BioMarin Reports 28% Q3 Revenue Growth and Raises 2024 Guidance; Confirms Long-term Outlook

In the third quarter of 2024, BioMarin Pharmaceutical Inc. reported notable financial performance, with total revenues reaching $746 million, a 28% increase year-over-year, and an impressive growth of 32% at constant currency. The company’s year-to-date revenues amounted to $2.11 billion, marking a 19% rise year-over-year and 23% at constant currency. The quarter saw significant demand for the drug VOXZOGO, which led to a 54% year-over-year revenue growth.

During this period, BioMarin's enzyme therapies portfolio also experienced a robust 27% year-over-year revenue increase. The company reported a GAAP diluted earnings per share (EPS) of $0.55 for the third quarter, which is a substantial 162% increase year-over-year. Additionally, the non-GAAP diluted EPS for the same period rose to $0.91, reflecting a 98% year-over-year growth. Year-to-date, the GAAP diluted EPS was $1.56, up by 103%, while the non-GAAP diluted EPS reached $2.60, an increase of 63%.

Alexander Hardy, President and CEO of BioMarin, attributed the strong performance to strategic decisions and operational efficiency. He highlighted the company’s focus on innovation, growth, and value commitment as key drivers. Hardy noted the substantial global demand for VOXZOGO, with over 3,800 infants and children undergoing treatment by the end of the third quarter. In the U.S., the therapy saw significant adoption among children under five.

BioMarin's enzyme therapies portfolio also showed promising results, demonstrating the portfolio's growth potential. Hardy emphasized the progress in developing innovative therapies, including advancing studies in five new indications with VOXZOGO within the CANOPY clinical program. The company remains on track to complete the Phase 3 registration-enabling study for hypochondroplasia by the first half of 2025.

Financially, the third quarter saw a substantial increase in GAAP net income by $66 million, reaching $106 million, primarily driven by higher gross profit. This increase was partially offset by higher expenses in Selling, General and Administrative (SG&A) due to severance and restructuring costs, increased income tax expenses, and the impact of ROCTAVIAN inventory reserves on the cost of sales.

Non-GAAP income also rose significantly by $89 million, totaling $178 million for the third quarter. This increase was attributed to higher gross profit and lower SG&A expenses related to sales and marketing activities for ROCTAVIAN outside of the U.S., Germany, and Italy. However, it was partially offset by higher income tax expenses and the impact of ROCTAVIAN inventory reserves on the cost of sales.

BioMarin’s strategic focus on innovation was evident with progress in developing treatments for skeletal conditions. The company advanced its VOXZOGO studies for idiopathic short stature, Noonan syndrome, Turner syndrome, and SHOX deficiency. It also remains on track for initiating the first-in-human study for its long-acting C-type natriuretic peptide (CNP), BMN 333, in early 2025.

Additionally, BioMarin participated in the 16th International Skeletal Dysplasia Society meeting, contributing to presentations that highlighted VOXZOGO's impact on children with achondroplasia. The data presented showed significant improvements in health-related quality of life and increased bone length while maintaining bone strength.

Other clinical pipeline programs also showed progress. The BMN 351 program for Duchenne Muscular Dystrophy completed enrollment in the first dose cohort, with initial data expected in 2025. The BMN 349 program for Alpha-1 antitrypsin deficiency-associated liver disease completed the single-ascending dose phase and is set to start the multiple-ascending dose phase by the year’s end. The Phase 3 study with PALYNZIQ in adolescents is on track for a data readout in 2025 to support a potential U.S. Supplemental Biologics License Application in the second half of the year.

BioMarin’s commitment to growth was further demonstrated by over 3,800 children globally receiving VOXZOGO for achondroplasia treatment by the end of the third quarter. The company continues to pursue VOXZOGO access in more than 20 additional countries by 2027, aiming to broaden its reach.

In terms of financial strategy, BioMarin made significant strides, with an ongoing $500 million cost transformation program. The company generated $221 million in operating cash flows in the third quarter, a 63% increase year-over-year. Total cash and investments at the end of the quarter were approximately $1.5 billion. BioMarin settled $495 million of convertible debt during the quarter, leading to the retirement of approximately four million potentially dilutive shares.

BioMarin also updated its full-year 2024 guidance, reflecting sustained VOXZOGO demand and enzyme therapies growth. The company reaffirmed its long-term outlook, targeting $4 billion in total revenues by 2027 and a 40% non-GAAP operating margin starting in 2026. BioMarin aims to generate over $1.25 billion in annual operating cash flow from 2027 and expects a mid-teen compound annual growth rate (CAGR) for total revenues through 2034.