Laura Guido, PharmD
- Medical Director, Global Medical Affairs
- BioMarin Pharmaceutical Inc.
Established in 1997, BioMarin is a world leader in developing and commercializing first- or best-in-class therapies for rare genetic diseases. We take pride in going where the science leads us, pioneering breakthrough treatments for debilitating and life-threatening conditions where we can significantly improve upon the current standard of care.
Our culture revolves around the ethos that no disease should go untreated, and our people are driven to discover, develop, and commercialize medicines that give patients, their families, and their caregivers hope where there was little or none. We fuel our R&D engine by looking for opportunities that align with our strengths and competencies. And we relentlessly pursue exciting, early-stage science that has the potential to change the course of disease.
Voted one of America’s Best Midsize Employer by Forbes in 2019, our employees feel connected to their work at BioMarin because they believe in our purpose: the patient. The passion and dedication that our employees bring to work each day is a testament to the inspiration our patients provide, and the knowledge of the impact we can make in their lives.
Vosoritide is an investigational analog of C-type Natriuretic Peptide (CNP) under evaluation for the treatment of children with achondroplasia, the most common form of skeletal dysplasia.
Vosoritide is being tested in children whose growth plates are still “open,” typically those under 18 years of age. This is approximately 25 percent of people with achondroplasia. In the US, Europe, Latin America, the Middle East and APAC (except for Japan), there are currently no licensed medications for achondroplasia.
Achondroplasia is characterized by failure of normal conversion of cartilage into bone, which results in impaired bone growth. This condition is caused by a change in the fibroblast growth factor receptor 3 gene (FGFR3), a negative regulator of bone growth. Beyond disproportionate short stature, people with achondroplasia can experience serious health complications, including foramen magnum compression, sleep apnea, bowed legs, mid-face hypoplasia, permanent sway of the lower back, spinal stenosis and recurrent ear infections. Some of these complications can result in invasive surgeries such as spinal cord decompression and straightening of bowed legs. In addition, some studies show increased mortality at every age.
More than 80% of children with achondroplasia have parents of average stature and have the condition as the result of a spontaneous gene mutation. The worldwide incidence rate of achondroplasia is about one in 25,000 live births.
Vosoritide for Achondroplasia
The latest information on Vosoritide for Achondroplasia. Vosoritide is an investigational analog of C-type Natriuretic Peptide (CNP) under evaluation for the treatment of children with achondroplasia, the most common form of skeletal dysplasia.