The FDA Giveth and Taketh Away: Grants Pfizer's MenB Breakthrough Therapy Designation and Turns Down a Biosimilar

The U.S. Food and Drug Administration (FDA) granted Pfizer’s Trumenba, a vaccine for meningococcal B disease in children ages one to nine years, Breakthrough Therapy Designation.

The vaccine is to prevent invasive diseases caused by Neisseria meningitides group B in children. The vaccine was approved in April 2017 for people 10 to 25 years of age. The vaccine received Breakthrough Therapy designation in 2014 for the prevention of MenB for that age group.

"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 Luis Jodar, Pfizer’s chief medical officer and scientific affairs officer, Vaccines Medical Development, Scientific and Clinical Affairs, in a statement. "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."

Most noninvasive meningococcal diseases worldwide come from six Neisseria meningitidesserogroups, A, B, C, W, X, and Y. A, B, C, W, and Y account for 90 percent of all cases, with MenB accounting for most of the disease in adolescents and young adults in the U.S. and Europe.

Perhaps the expansion of the vaccine will take some of the sting out of the FDA declining to approve its biosimilar of Roche’s Herceptin for breast cancer. The FDA required additional technical data on the biosimilar.

A biosimilar is a generic version of a biologics drug. Although not a direct copycat, it is biologically similar enough, but as a result, requires more clinical study and approvals than a direct copycat.

Herceptin generated $7.18 billion in sales last year for Roche. The FDA approved Mylan’s biosimilar for Herceptin in December. 

Apparently, the FDA did not request additional information related to safety or clinical data. In September 2017, Pfizer presented positive results for the biosimilar, PF-05280014, at the European Society for Medical Oncology. In its REFLECTIONS B327-02 trial, the biosimilar had an equivalent objective response rate compared to Herceptin in patients receiving first-line treatment in combination with paclitaxel for HER2-positive metastatic breast cancer.

On April 5, the FDA provided a Complete Response Letter (CRL) for Celltrion and Teva’s biosimilar for Herceptin.

The Center for Biosimilars writes, "These two regulatory setbacks for biosimilar trasfuzumab leave Mylan and BioCon’s Ogivri, which received the FDA’s approval in 2017, as the only approved trastuzumab biosimilar in the United States. No launch date for Ogivri has yet been announced, however."

Pfizer has 29 late-stage programs in its pipeline and another 18 phase II programs. Its pipeline is also broad, developed for numerous therapeutic areas, including inflammation and immunology, cancer, cardiovascular and metabolic diseases, rare diseases, and biosimilars and vaccines. Many programs are for new indications for already-approved drugs, such as Ibrance and Xeljanz, but others are new drugs, such as dacomitinib for lung cancer and talazoparib for prostate cancer. The company has projected that it could get approval for 15 possible blockbusters over the next five years.

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