Company Reports

BioMarin posted second-quarter revenue of $990 million, a 20% increase from the prior year. Management raised its 2026 revenue outlook by 65 basis points to a midpoint of $3.9 billion and adjusted earnings per share by 100 basis points to a midpoint of $5 per share. Shares rose nearly 4% on Aug. 7.

BioMarin reported positive phase 3 data for Voxzogo in children with hypochondroplasia, a rare short‑stature condition. The study met its primary endpoint as patients grew 2.33 centimeters per year faster than placebo and showed significant improvements in arm span. Shares rose nearly 8% on May 21.

BioMarin posted $766 million in first-quarter revenue, up 3% year over year. Management lifted its 2026 enzyme therapy outlook to $2.75 billion at the midpoint (representing 30% growth over 2025) after closing the Amicus acquisition. Shares fell 4% on May 5.

BioMarin generated $3.2 billion in 2025 revenue, up 13% year over year, led by 26% growth for achondroplasia drug Voxzogo and 9% growth for its enzyme therapies. For 2026, management is guiding to $1 billion in Voxzogo sales at the midpoint, implying 8% growth.

BridgeBio reported phase 3 top-line results for infigratinib, an oral drug for achondroplasia, showing strong efficacy and safety, and posing a competitive challenge to BioMarin's approved Voxzogo. On Feb. 12, BridgeBio shares closed nearly 4% up while BioMarin shares closed flat on the news.

BioMarin plans to acquire Amicus Therapeutics for $14.50 per share in an all-cash transaction, representing a 33% premium to Amicus' last close for a total equity value of $4.8 billion. BioMarin's stock rose nearly 18% on Dec.19, and the deal is expected to close in the second quarter of 2026.

BioMarin is amassing a portfolio of rare genetic-disease therapies, making historical comparisons with Genzyme (acquired by Sanofi) difficult to avoid. Commercialization and research and development expenses kept BioMarin in the red for years despite multiple approved products, but we're confident in the durable, profit-generating power of its current portfolio. With a deep in-house pipeline and the ability to supplement growth with strategic acquisitions, BioMarin is in a strong position.

BioMarin's third-quarter revenue of $776 million increased 4% from the prior-year period. Management said it intends to divest approved hemophilia gene therapy Roctavian. BioMarin raised its 2025 total revenue guidance by 40 basis points at the midpoint.

BioMarin is amassing a portfolio of rare genetic-disease therapeutics, making historical comparisons with Genzyme (acquired by Sanofi) difficult to avoid. Commercialization and research and development expenses kept BioMarin in the red for years despite multiple approved products, but we're confident in the durable, profit-generating power of its current portfolio. With a deep in-house pipeline and the ability to supplement growth with strategic acquisitions, BioMarin is in a strong position.

BioMarin delivered second-quarter revenue of $825 million, representing a 16% increase from the prior year period. GAAP net income increased to $241 million in the second quarter, representing 125% growth year over year.

Narrow-moat BioMarin reported robust first-quarter results highlighted by revenue of $745 million, representing growth of 15% year over year. BioMarin is seeing the benefits of its cost transformation initiatives that were implemented in 2024, as it delivered 30% GAAP operating margin in the first quarter, expanding significantly from the prior-year period’s 14% operating margin. BioMarin is tracking our expectations, and we maintain our fair value estimate of $83 per share. We view shares as undervalued, currently trading in 4-star territory.

Narrow-moat BioMarin reported strong 2024 results highlighted by revenue of $2.85 billion and a healthy GAAP operating margin of nearly 17%, demonstrating the company’s focus on operational efficiency. Investors reacted positively and sent the stock up 5%. BioMarin is tracking our expectations, and we maintain our fair value estimate of $83 per share. We view shares as undervalued, currently trading at a 17% discount.

BioMarin is amassing a portfolio of genetic-disease therapeutics, making historical comparisons with Genzyme (acquired by Sanofi) difficult to avoid. Commercialization and research and development expenses kept BioMarin in the red for years despite multiple approved products, but we're confident in the durable, profit-generating power of its current portfolio. With a deep in-house pipeline and the ability to supplement growth with strategic acquisitions, BioMarin is in a strong position.

BioMarin is amassing a portfolio of genetic-disease therapeutics, making historical comparisons with Genzyme (acquired by Sanofi) difficult to avoid. Commercialization and research and development expenses kept BioMarin in the red for years despite multiple approved products, but we're confident in the durable, profit-generating power of its current portfolio. With a deep in-house pipeline and the ability to supplement growth with strategic acquisitions, BioMarin is in a strong position.

We are maintaining our $83 fair value estimate for narrow-moat BioMarin following a strong third-quarter performance. The company reported a 28% increase in top-line revenue, driven primarily by 54% growth in Voxzogo's revenue and 27% increase in its enzyme therapies portfolio. In light of these results, management has updated its 2024 full-year revenue guidance to a range of $2.790 billion to $2.825 billion, indicating projected growth of 16% at the midpoint. It also reaffirmed a long-term outlook that surpasses our forecasts, projecting $4 billion in sales by 2027, compared to our estimate of $3.75 billion.

BioMarin shares fell nearly 18% on Sept. 16 following solid data from a potential competitor to the firm’s key growth driver, achondroplasia drug Voxzogo. We’re slightly dialing back our fair value estimate to $83 per share, from $87, after assuming more significant competition that should slow BioMarin’s market share gains. Based on pivotal data, Ascendis’ TransCon CNP appears capable of generating similar increases in growth velocity as Voxzogo over a one-year period but with faster and clearer benefit to proportionality. In addition, the once-weekly injection could be more convenient for patients than the once-daily Voxzogo injections. Ascendis plans to file for approval in 2025, suggesting this could be a meaningful competitor by 2027. That said, we think BioMarin shares are trading at a slight discount to our new fair value estimate after this news. Our valuation does not include any explicit forecast for revenue from the firm’s pipeline over the next 10 years, and our revenue and operating margin assumptions over this period remain below the guidance outlined at the firm’s Sept. 4 investor day. We continue to see BioMarin’s portfolio of rare disease drugs supporting a narrow moat.

BioMarin is amassing a portfolio of genetic-disease therapeutics, making historical comparisons with Genzyme (acquired by Sanofi) difficult to avoid. Commercialization and research and development expenses kept BioMarin in the red for years despite multiple approved products, but we're confident in the durable, profit-generating power of its current portfolio. With a deep in-house pipeline and the ability to supplement growth with strategic acquisitions, BioMarin is in a strong position.

We’re maintaining our $87 fair value estimate for BioMarin after a strong second quarter and clarity surrounding the commercial strategy for hemophilia A gene therapy Roctavian. Overall, our increased expectations for margin improvement and slight increase to our revenue expectations for BioMarin’s portfolio were countered by our decision to further reduce our Roctavian sales assumption. BioMarin saw 20% revenue growth in the second quarter, driven by 62% growth of achondroplasia drug Voxzogo and 15% growth for BioMarin’s portfolio of enzyme therapies. We were most impressed with BioMarin’s nearly 10-percentage-point improvement in non-GAAP operating margin to 31%, driving an increase in management guidance for the full-year margin to a range of 26%-27% (roughly 7 percentage points higher than 2023). While much of the second-quarter margin was tied to Voxzogo stocking and government enzyme therapy orders pulled forward from the third quarter, non-GAAP operating margin was still an impressive 28% excluding these tailwinds, showing fundamental improvement. We think BioMarin’s solid Voxzogo prospects and clearer focus help support its narrow moat and that shares look fairly valued at recent prices.

BioMarin is amassing a portfolio of genetic-disease therapeutics, making historical comparisons with Genzyme (acquired by Sanofi) difficult to avoid. Commercialization and research and development expenses kept BioMarin in the red for years despite multiple approved products, but we're confident in the durable, profit-generating power of its current portfolio. With a deep in-house pipeline and the ability to supplement growth with strategic acquisitions, BioMarin is in a strong position.

BioMarin Pharmaceutical's nearly $650 million in first-quarter revenue (9% growth) puts the company on track to reach management’s $2.7 billion-$2.8 billion revenue guidance for the full year, with achondroplasia drug Voxzogo continuing to see impressive growth as more children initiate therapy. However, the launch of hemophilia gene therapy Roctavian is still stalled due to reimbursement issues in the US and Europe. We’ve reduced our Roctavian forecast to less than $500 million in peak annual sales, although we recognize that BioMarin could opt to divest the program; management will outline its long-term strategy at the investor day in September. BioMarin is also culling its pipeline in an effort to focus on the most impactful programs, leading management to reduce guidance for research and development expenses this year and increase guidance for operating margins and earnings per share. All of the programs that were discontinued were too early to be explicitly included in our valuation model. After these adjustments, we’re reducing our fair value estimate to $87 per share from $96 and think the shares look fairly valued. We think BioMarin’s current rare-disease portfolio already warrants a narrow moat, but we’re watching for advancement of Voxzogo in new indications and data from earlier-stage pipeline drugs as potential catalysts for raising our fair value estimate.