US FDA grants breakthrough therapy status to Pfizer's Trumenba for prevention of invasive meningococcal B disease in children ages 1 to 9 years

New YorkTuesday, April 24, 2018, 15:00 Hrs  [IST]

Pfizer Inc. announced that Trumenba (Meningococcal Group B Vaccine) received Breakthrough Therapy designation from the US Food and Drug Administration (FDA) for active immunization to prevent invasive disease caused by Neisseria meningitidis group B (MenB) in children ages 1 through 9 years. This is the first Breakthrough Therapy designation for a MenB vaccine to help protect children as young as 1 year of age. Trumenba previously received Breakthrough Therapy designation in 2014 for the prevention of MenB in adolescents and young adults ages 10 through 25 years, and later the same year received FDA approval as the first MenB vaccine approved in the US.

As noted in the October 2014 Approval Letter, Pfizer was required to assess the safety and effectiveness of Trumenba in children down to 1 year of age. Pfizer has successfully completed Phase 2 studies in this investigational age group and these data have been submitted to the FDA. These data supported Pfizer’s request for Breakthrough Therapy designation.

“Despite the occurrence of invasive serogroup B disease in children ages 1 through 9 years, and the potential life-altering and long-term consequences that may result from this uncommon disease, there is no MenB vaccine licensed in the U.S. for this age group,” said Dr. Luis Jodar, chief medical and scientific affairs officer, vaccines medical development, scientific and clinical affairs, Pfizer Inc. “We look forward to working closely with the FDA toward our goal to extend the range of individuals who may benefit from immunization with Trumenba.”

In April 2017, Trumenba received traditional approval from the FDA in individuals 10 to 25 years of age for the three-dose schedule based on Phase 3 data, making it the only MenB vaccine in the U.S. with this full approval. Trumenba can be administered as a two- or three-dose schedule to adolescents and young adults 10 through 25 years of age depending on an individual’s risk of exposure and susceptibility to MenB. A study to confirm the effectiveness of the two-dose schedule is ongoing.

The majority of invasive meningococcal disease cases worldwide can be attributed to six Neisseria meningitidis serogroups (A, B, C, W, X, and Y). Together, serogroups A, B, C, W, and Y account for 90% of all invasive meningococcal disease (IMD), with MenB accounting for the majority of disease in adolescents and young adults in the US and Europe. As of 2016, the burden of MenB is highest in adolescents/young adults (32%) and infants (20%), followed by children ages 1 to 4 years (12%) and children ages 5 to 10 years (4%).

Breakthrough Therapy designation was initiated as part of the Food and Drug Administration Safety and Innovation Act (FDASIA) signed in 2012. As defined by the FDA, a breakthrough therapy is a drug intended to be used alone or in combination with one or more other drugs to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development. If a drug is designated as a breakthrough therapy, the FDA may expedite the development and review of such drug.

Trumenba is a vaccine indicated for individuals 10 through 25 years of age for active immunization to prevent invasive disease caused by Neisseria meningitidis (N meningitidis) group B. The effectiveness of the two-dose schedule of Trumenba against diverse N meningitidis group B strains has not been confirmed.

Trumenba (Meningococcal Group B Vaccine) is a sterile suspension composed of two recombinant lipidated factor H binding protein (fHBP) variants from N meningitidis serogroup B, one from fHBP subfamily A and one from subfamily B (A05 and B01, respectively). fHBP is one of many proteins found on the surface of meningococci and contributes to the ability of the bacterium to avoid host defenses. fHBPs can be categorized into two immunologically distinct subfamilies, A and B. The susceptibility of serogroup B meningococci to complement-mediated, antibody-dependent killing following vaccination with Trumenba is dependent on both the antigenic similarity of the bacterial and vaccine fHBPs, as well as the amount of fHBP expressed on the surface of the invading meningococci.