BioMarin is committed to the development of new therapies for serious diseases and medical conditions. Successful clinical trials are required to gain Regulatory Agency (Food and Drug Administration (FDA) in the US) approval for new medications to advance patient care, as well as to support existing products.
A clinical trial is a research study done to evaluate new potential treatments in people. During a clinical trial, information is collected to determine if a study drug is safe and effective, as well as to evaluate the risks and benefits of the study drug. For more information about clinical trials in general, please click here
Pre-clinical testing is conducted in the laboratory and in animals to evaluate the safety and efficacy of a study drug before it is given to humans.
An IND (Investigational New Drug) application is submitted to the regulatory agency (eg FDA) so that they can evaluate the study drug for safety to assure that human research subjects will not be subjected to an unreasonable risk
Clinical trials are conducted in 4 phases. Each trial phase has a different purpose and helps answer different questions:
Phase 1: a study drug is tested in people for the first time to evaluate its safety
Phase 2: the study drug is tested to determine a safe dose, to further evaluate its safety, and to begin testing if it is effective
Phase 3: the study drug is further tested to confirm if it is effective, further evaluate safety, and compare it to commonly used treatments or placebos
When the above studies are completed, a New Drug Application (NDA) is submitted to the regulatory agency (eg FDA). The NDA contains all data gathered about the safety and effectiveness of the study drug from the above studies. The regulatory agency (eg FDA) reviews the data and, if approved, the new treatment can be marketed and distributed to the public.
Biologic License Application (BLA) is submitted to the regulatory agency (eg FDA) and contains specific information on the manufacturing processes, chemistry, pharmacology and the medical effects of the biologic product. If the information provided meets regulatory requirements, the application is approved and a license is issued allowing the company to market the product.
Phase 4: post marketing studies are performed after regulatory agency (eg FDA) approval to collect additional information including the drug's risks, benefits, and optimal use in a broader patient population. An example would be a Registry to collect medical information on the disease population, whether they are taking the drug or not
For the clinical trials that BioMarin is currently conducting, please see:
- Genetically defined cancer
- MPS IVA (mucopolysaccharidosis IVA), also known as Morquio A Syndrome
- MPS VI (mucopolysaccharidosis VI), also known as Maroteaux-Lamy Syndrome