BioMarin Pharmaceutical Inc. today announced its financial results for the first quarter ended March 31, 2025.

We experienced robust demand for our innovative therapies during the first quarter, driving strong revenue growth and profitability, stated Alexander Hardy, President and Chief Executive Officer of BioMarin. "Our pipeline programs also progressed as planned. In April, we announced positive top-line results from the Phase 3 PALYNZIQ® trial for adolescents with phenylketonuria, and we successfully completed enrollment in the pivotal VOXZOGO® study for hypochondroplasia, keeping us on track for a potential 2027 launch, pending favorable data.

Hardy added, As we look to the remainder of 2025, we anticipate continued momentum in the global expansion of VOXZOGO for achondroplasia. We also plan to build on PALYNZIQ's strong quarterly performance and pursue initiatives to increase access to our other enzyme therapies worldwide. Beyond a strong financial outlook, we aim to advance multiple VOXZOGO indications through our CANOPY program, share early clinical data from BMN 351 for Duchenne muscular dystrophy and BMN 333, our long-acting C-type natriuretic peptide, and continue executing on our business development strategy. We are delivering meaningful growth and profitability while advancing BioMarin’s evolving strategy and operating model, and we expect to see the benefits of this transformation reflected in future results.

First Quarter 2025 Financial Highlights

Total Revenues reached $745 million, up 15% year-over-year, driven primarily by a 40% increase in VOXZOGO revenues due to strong global patient uptake. Revenue from BioMarin’s enzyme therapies ALDURAZYME®, BRINEURA®, NAGLAZYME®, PALYNZIQ®, and VIMIZIM® grew 8% year-over-year, reflecting increased patient demand and the timing of significant government orders, partially offset by continued erosion in KUVAN® sales due to generic competition.

GAAP Net Income rose by $97 million to $186 million, a 109% increase compared to Q1 2024, primarily reflecting higher gross profit and lower operating expenses following the discontinuation of select early-stage programs after BioMarin’s 2024 portfolio review.

Non-GAAP Income increased by $81 million to $221 million, representing 58% growth, driven by the same factors.

First Quarter 2025 Business Highlights

Pipeline and Innovation

Skeletal Conditions: In March, BioMarin presented real-world data demonstrating favorable safety and high treatment adherence with VOXZOGO in children under age 3 with achondroplasia at the ACMG Annual Meeting. No treatment-related adverse events or dose interruptions were reported among 63 children monitored for up to nearly two years. These findings reinforce VOXZOGO’s established safety profile and support early treatment initiation, consistent with recent international guidelines published in Nature Reviews Endocrinology.

In April, BioMarin completed enrollment in its Phase 3 VOXZOGO trial for hypochondroplasia, with top-line results expected in 2026 and a potential launch in 2027. The CANOPY program also continues to explore VOXZOGO for additional indications, including idiopathic short stature, Noonan syndrome, Turner syndrome, and SHOX deficiency.

BMN 333, BioMarin’s long-acting CNP, advanced in its first-in-human study, with initial PK data anticipated by year-end and detailed results expected in early 2026. Following alignment with the FDA on a clinical development plan for achondroplasia, BioMarin plans to initiate a registration-enabling study in 2026, targeting approval by 2030. Similar regulatory engagements are planned globally.

In April, BioMarin announced its pivotal PALYNZIQ trial for adolescents met its primary endpoint, supporting regulatory submissions in the U.S. and Europe in the second half of 2025 to expand the approved age range.

Other Clinical Programs

BMN 351, an oligonucleotide therapy for Duchenne muscular dystrophy, and BMN 349, an oral treatment for AATD-associated liver disease, continue to advance. Initial BMN 351 data—including muscle dystrophin levels—will be presented in late 2025.

Following a strategic review, BioMarin discontinued development of BMN 370 for von Willebrand disease, as it no longer met the company’s development criteria. Affected employees have been reassigned within BioMarin.

Source: https://investors.biomarin.com/news/news-details/2025/BioMarin-Reports-First-Quarter-2025-Results-and-Reaffirms-Full-year-Guidance/default.aspx