenue and non-GAAP EPS guidance, citing international order timing uncertainty and acquisition-related charges.
• BioMarin continues to expect approximately US$280 million in GAAP cost synergies from the Amicus integration by 2028.

Latest Financial and Operating Results

Revenue growth was led by a 44% year-over-year increase in metabolic conditions revenue to US$744 million, alongside a 12% increase in skeletal conditions revenue. The quarter marked BioMarin's first full reporting period incorporating products acquired through its April 2026 purchase of Amicus Therapeutics. GALAFOLD contributed US$152 million, while POMBILITI + OPFOLDA generated US$49 million. The number of patients treated with the therapies increased approximately 13% and 69%, respectively, compared with the prior-year period.

At the same time, VOXZOGO revenue increased 12% to US$245 million, while the number of children receiving the treatment globally rose 17%. Patients with achondroplasia were receiving VOXZOGO in 58 countries by the end of the quarter. More than 75% of the therapy's revenue came from outside the U.S., and BioMarin said a large international order originally expected in Q3 had shifted into Q4.

Matthew Harrison of Morgan Stanley lowered the price target from US$122 to US$119 while maintaining an "Overweight" rating

Other product results included 18% revenue growth for VIMIZIM to US$216 million. NAGLAZYME revenue declined 11% to US$108 million, which the company said was due to the timing of large international orders.

GAAP net loss widened to US$123 million from US$31 million a year earlier. BioMarin attributed the increase primarily to the Amicus acquisition, including intangible asset amortization, interest expense on debt issued to finance part of the transaction, amortization of the inventory fair-value step-up, and integration and restructuring costs. Acquired in-process research and development (IPR&D) charges associated with Alesta Therapeutics also contributed to the loss.

BioMarin's Pipeline Updates

BioMarin expanded its Fabry disease pipeline through an exclusive license agreement with GC Biopharma and Hanmi Pharm for HM15421/GC1134A, which the company refers to as BMN 450. The agreement grants BioMarin worldwide rights to the drug, excluding Korea, where GC Biopharma and Hanmi will retain commercialization rights. The investigational, long-acting enzyme replacement therapy (ERT) is administered subcutaneously and is in a global Phase 1/2 clinical study. BioMarin said BMN 450 could extend its reach to people with Fabry disease beyond those with genetic variants amenable to GALAFOLD and noted that the drug has the potential to become the first subcutaneous ERT for the condition. Under the agreement announced October 29, BioMarin will pay US$70 million upfront. GC Biopharma and Hanmi are also jointly eligible to receive up to an additional US$405 million upon the completion of certain development and commercial milestones, as well as tiered royalties on net sales.

BioMarin also reported regulatory progress for VOXZOGO. The U.S. Food and Drug Administration (FDA) assigned a May 30, 2027, Prescription Drug User Fee Act (PDUFA) target action date for the company's supplemental new drug application seeking to expand VOXZOGO's use to treat hypochondroplasia (HCH). According to the company, VOXZOGO could be the first potential targeted therapy for the condition. The European Medicines Agency (EMA) also validated BioMarin's application for HCH. A separate FDA decision is expected by February 28, 2027, on BioMarin's application for full approval of VOXZOGO in children with achondroplasia. The application includes long-term safety and efficacy data, including final adult height and clinical outcomes beyond linear growth.

Finally, according to the Q3 press release, the company's Phase 2/3 study of BMN 333, a long-acting C-type natriuretic peptide candidate for achondroplasia, continues to enroll patients. BioMarin expects to report dose-response data in 2027. The company also expects to provide a program update by the end of 2026 for BMN 351, its Phase 1/2 candidate for Duchenne muscular dystrophy.

2026 Guidance Update

BioMarin lowered its full-year 2026 revenue and non-GAAP diluted earnings per share (EPS) guidance, citing uncertainty around the timing of multiple orders from a single international customer as the reason for its revised revenue outlook. It also expects acquired IPR&D charges and ongoing expenses associated with the Alesta acquisition and BMN 450 license agreement to affect its full-year earnings.

The company now expects:

• Total revenue: US$3.8 billion to US$3.9 billion, compared with previous guidance of US$3.875 billion to US$3.925 billion.
• Metabolic conditions revenue: US$2.675 billion to US$2.75 billion, down from US$2.725 billion to US$2.775 billion.
• VOXZOGO revenue: US$975 million to US$1.025 billion, compared with the previous range of US$1 billion to US$1.05 billion.
• Non-GAAP diluted EPS: US$2.80 to US$3.10, compared with previous guidance of US$4.90 to US$5.10.

BioMarin said the Alesta acquisition and BMN 450 license agreement are expected to result in approximately US$320 million and US$83 million, respectively, in total pre-tax charges during 2026. These amounts include acquired IPR&D charges and ongoing operating expenses. The company continues to expect approximately US$280 million in GAAP cost synergies from the Amicus integration by 2028, or approximately US$220 million on a non-GAAP basis.

Potential investors interested in hearing more about the company directly can tune into the following upcoming investor conferences:

• Stifel Healthcare Conference, November 11 at 3:20 pm EST
• UBS Global Biopharma Conference, November 16 at 9:20 am GMT
• Jefferies Global Healthcare Conference, November 17 at 11:30 am GMT

A live webcast will be available here.

Analysts Awaiting Future Revenue Proof

Both Wedbush and H.C. Wainwright & Co. released updated research reports about BioMarin on November 3, 2026. Yun Zhong, Ph.D., of Wedbush reiterated an "Outperform" rating but lowered the price target, writing, "We lower our PT from US$94 to US$89 after model adjustments based on reported numbers and guidance, and we continue to believe that BMRN's strengthened revenue stream should provide a strong support for multiple new growth opportunities."

H.C. Wainwright & Co.'s Mitchell S. Kapoor reiterated a "Neutral" rating, stating, "VOXZOGO's ~85% U.S. retention keeps us cautious as oral competition could arrive before hypochondroplasia adds revenue." Kapoor maintained a US$60 price target.

Other recent analyst coverage includes: 

Common Questions from Investors

What drove BioMarin's Q3 2026 revenue growth?

The primary contributors were the addition of GALAFOLD and POMBILITI + OPFOLDA following the Amicus acquisition, along with higher VIMIZIM and VOXZOGO revenue. Metabolic Conditions revenue increased 44%, while Skeletal Conditions revenue rose 12%.

What is the status of VOXZOGO's regulatory applications?

The FDA assigned a May 30, 2027, PDUFA date for VOXZOGO in hypochondroplasia. A separate FDA decision on full approval for achondroplasia is expected by February 28, 2027. The EMA has also validated the HCH submission.

What is BMN 450?

BMN 450, also identified as HM15421/GC1134A, is an investigational long-acting enzyme replacement therapy for Fabry disease. The candidate is in global Phase 1/2 development and is designed for subcutaneous administration.

Why did BioMarin lower its 2026 guidance?

The company cited uncertainty around the timing of multiple orders from a single international customer, as well as acquired IPR&D charges and ongoing expenses associated with the Alesta acquisition and BMN 450 license agreement.

How much does BioMarin expect to save from the Amicus integration?

BioMarin continues to expect approximately US$280 million in GAAP cost synergies, or US$220 million on a non-GAAP basis, to be fully realized by 2028.