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Investing.com - Jefferies maintained its Buy rating and $110.00 price target on BioMarin Pharmaceutical (TADAWUL:2070) Inc. (NASDAQ:BMRN) stock on Wednesday. Currently trading at $54.31, the stock has significant upside potential according to InvestingPro data, which shows analyst targets ranging from $65 to $122. The company maintains a "GREAT" financial health score and boasts a perfect Piotroski Score of 9, indicating strong operational efficiency.
The research firm highlighted BioMarin’s recent Citizen Petition asking the FDA to not approve Ascendis Pharma (NASDAQ:ASND)’s TransCon CNP before BioMarin’s Voxzogo orphan drug exclusivity expires in 2028 and 2030. The petition was submitted to the FDA on June 12, requiring a response within 180 days, which would fall on December 9, after Ascendis Pharma’s PDUFA date of November 30, 2025. This regulatory battle comes as BioMarin demonstrates strong financial performance, with revenue growing at 19.4% and maintaining healthy profit margins above 80%.
Jefferies estimates a 70/30 probability that BioMarin’s petition request will fail, with the firm modeling TransCon CNP market entry in 2026. Despite this assessment, the firm considers the regulatory challenge worth monitoring.
BioMarin has taken additional legal actions to protect its market position, filing a lawsuit against Ascendis in Germany with potential news expected around the first half of 2026. The company has also filed an International Trade Commission lawsuit aimed at preventing TransCon CNP importation into the United States.
The Voxzogo exclusivity battle represents a significant regulatory challenge for BioMarin as it works to maintain market protection for one of its key products against emerging competition. Despite trading near its 52-week low, InvestingPro analysis suggests the stock is undervalued, with strong fundamentals including a current ratio of 5.52 and moderate debt levels. For deeper insights into BioMarin’s market position and growth potential, access the comprehensive Pro Research Report, available exclusively to InvestingPro subscribers.
In other recent news, BioMarin Pharmaceutical Inc. reported sustained efficacy and safety results from its hemophilia A gene therapy, ROCTAVIAN, five years post-treatment. The Phase 3 GENEr8-1 trial highlighted that 81.3% of participants remained off prophylactic treatment, maintaining factor VIII activity levels in the mild hemophilia range. Additionally, BioMarin announced its acquisition of Inozyme Pharma for $270 million, a strategic move to bolster its enzyme therapy portfolio with INZ-701, a Phase 3 candidate for ENPP1 Deficiency. This acquisition aligns with BioMarin’s strategy to enhance its enzyme replacement therapies and is expected to close in the third quarter of 2025. Analyst firms, including Stifel and TD Cowen, have maintained Buy ratings on BioMarin, with price targets set at $91 and $120, respectively. Stifel’s confidence is supported by BioMarin’s growth potential in its drug portfolio, while TD Cowen views the Inozyme acquisition as a strategic fit. Bernstein also raised its price target for BioMarin to $95, maintaining an Outperform rating, citing the acquisition as a strong strategic fit. These developments reflect BioMarin’s ongoing commitment to advancing treatments for rare genetic diseases.
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