BioMarin Pharmaceutical Inc.
ABOUT
BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with multiple commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com.
770 Lindaro Street
San Rafael, CA 94901
105 Digital Drive
Novato, CA 94949
San Rafael, CA 94901
105 Digital Drive
Novato, CA 94949
NEWS
With Ascendis Pharma entering the achondroplasia space last month and BridgeBio on deck, BioMarin faces competition. Adding to the pressure, the company suffered a setback Monday when it halted two studies of Voxzogo in other growth-related conditions following multiple cases of hip injuries in other trials of the drug.
The rare disease drugmaker is facing potential competitors for achondroplasia drug Voxzogo. Is a big M&A deal with two approved assets enough to maintain investor interest?
In what is shaping up to be a back-loaded month, the FDA is set to release a slew of regulatory decisions in February, including two that would expand the labels of blockbuster drugs.
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BioMarin Pharmaceutical has faced a rocky road, promising and then backing off revenue targets and cutting assets that have underperformed. But Amicus’ rare disease portfolio is already bringing in $600 million annually.
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As third-quarter earnings continue to roll out, Novartis makes headlines with the second biggest acquisition of the year; Novartis’ CEO also downplayed the impact of Big Pharma pricing deals with the Trump administration; Regeneron continued the trend of dropping cell therapy assets; BioSpace takes a look at how the FDA is functioning mid-shutdown.
BioMarin no longer expects to hit its $4 billion revenue target by 2027, citing various market factors such as impending competition for achondroplasia therapy Voxzogo and the divestment of the gene therapy Roctavian.
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