Print“Imbruvica’s approval demonstrates the FDA’s commitment to making treatments available to patients with rare diseases, says Richard Pazdur of FDA CDER”
The U.S. Food and Drug Administration approved Pharmacyclics’ and Johnson & Johnson’s Imbruvica to treat patients with mantle cell lymphoma who have received at least one prior therapy. The approval, which came more than three months ahead of schedule, is the second drug to be approved under the Breakthrough Therapy designation.
Approval of Imbruvica to treat mantle cell lymphoma is the first, and rarest, of three cancers the drugmakers hope to gain the agency’s authorization to treat. An FDA decision on Imbruvica as a treatment for the more common refractory chronic lymphocytic leukemia or small cell lymphocytic lymphoma is still pending. If that happens, the drug could generate sales of $2.1 billion by 2020, according to EP Vantage.
The Breakthrough Therapy designation is awarded to experimental drugs if preliminary clinical evidence suggests they may offer a substantial improvement over available therapies for patients with serious or life-threatening diseases. Imbruvica was granted Breakthrough Therapy designation for all three of the indications Pharmacyclics and Johnson & Johnson are seeking approval to market it for. Genentech’s Gazyva was approved on November 1 as a first line treatment for chronic lymphocytic leukemia in combination with chlorambucil. It was the first drug to win approval as a Breakthrough Therapy.
“Imbruvica’s approval demonstrates the FDA’s commitment to making treatments available to patients with rare diseases,” says Richard Pazdur, director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research. “The agency worked cooperatively with the companies to expedite the drug's development, review and approval, reflecting the promise of the Breakthrough Therapy Designation program.”
Mantle cell lymphoma, a rare and aggressive type of blood cancer, represents about 6 percent of all non-Hodgkin lymphoma cases in the United States. By the time it is diagnosed, it usually has already spread to the lymph nodes, bone marrow and other organs.
Imbruvica is a first-in-class inhibitor of Bruton’s tyrosine kinase, or BTK, a key signaling molecule that plays an important role in the survival of malignant B cells. The accelerated approval was based on a mid-stage study where 111 participants were given Imbruvica daily until their disease progressed or side effects became intolerable. Results showed nearly 66 percent of participants had their cancer shrink after treatment, with tumors disappearing in 17 percent of patients. An improvement in survival or disease-related symptoms has not been established.
Imbruvica is the third drug approved to treat mantle cell lymphoma. Millenium’s Velcade and Celgene’s Revlimid are also approved to treat the disease. Pharmacyclics and Johnshon & Johnson are co-marketing Imbruvica in the United States and sharing equally in all development and commercialization costs and profits worldwide.
November 15, 2013
http://www.burrillreport.com/article-fda_gives_ok_for_imbruvica_to_treat_rare_blood_cancer.html
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