By: Karen Midthun, M.D.
Meningitis has been in the news recently because of outbreaks of a specific strain (called “serogroup B” or “MenB”) on college campuses. Infections caused by MenB are uncommon in the U.S, but can be very serious. According to the Centers for Disease Control and Prevention (CDC), 160 of the 500 cases of meningococcal disease in the U.S. in 2012 were caused by MenB. There are vaccines licensed (approved) in the U.S. to prevent meningitis, but none include this strain.
To address this critical public health need, FDA worked closely with CDC, in order for CDC to make an unapproved MenB vaccine available as quickly as possible to the universities where CDC determined outbreaks had occurred. This was accomplished under FDA’s expanded access program for investigational (or unapproved) products. The program allows the use of unapproved drugs or vaccines to treat or prevent serious or immediately life-threatening conditions when other options are not available.
FDA has been working closely with manufacturers pursuing the development and approval of MenB vaccines for the U.S. The approval of any vaccine in the U.S. is an extensive process that requires submission of a Biologics License Application (or BLA) by a manufacturer. FDA medical and scientific staff then perform a detailed review of data supporting the safety and effectiveness of the vaccine, and FDA staff inspect the quality of the manufacturing process. Because the potential usefulness of a preventative vaccine must be weighed against any unintended side effects, the evaluation of each submission includes a careful assessment of the benefits and risks to public health.
The agency has a variety of regulatory tools – breakthrough therapy designation, accelerated approval, the fast-track program, and priority review – that have enabled FDA to help make innovative and effective new treatment options available to patients more rapidly for serious conditions such as MenB. More information about these programs is available on FDA’s web site.
Although the law generally prohibits FDA from disclosing the existence of pending applications, Novartis has given the agency permission to disclose that the firm plans to submit a BLA for Bexsero (serogroup B meningococcal vaccine) for review in the second quarter of 2014. Pfizer, which also is developing a serogroup meningococcal B vaccine, issued a statement on March 20, 2014, in which it acknowledged receiving breakthrough designation from the FDA for its vaccine, and that it intends to submit an application for review by mid-2014.
FDA is committed to working with manufacturers to bring important medical products to patients as quickly as possible. The health and well-being of patients is our top priority.
Karen Midthun, M.D., is the director of FDA’s Center for Biologics Evaluation and Research