`Protecting and Promoting Your Health
`
`FDA NEWS RELEASE
`For Immediate Release: Nov. 13, 2013
`Media Inquiries: Stephanie Yao, 3017960394, stephanie.yao@fda.hhs.gov
`(mailto:stephanie.yao@fda.hhs.gov)
`Consumer Inquiries: 888INFOFDA
`
`FDA approves Imbruvica for rare blood cancer
`Second drug with breakthrough therapy designation to receive FDA approval
`
`The U.S. Food and Drug Administration today approved Imbruvica (ibrutinib) to treat patients with
`mantle cell lymphoma (MCL), a rare and aggressive type of blood cancer.
`
`MCL is a rare form of nonHodgkin lymphoma and represents about 6 percent of all nonHodgkin
`lymphoma cases in the United States. By the time MCL is diagnosed, it usually has already spread
`to the lymph nodes, bone marrow and other organs.
`
`Imbruvica is intended for patients with MCL who have received at least one prior therapy. It works
`by inhibiting the enzyme needed by the cancer to multiply and spread. Imbruvica is the third drug
`approved to treat MCL. Velcade (2006) and Revlimid (2013) are also approved to treat the
`disease.
`
`“Imbruvica’s approval demonstrates the FDA’s commitment to making treatments available to
`patients with rare diseases,” said Richard Pazdur, M.D., 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.”
`
`Imbruvica is the second drug with breakthrough therapy designation to receive FDA approval. The
`Food and Drug Administration Safety and Innovation Act, passed in July 2012, gave the FDA the
`ability to designate a drug a breakthrough therapy at the request of the sponsor if preliminary
`clinical evidence indicates the drug may offer a substantial improvement over available therapies
`for patients with serious or lifethreatening diseases.
`
`The FDA is approving Imbruvica under the agency's accelerated approval program, which allows
`the FDA to approve a drug to treat a serious disease based on clinical data showing that the drug
`has an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to
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`Press Announcements > FDA approves Imbruvica for rare blood cancer
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`http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm374761.htm
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`Exhibit 2001 Page 001
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`Pharmacyclics LLC - Ex. 2001
`Coalition for Affordable Drugs IV LLC v. Pharmacyclics LLC
`Case IPR2015-01076
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`patients. This program provides earlier patient access to promising new drugs while the company
`conducts confirmatory clinical trials. The FDA also granted Imbruvica priority review and orphan
`product designation because the drug demonstrated the potential to be a significant improvement
`in safety or effectiveness in the treatment of a serious condition and is intended to treat a rare
`disease, respectively.
`
`Imbruvica’s accelerated approval for MCL is based on a 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 or disappear after treatment (overall
`response rate). An improvement in survival or diseaserelated symptoms has not been established.
`
`The most common side effects reported in participants receiving Imbruvica are low levels of
`platelets in the blood (thrombocytopenia), diarrhea, a decrease in infectionfighting white blood
`cells (neutropenia), anemia, fatigue, musculoskeletal pain, swelling (edema), upper respiratory
`infection, nausea, bruising, shortness of breath (dyspnea), constipation, rash, abdominal pain,
`vomiting, and decreased appetite. Other clinically significant side effects include bleeding,
`infections, kidney problems and the development of other types of cancers.
`
`Imbruvica is comarketed by Sunnyvale, Calif.based Pharmacyclics and Raritan, N.J.based
`Janssen Biotech, Inc. Velcade (bortezomib) is marketed by Millennium Pharmaceuticals, based in
`Cambridge, Mass. Revlimid (lenalidomide) is marketed by Summit, N.J.based Celgene.
`
`For more information:
`
`FDA: Office of Hematology and Oncology Products
`(/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDER/ucm091745.htm)
`
`FDA: Breakthrough Therapies
`(/RegulatoryInformation/Legislation/FederalFoodDrugandCosmeticActFDCAct/SignificantA
`mendmentstotheFDCAct/FDASIA/ucm341027.htm)
`
`FDA: Drug Innovation
`(http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/default.htm)
`
`FDA: Approved Drugs: Questions and Answers
`(/Drugs/ResourcesForYou/Consumers/ucm054420.htm)
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`NCI: NonHodgkin Lymphoma (http://www.cancer.gov/cancertopics/types/nonhodgkin)
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`The FDA, an agency within the U.S. Department of Health and Human Services, protects the
`public health by assuring the safety, effectiveness, and security of human and veterinary drugs,
`
`Press Announcements > FDA approves Imbruvica for rare blood cancer
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