BIOMARIN PHARMACEUTICAL INC (BMRN): Results of Operations and Financial Condition
BIOMARIN PHARMACEUTICAL INC (BMRN) filed an SEC Form 8-K — Results of Operations and Financial Condition. Exhibit 99.1 Contact: Investors: Media: Traci McCarty Marni Kottle BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical Inc. (415) 455-7558 (650) 374-2803 BioMarin Reports Second Quarter 2026 Financial and Operating Results Second Quarter 2026 Total Revenues Increased 20% Year-ov
How this was made
The 30-second read
Why it matters
The most tradable elements are the raised full-year VOXZOGO revenue guidance, the VOXZOGO hypochondroplasia sNDA submission, FDA acceptance for achondroplasia with a Feb 28, 2027 PDUFA target, and the EU approval of PALYNZIQ for adolescents 12+ with PKU. The discontinuation of BMN 401 is a negative offset.
Market read
A guidance raise plus multiple regulatory milestones increases the probability-weighted outlook for BioMarin’s growth engines, likely driving repricing ahead of the next earnings update.
What to watch
The filing emphasizes guidance and milestones but does not provide detailed financial statement line items in the excerpt; traders may need to verify margin, cash flow, and integration cost trajectory from the full exhibit.
Second Quarter 2026 Total Revenues Increased 20% Year-over-year to $990 million
Revenue increased 20% year-over-year, supported by acquired products and growth in VOXZOGO and PALYNZIQ, but GAAP net income declined 81%, non-GAAP income declined 16%, and both GAAP and non-GAAP operating margins contracted.
Key metrics
as reported| Metric | Value | q/q | y/y |
|---|---|---|---|
| Total RevenuesGAAP | $990 million | – | 20% |
| GAAP Net IncomeGAAP | $45 million | – | (81)% |
| Non-GAAP Incomenon-GAAP | $236 million | – | (16)% |
| GAAP Operating Margin %GAAP | 11.2% | – | – |
| Non-GAAP Operating Margin %non-GAAP | 36.4% | – | – |
| GAAP Diluted EPSGAAP | $0.23 | – | (81)% |
| Non-GAAP Diluted EPSnon-GAAP | $1.20 | – | (17)% |
| Six Months Total RevenuesGAAP | $1,756 million | – | 12% |
| Six Months GAAP Net IncomeGAAP | $150 million | – | (65)% |
| Six Months Non-GAAP Incomenon-GAAP | $385 million | – | (23)% |
| Six Months GAAP Operating Margin %GAAP | 13.7% | – | – |
| Six Months Non-GAAP Operating Margin %non-GAAP | 31.0% | – | – |
| Six Months GAAP Diluted EPSGAAP | $0.77 | – | (65)% |
| Six Months Non-GAAP Diluted EPSnon-GAAP | $1.96 | – | (24)% |
Segments
| Segment | Revenue | q/q | y/y |
|---|---|---|---|
| VOXZOGONew patients initiating VOXZOGO therapy across all regions. | $253 million | – | 14% |
| VIMIZIMLower revenue due to the timing of large government orders outside the U.S. | $194 million | – | (10)% |
| NAGLAZYMEThe filing did not specify a product-specific revenue driver. | $135 million | – | 5% |
| PALYNZIQGrowth in U.S. patients treated with PALYNZIQ. | $135 million | – | 27% |
| BRINEURAThe filing did not specify a product-specific revenue driver. | $51 million | – | 4% |
| ALDURAZYMELower sales volume due to the timing of order fulfillment to Sanofi. | $44 million | – | (21)% |
| KUVANThe filing did not specify a product-specific revenue driver. | $24 million | – | (11)% |
| ROCTAVIANIn 2026, the company announced that it will no longer market ROCTAVIAN. | $12 million | – | 33% |
Full-year 2026 outlook
- NoteVOXZOGO revenue: at least $1 billion in 2026.
- NoteApproximately $280 million of cost reductions on a GAAP basis, expected to be fully realized in 2028.
- NoteApproximately $220 million of cost reductions on a Non-GAAP basis, expected to be fully realized in 2028.
- NoteGALAFOLD and POMBILITI + OPFOLDA, combined, are expected to reach over 60% Non-GAAP Operating Margin by 2030.
- NoteThe company is targeting gross leverage below 2.5 times by mid-year 2027.
- NoteBioMarin expects peak revenue for GALAFOLD to be approximately $1.4 billion by the mid-2030s.
- NoteBioMarin expects peak revenue for POMBILITI + OPFOLDA to be approximately $1.2 billion by the mid-to-late-2030s.
- NoteThe company expects to provide a program update for BMN 351 by year-end.
- NoteThe Phase 2/3 study of BMN 333 is enrolling, with a data update expected in 2027.
- NoteThe Phase 3 ACTION 3 trial for BMN 820 is ongoing, with pivotal data expected in 2028.
- NoteVOXZOGO hypochondroplasia has a potential 2027 launch if approved.
- NoteThe PDUFA target action date for full approval of VOXZOGO in children with achondroplasia is February 28, 2027.
What drove it
- Total revenue growth was primarily driven by revenues from GALAFOLD and POMBILITI + OPFOLDA, acquired from Amicus on April 27, 2026.
- New patients initiating VOXZOGO therapy across all regions contributed to revenue growth.
- Growth in U.S. patients treated with PALYNZIQ contributed to revenue growth.
- Metabolic Conditions revenue grew 25% Y/Y, driven by the additions of GALAFOLD and POMBILITI + OPFOLDA and continued strength from PALYNZIQ.
- The number of patients on therapy grew across all BioMarin-marketed therapies, both Y/Y and sequentially.
- The number of children being treated with VOXZOGO globally increased by more than 20% Y/Y in the second quarter.
- The U.S. drove approximately 25% of total VOXZOGO revenue during the quarter.
Concerns
- GAAP net income decreased to $45 million from $241 million, primarily due to Amicus integration and restructuring costs, intangible asset amortization, interest expense, and amortization of inventory fair value step-up.
- Non-GAAP income decreased to $236 million from $282 million, primarily due to higher interest expense, higher sales and marketing spend, and higher R&D spend related to BMN 401.
- GAAP operating margin decreased to 11.2% from 33.5%, while non-GAAP operating margin decreased to 36.4% from 39.9%.
- VIMIZIM revenue decreased 10% due to the timing of large government orders outside the U.S.
- ALDURAZYME revenue decreased 21% due to the timing of order fulfillment to Sanofi.
- BioMarin discontinued development of BMN 401 across all indications after the pivotal ENERGY 3 trial did not meet one of its two co-primary endpoints.
What to watch
- The company plans to provide an update on the VOXZOGO hypochondroplasia sNDA as part of its third quarter earnings update.
- The full Phase 3 CANOPY-HCH-3 dataset is scheduled for a late-breaking oral presentation in September.
- FDA action on the VOXZOGO sNDA for full approval in children with achondroplasia has a PDUFA target action date of February 28, 2027.
- Execution of the approximately $280 million GAAP and approximately $220 million Non-GAAP Amicus cost reductions expected to be fully realized in 2028.
- Progress toward the gross leverage target below 2.5 times by mid-year 2027.
- The expected BMN 351 program update by year-end.
- Pivotal data from the Phase 3 ACTION 3 trial of BMN 820 expected in 2028.
Balance sheet and cash flow
- The acquisition of Amicus closed on April 27, 2026.
- Interest expense from debt issued to finance a portion of the transaction reduced second-quarter GAAP net income and non-GAAP income.
- The company is targeting gross leverage below 2.5 times by mid-year 2027.
Analysis
BioMarin reported second-quarter total revenues of $990 million, up 20% from $825 million. The filing attributes the increase primarily to the April 27, 2026 acquisition of GALAFOLD and POMBILITI + OPFOLDA, new VOXZOGO patients across all regions, and growth in U.S. PALYNZIQ patients. VOXZOGO revenue was $253 million, up 14%, while PALYNZIQ revenue was $135 million, up 27%. Metabolic Conditions revenue grew 25% Y/Y, although the filing does not provide a reported dollar total for that category in the financial highlights table.
Growth was not uniform across the legacy portfolio. VIMIZIM revenue declined 10% to $194 million because of the timing of large government orders outside the U.S., and ALDURAZYME declined 21% to $44 million because of the timing of Sanofi order fulfillment. The company reported that patient counts increased both Y/Y and sequentially across all BioMarin-marketed therapies. For VOXZOGO, global children on therapy increased by more than 20% Y/Y, and the U.S. accounted for approximately 25% of quarterly VOXZOGO revenue.
Profitability declined following the Amicus transaction. GAAP net income fell to $45 million from $241 million and GAAP diluted EPS fell to $0.23 from $1.23. Non-GAAP income declined to $236 million from $282 million, while non-GAAP diluted EPS decreased to $1.20 from $1.44. GAAP operating margin was 11.2%, versus 33.5%, and non-GAAP operating margin was 36.4%, versus 39.9%. Management cited Amicus integration and restructuring costs, intangible asset amortization, debt-related interest expense, inventory fair value step-up amortization, higher sales and marketing spending, and higher BMN 401 R&D spending.
The company increased its full-year 2026 VOXZOGO revenue guidance to at least $1 billion, but the provided filing text does not include the numerical full-year total revenue or non-GAAP diluted EPS guidance range referenced in the headline. Management expects approximately $280 million of GAAP cost reductions and approximately $220 million of Non-GAAP cost reductions from Amicus, fully realized in 2028. It also targets gross leverage below 2.5 times by mid-year 2027 and expects GALAFOLD plus POMBILITI + OPFOLDA to reach over 60% Non-GAAP Operating Margin by 2030.
Pipeline developments include a submitted VOXZOGO hypochondroplasia sNDA, an FDA-accepted VOXZOGO achondroplasia sNDA with a February 28, 2027 PDUFA target action date, and continued enrollment in the BMN 333 Phase 2/3 study. The company discontinued BMN 401 across all indications after the ENERGY 3 trial did not meet one of two co-primary endpoints. The near-term operating focus is commercial integration of Amicus, delivery of the identified cost reductions, continued VOXZOGO patient expansion, and management's planned BMN 351 update by year-end.
Management, verbatim
Strong global demand led us to increase full-year VOXZOGO revenue guidance to at least $1 billion in 2026. Adding to this momentum is the opportunity to advance our second potential indication with VOXZOGO, hypochondroplasia, based on recent pivotal data that exceeded our expectations.
Alexander Hardy, President and Chief Executive Officer of BioMarin
With our larger, more diversified commercial portfolio of innovative medicines, we are positioned to deliver additional growth and increased profitability. We expect strong execution through the remainder of 2026, bringing together our expanded portfolio, scale and disciplined integration efforts to reach more patients living with serious genetic conditions around the world.
Alexander Hardy, President and Chief Executive Officer of BioMarin
Not in the filing
stated, not guessed- Reported second-quarter revenue for GALAFOLD, which is shown as #N/A in the financial highlights table.
- Reported second-quarter revenue for POMBILITI + OPFOLDA, which is shown as #N/A in the financial highlights table.
- Reported dollar amount for Total Metabolic Conditions Revenue, which is shown as #N/A in the financial highlights table.
- Reported GAAP operating income amount.
- Reported non-GAAP operating income amount.
- Gross margin.
- Operating expenses.
- Tax rate.
- Operating cash flow.
- Free cash flow.
- Cash and cash equivalents.
- Debt balance.
- Share repurchases.
- Dividends.
- Numerical full-year 2026 total revenue guidance.
- Numerical full-year 2026 non-GAAP diluted EPS guidance.
- Prior-quarter comparisons for reported metrics.
- Previous-release outlook for comparison with actual reported results.
AlphaAI analysis generated from the company’s SEC earnings filing (Form 8-K Item 2.02, or Form 6-K for a foreign private issuer). Every figure was cross-checked against the filing text; consensus estimates, price targets and share-price reactions are not shown because they are not in the filing. AI-generated research, not investment advice.
Background
This SEC 8-K (Item 2.02) includes BioMarin’s Q2 2026 financial and operating results and an accompanying earnings release exhibit.
Ticker impact
BioMarin’s 8-K reports Q2 results and raises full-year 2026 VOXZOGO revenue guidance to at least $1B, plus multiple pipeline/regulatory updates.
Bias toward upside or reduced downside risk versus prior guidance, with volatility likely around upcoming third-quarter VOXZOGO sNDA status update and later PDUFA milestones.
The filing is a primary disclosure with explicit guidance change and concrete regulatory/clinical milestones, which typically reprice expectations for revenue and probability-weighted pipeline value.
Market effects
Strength in rare-disease growth and expanding label/indication sets may support sentiment for specialty pharma and genetic-condition peers.
EU approval of PALYNZIQ for adolescents could reinforce European demand expectations for BioMarin’s metabolic franchise.
VOXZOGO hypochondroplasia and achondroplasia regulatory progress can shift global growth expectations for targeted therapies in skeletal dysplasias.
Counterpoint
Pipeline optimism may be partially offset by the decision to discontinue BMN 401 across indications, which can pressure longer-dated growth narratives.
Key entities
- companyBioMarin Pharmaceutical Inc.
Reports Q2 2026 results, raises 2026 VOXZOGO guidance, and updates multiple regulatory and clinical milestones across its rare-disease portfolio.
- productVOXZOGO
BioMarin’s therapy for skeletal dysplasias; guidance raised and hypochondroplasia sNDA submitted, with additional pivotal data cited.
- productPALYNZIQ
PKU therapy; European Commission approval expanded to adolescents 12 years and older.
- corporate_actionsAmicus acquisition
Integration and cost synergies are quantified, with expected realization by 2028.
