BioMarin Pharmaceutical Inc.

BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery. In 1997, the company was founded to make a big difference in small patient populations, focusing on giving much-needed attention to the underserved communities of those with rare diseases. BioMarin sought to help patients whose voices could not always be heard and treat conditions that were not always in mainstream medical conversations. Throughout its history, BioMarin has worked tirelessly to make a difference by pursuing bold science. The company’s first approved therapy was inspired by a father who refused to accept there was no therapy for his son’s rare diagnosis. That relentless pursuit to change the course of his son’s future through the power of science is at the core of who BioMarin is today.

Through the company’s expertise in genetics and molecular biology, BioMarin has developed eight first or best-in-class targeted therapies that address the root cause of the conditions it seeks to treat, helping to better the lives of those struggling with rare genetic disease. The company’s purposeful and scientifically precise team of physicians and scientists has honed each treatment modality to seek transformative therapies.

Now, BioMarin seeks to make an even greater impact by applying the same science-driven, patient-forward approach that propelled the last 25 plus years of drug development to larger genetic disorders, as well as genetic subsets of more common conditions. Each drug candidate pursued is guided by a fundamental understanding of the genetics and underlying biology of the condition it will address. The company believes that applying its knowledge to make a transformative impact is not just a calling, but an obligation to those who will benefit most. The more innovative solutions developed, the more lives BioMarin can impact.

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NEWS
The biotech beat Wall Street’s third-quarter revenue forecast by 6%, driven by increased uptake of its achondroplasia drug Voxzogo. However, William Blair downgraded BioMarin’s shares to market perform due to a “lack of near-term catalysts” and uncertainty around Voxzogo’s potential revenue growth.
In 2023, the FDA greenlit 55 new drugs and 34 cell and gene therapies. Follow along as BioSpace keeps you up to date on all of the FDA’s decisions in 2024.
For the first eight months of 2024, California had the most job postings live on BioSpace—37.7% more than Massachusetts. Last year, Massachusetts ranked No.1 for the same time period.
BioMarin executives sought to calm an anxious investor base Wednesday with a public address and pledge to achieve a nearly 50% bump in annual revenue by 2027. But analysts were left wanting.
Last week, BioMarin revealed changes to its C-suite; now, the company has announced its second round of layoffs this year, following the termination of 170 employees in May.
As the biotech implements a more focused strategy for its Roctavian hemophilia A gene therapy, BioMarin has recruited two seasoned pharma executives to bolster its C-suite.
BioMarin Pharmaceuticals on Monday said it is restricting sales of its hemophilia A gene therapy to three countries in an effort to reduce costs and help the treatment become profitable by 2025.
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The annual observance highlights how far the industry has come and serves as a wake-up call for how far it must still go to meet the needs of patients with rare diseases.
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