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Pink Sheet – Pfizer’s Meningitis B Vaccine Trumenba Is CBER’s First ‘Breakthrough’ Approval

Pink Sheet – Pfizer’s Meningitis B Vaccine Trumenba Is CBER’s First ‘Breakthrough’ Approval

FDA’s biologics center grants accelerated approval to the first U.S. vaccine targeting serogroup B meningococcal disease after a 4.5-month review; the vaccine is expected to come up for discussion, but not a vote, at CDC’s Advisory Committee on Immunization Practices meeting Oct. 30.

FDA clearance of Pfizer Inc.’s meningococcal group B vaccine Trumenba marks the Center for Biologics Evaluation and Research’s first approval of a product holding “breakthrough therapy” designation.

CBER granted accelerated approval to Trumenba Oct. 29 following a priority review that lasted just 4.5 months.

Trumenba is the first vaccine licensed in the U.S. to prevent invasive meningococcal disease caused by Neisseria meningitidis serogroup B. It is to be administered as a three-dose series over the course of six months in individuals ages 10-25 years old.

Labeling states that approval was based on a demonstration of immune response, as measured by serum bactericidal activity against four serogroup B strains that are representative of the prevalent strains in the U.S. Effectiveness of Trumenba against diverse serogroup B strains has not been confirmed.

As a condition of accelerated approval, Pfizer must complete ongoing studies to confirm the effectiveness of Trumenba against additional serogroup B strains.

Pfizer said it has been conducting a global clinical development program that includes Phase II and III trials evaluating more than 20,000 participants, approximately 14,000 of whom will receive the vaccine. The Phase III program began in November 2012 with the initiation of a large-scale safety study. Approximately 4,500 individuals received at least one dose of Trumenba and about 4,200 were in the indicated age group of 10-25 years of age. Additional immunogenicity and safety studies are ongoing.

Speedy Path To Market

Trumenba is one of just nine CBER-regulated products that have received breakthrough designation.

Created by the FDA Safety and Innovation Act of 2012, the breakthrough therapy program is aimed at expediting development and review of treatments for which preliminary clinical evidence suggests they may offer a substantial improvement over existing therapies for serious diseases.

The designation entitles sponsors to intensive guidance from FDA on an efficient development program, with an organizational commitment by the agency that includes senior managers.

The expedited regulatory program has proven enormously popular with industry, with FDA receiving more than 200 requests for breakthrough designation. However, the bulk of the activity has been located in the Center for Drug Evaluation and Research.

Through Oct. 17, CDER had granted 60 requests for breakthrough designation and denied 100 (“FDA ‘Breakthrough Therapy’ Designations” — Pharmaceutical Approvals Monthly, October 2014). Nine novel products and four supplemental indications with breakthrough designation have been approved by the drugs center.

In contrast, CBER had granted just seven breakthrough designation requests and denied 26 through Sept. 30, with no approvals.

Pfizer announced receipt of the breakthrough designation for Trumenba in March. The designation was based, in part, on data from two Phase II trials.

The company completed a rolling BLA submission in June (“Same-Day BLA Filings Show Pfizer Has Gained Ground On Novartis In Meningitis B” — “The Pink Sheet” DAILY, Jun. 18, 2014). Under a priority review, the user fee goal date was Feb. 14, 2015.

During an Oct. 29 press call, CBER Director Karen Midthun said the breakthrough designation primarily allowed for an intense engagement between FDA staff and Pfizer, “and I think it really was pivotal in helping focus on what was really needed in the IND stage to be able to actually move into the licensed application stage.”

FDA staff worked very closely with Pfizer to facilitate the review process and the agency completed its evaluation of the BLA as quickly as possible because of the public health importance of a vaccine against serogroup B meningococcal disease, Midthun said.

FDA also has designated Novartis AG’s meningococcal group B vaccine Bexsero for breakthrough status.

Following receipt of the breakthrough designation in April, Novartis announced the start of a rolling BLA submission in June.

Novartis said Bexsero is undergoing priority review. “Our submission file includes a comprehensive set of data from randomized clinical trials and real-world experience including safety results from more than 15,000 adolescents and adults at Princeton University and the University of California-Santa Barbara, who received the vaccine under a CDC-sponsored expanded use Investigational New Drug designation from the FDA following outbreaks in 2013 and 2014,” the company said.

Bexsero is licensed in 35 countries outside the U.S., including in the European Union, Australia and Canada.

ACIP Discussion Up Next

Prior to Trumenba, approved meningococcal vaccines in the U.S. covered four of the five main serogroups of N. meningitidis bacteria that cause the disease (A, C, Y and W). This includes two quadrivalent vaccines – Sanofi’s Menactra and Novartis’ Menveo. GlaxoSmithKline PLC’sMenHibrix protects against serogroups C and Y and Haemophilus influenzae type b.

“Recent outbreaks of serogroup B meningococcal disease on a few college campuses have heightened concerns for this potentially deadly disease,” Midthun said in a press release. “FDA’s approval of Trumenba provides a safe and effective way to help prevent this disease in the U.S.”

Pfizer told “The Pink Sheet” DAILY it is “working as quickly as possible to make Trumenba available within weeks to health care providers to administer the vaccine.”

“We look forward to participating in discussions with the Centers for Disease Control and Prevention’s Advisory Committee on Immunization Practices regarding potential meningococcal group B vaccination recommendations,” the company added.

Trumenba was approved on the first day of ACIP’s regularly scheduled, two-day meeting. The committee’s public agenda for the meeting states that an “information and discussion” session about Pfizer’s and Novartis’ serogroup B meningococcal vaccines is scheduled for the morning of Oct. 30, although there is no indication a vote will take place.…