VOXZOGO shows promise in tibial bowing treatment

SAN RAFAEL, Calif. - BioMarin Pharmaceutical Inc. (NASDAQ:BMRN), a pharmaceutical company with exceptional financial health and a perfect Piotroski Score according to InvestingPro, revealed new data indicating that VOXZOGO (vosoritide) may have a significant impact on tibial bowing in children with achondroplasia, a common cause of pain and impaired function in this population. These findings were presented at recent endocrinology meetings in Copenhagen and Maryland.

An analysis from the VOXZOGO Phase 2 CANOPY clinical studies showed that children with achondroplasia who received VOXZOGO experienced a significant reduction in tibial bowing compared to those given a placebo. With an impressive gross profit margin of 81% and strong revenue growth of 19.4% over the last twelve months, BioMarin continues to demonstrate robust commercial execution. Notably, these improvements were sustained over several years of treatment. Michael Hughes of Little People of America emphasized the importance of addressing outcomes beyond height, such as tibial bowing, which can require surgery for some children.

Dr. Klane White, a pediatric orthopedic surgeon, noted the meaningful improvements in proportionality, quality of life, and tibial bowing observed in children treated with VOXZOGO. Additionally, new modeling data suggest that early and continuous treatment with VOXZOGO could lead to an increase in final adult height by up to 26.4 cm in boys and 21.7 cm in girls compared to untreated children.

Furthermore, research on hypochondroplasia, a form of skeletal dysplasia, showed that individuals with the condition had higher rates of comorbidities, surgeries, and doctor visits. BioMarin’s Dr. Greg Friberg highlighted the significance of early diagnosis and intervention.

VOXZOGO is also being evaluated for other skeletal conditions, including Turner syndrome. Early results from a Phase 2 study indicate a promising increase in annualized growth velocity, even in children who had previously seen suboptimal results from human growth hormone treatment. With a strong current ratio of 5.52 and moderate debt levels, InvestingPro analysis suggests BioMarin is well-positioned to continue investing in its development pipeline. For detailed insights and additional ProTips about BioMarin’s financial outlook, investors can access the comprehensive Pro Research Report, available exclusively to InvestingPro subscribers.

BioMarin is advancing its CANOPY clinical program for VOXZOGO across various conditions, with a pivotal Phase 3 study for hypochondroplasia completed in April and topline data expected in 2026.

VOXZOGO is approved in multiple regions for children with achondroplasia and open epiphyses, with its U.S. approval based on improved annualized growth velocity. It is administered daily via injection, and its continued approval may be contingent on further clinical benefit verification.

The information in this article is based on a press release statement from BioMarin Pharmaceutical Inc. According to InvestingPro analysis, BioMarin’s stock currently appears undervalued, presenting a potential opportunity for investors interested in the biotechnology sector. Discover more undervalued opportunities at Investing.com’s Most Undervalued Stocks.

In other recent news, BioMarin Pharmaceutical Inc. reported strong financial results for the first quarter of 2025, exceeding Wall Street’s expectations. The company posted earnings per share (EPS) of $1.13, significantly higher than the forecasted $0.70, and achieved revenue of $745 million, surpassing the anticipated $741.02 million. BioMarin confirmed its full-year EPS guidance to be in the range of $4.20 to $4.40, indicating a year-over-year growth of 19-25%. Cantor Fitzgerald maintained an Overweight rating on BioMarin with a $90 price target, highlighting the company’s consistent performance and positive growth outlook. The firm’s analysts anticipate that BioMarin will continue to experience double-digit earnings growth through 2029. Additionally, the company’s operating margins are projected to improve, with expectations of reaching between 31% and 33% by 2025. BioMarin remains optimistic about its future prospects, aiming for a $4 billion revenue target by 2027. These developments underscore BioMarin’s strategic focus on innovation and expanding its market presence.

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