The US Food and Drug Administration (FDA) has granted breakthrough therapy status to Pfizer’s vaccine candidate, bivalent rLP2086, currently being investigated for the prevention of invasive meningococcal disease due to Neisseria meningitidis serogroup B in people aged ten to 25.
Globally, there are between 20,000 and 80,000 cases per annum due to the disease caused by Neisseria meningitidis serogroup B.
Caused by Neisseria meningitidis serogroup B, the disease can result in death or significant long-term disabilities, including brain damage and hearing loss.
Breakthrough therapy status was based, in part, on data secured from two clinical trials studying the safety and immunogenicity of rLP2086.
Data from a Phase II trial demonstrated that the investigational rLP2086 vaccine induced bactericidal antibodies in healthy adolescents (aged 11-18 years) that were broadly active against meningococcal B bacteria.
Safety data from the trial also showed the vaccine had an acceptable safety profile in this healthy adolescent study population and supported the further evaluation of the vaccine in Phase III studies.
In addition, data from another randomised, placebo-controlled, single-blind Phase II trial of two and three-dose schedules of rLP2086 in healthy adolescents (aged 11-18 years), showed that the investigational vaccine had an acceptable safety profile.
FDA breakthrough therapy status is intended to speed-up development and review of potential new medicines for serious and life-threatening diseases.
Pfizer senior vice-president of vaccine R&D Emilio Emini said the company is developing this meningococcal B vaccine candidate to help protect adolescents and young adults against a difficult to diagnose and often deadly disease.
"We are encouraged by the FDA’s recognition of the need to prevent meningococcal B disease, and the breakthrough therapy designation highlights the urgent need for prevention of meningococcal B disease," Emini said.
The company is currently carrying out global clinical development programme for rLP2086, which includes both Phase II and Phase III trials assessing more than 20,000 participants and around 14,000 will receive the investigational vaccine.
The company intends to submit a biologics licence application (BLA) to the FDA for bivalent rLP2086 by mid-2014.
Invasive meningococcal disease can lead to serious disabilities and can be life-threatening for those infected.
Out of the five meningococcal serogroups (A, B, C, W-135 and Y) that have been responsible for the majority of meningococcal disease, serogroup B is the only one for that broadly protective vaccine is currently approved for in the US.