Ibruvica, a Drug Used to Treat Rare Blood Diseases, Has Been Granted Broader Approval by the FDA

FDA Expands Approval of Imbruvica to Treat Chronic Lymphocytic Leukemia

In Market Access, Regulation & Government by Cecile

Imbruvica, a Drug Used to Treat Rare Blood Diseases, Has Been Granted Broader Approval by the FDA

In 2014, the US FDA granted pharmaceutical companies Pharmacyclics and Janssen Biotech accelerated approval for the drug Imbruvica, under a designation to treat Chronic Lymphocytic Leukemia (CLL.) The FDA accelerated approval regulation allows for a drug that meets an unmet need for serious conditions to be approved based on a surrogate endpoint. A surrogate endpoint is a marker in the form of laboratory measurement, physical sign or other measure that id though to be able to predict clinical benefit. Studies that demonstrate a drug’s effect on a surrogate or intermediate clinical endpoint must be “adequate and well controlled” as required by the FD&C Act.

However, the approved use of the drug does not end here. Based on evidence from a recent clinical trial involving 391 CLL patients, the FDA has also approved its use to treat CLL patients with a specific 17p chromosomal deletion. Overall, whether the patient exhibited a 17p deletion or not, treatment with Imbruvica resulted in a 78 percent drop in disease progression or death, as well as a 57 percent drop in death risk.

Overall, whether the patient exhibited a 17p deletion or not, treatment with Imbruvica resulted in a 78 percent drop in disease progression or death, as well as a 57 percent drop in death riskImbruvica clinical trial

The initial approval granted by the FDA was based on very limited evidence, but as a result of the new clinical trial, the FDA was reassured that the drug performs well in terms of disease progression time and risk of death.

The FDA has also approved new labelling of the drug in light of this new approval, another success for the two companies concerned.

Dr.Richard Pazdur, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, has said that ‘Imbruvica is the fourth drug approved to treat CLL that received a breakthrough therapy designation, reflecting the promise of the breakthrough therapy designation program and demonstrating the FDA’s commitment to working cooperatively with companies to expedite the development, review and approval of these important new drugs.’

Read more about it here: FDA expands approved use of Imbruvica for chronic lymphocytic leukemia