Breakthrough Therapy Designation Is Granted by FDA for Pfizer's MenB Vaccine In Children

April 23, 2018

This is the first Breakthrough Therapy designation for a MenB vaccine to protect children as young as 1 year of age.

The US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to Pfizer Inc.’s meningococcal group B (MenB) vaccine (Trumenba) to protect children between the ages of 1 and 9 years against invasive disease.

This is the first Breakthrough Therapy designation for a MenB vaccine to protect children as young as 1 year of age, according to Pfizer’s recent media statement.

“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 United States for this age group,” Luis Jodar, PhD, Chief Medical and Scientific Affairs Officer, Vaccines Medical Development, Scientific and Clinical Affairs, Pfizer Inc., said in a recent statement.

Trumenba is a sterile suspension comprised of 2 recombinant lipidated factor H binding protein (fHBP) variants from Neisseria meningitidis serogroup B. Currently, 5 types of bacteria caused the most meningococcal disease in the US: A, C, Y, W and B.

According to the Centers for Disease Control and Prevention (CDC), even when the disease is treated, it’s estimated to kill 10 to 15 infected individuals out of 100. Meningitis B can become deadly within just 24 hours.

Ten to 20 of those who survive out of every 100 run the risk of suffering from disabilities including but not limited to loss of hearing, brain or kidney damage, amputations and nervous system-associated complications.

In 2014, the FDA granted Breakthrough designation to the MenB vaccine for the prevention of the disease in adolescents and young adults between 10 and 25 years of age. Later in the year, Trumenba received the green light from the FDA, making it the first MenB vaccine approved in the US.

Later, in April 2017, the FDA approved Trumenba for adolescents between 10 and 25 years of age for the 3-dose schedule based on phase 3 data.

Depending on each individual’s risk of exposure and susceptibility to MenB, the vaccine is designated to be administered in either 2- or 3-dose schedules for the aforementioned population. A study is currently being conducted to assess the effectiveness of the 2-dose schedule.

In an October 2014 approval letter, Pfizer was instructed to look into the safety and efficacy of the vaccine in children 1 year of age. After completing phase 2 studies in this population, Pfizer submitted their data to the FDA.

“These data supported Pfizer’s request for Breakthrough Therapy designation,” according to the statement.

A previous version of this article appeared on ContagionLive.