SAN RAFAEL, Calif.—BioMarin Pharmaceutical Inc. today announced that CenterWatch, a leading source for global clinical trial information, has named it one of the fastest drug developers in the industry. CenterWatch reviewed 307 therapies approved between 2000 and 2013 and concluded that BioMarin ranked within the top five in all categories. The three assessed categories were clinical duration, NDA approval duration and total duration of approval. BioMarin was also among the fastest companies named in drug development for endocrine disorders.
CenterWatch states its mission is to be the leading source of clinical trials information for both clinical research professionals and patients. To support its mission, the group offers several professional, educational and informative services and resources, from news and analysis on the industry to trial listings seeking study volunteers
Its analysis of drug development showed that BioMarin took a median 61 months to bring a treatment through the entire regulatory process, from IND filing to approval. With regard to clinical duration, the time to take a therapy from IND filing through to NDA submission, the company was able to complete the process in a median 55 months, while needing just six months to complete the NDA approval process. BioMarin was also among the top five fastest companies in endocrine development with a median development time of 2.6 years. This compares to a median clinical phase of 5.3 years for endocrine development for all companies analyzed.
"The analysis by CenterWatch showed that BioMarin develops treatments 12 months faster than the industry median. For the patients we seek to treat, those with ultra-rare conditions, one year can make a lifetime of a difference," said Hank Fuchs, M.D., chief medical officer of BioMarin in a press statement. "We thank CenterWatch for conducting this analysis, which truly underscores our continued commitment to developing therapies that address unmet medical needs as quickly and efficiently as possible."
The results of the analysis were published in the September issue of CenterWatch Monthly, a newsletter providing clinical trial information to patients and professionals. This is the second such analysis conducted by CenterWatch. The September issue has not yet been published on the CenterWatch website.
“BioMarin leads the biotechnology industry in delivering therapies for chronic and degenerative genetic conditions that reduce patients' quality of life and shorten life spans,” states company CEO Jean-Jacques Bienaimé. “We target diseases that lack effective therapies and affect relatively small numbers of patients, many of whom are children. As we enter 2014, BioMarin will continue to focus on advancing therapies that are the first or best of their kind and make a big difference in improving patients' lives.”
“The last 18 months have been a phenomenal time for BioMarin, Bienaimé continues. “With the approval of VIMIZIM® (elosulfase alfa), the first and only specific treatment for Morquio A or MPS IVA, we fulfilled a significant unmet medical need for patients and their families and firmly established our leadership in developing therapies to treat MPS diseases.”
“With VIMIZIM, we believe we can bring meaningful and unprecedented benefits to patients related to improvement in basic activities of daily living, such as walking or time away from a wheelchair.”
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises five approved products and multiple clinical and pre-clinical product candidates. Approved products include: Naglazyme® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; KUVAN® (sapropterin dihydrochloride) Powder for oral solution and tablets for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; Firdapse® (amifampridine), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS); and VIMIZIM(R) (elosulfase alfa) for the treatment of Morquio A (MPS IVA).