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THOUSAND OAKS, Calif. and SOUTH SAN FRANCISCO, Calif. (Dec. 3, 2014) - Amgen (NASDAQ:AMGN) today announced that the U.S. Food and Drug Administration (FDA) has granted approval of BLINCYTO™ (blinatumomab) for the treatment of patients with Philadelphia chromosome-negative (Ph-) relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). This indication is approved under accelerated approval. Continued approval for this indication may be contingent upon verification of clinical benefit in subsequent trials. With this approval, BLINCYTO becomes the first FDA-approved bispecific CD19-directed CD3 T-cell engager (BiTE®) antibody construct product, and the first single-agent immunotherapy to be approved for the treatment of patients with Ph- relapsed or refractory B-cell precursor ALL, a rare and rapidly progressing cancer of the blood and bone marrow.1-3
“The FDA’s breakthrough therapy designation and accelerated approval of BLINCYTO underscores the critical need for new treatment options for patients with relapsed or refractory B-cell precursor ALL, who are often young adults,” said Sean E. Harper, M.D., executive vice president of Research and Development at Amgen. “BLINCYTO is the first clinical and regulatory validation of the BiTE® platform, a new and innovative approach that helps the body’s own immune system fight cancer.”
The BLINCYTO approval is based on results of Amgen’s ‘211 trial, a Phase 2, multicenter, single-arm open-label study. Eligible patients were ≥ 18 years of age with Ph- relapsed or refractory B-cell precursor ALL. Relapsed or refractory was defined as relapsed with first remission duration of < 12="" months="" in="" the="" first="" salvage,="" or="" relapsed="" or="" refractory="" after="" first="" salvage="" therapy,="" or="" relapsed="" within="" 12="" months="" of="" allogeneic="" hematopoietic="" stem="" cell="" transplantation="" (hsct),="" and="" had="">= 10 percent blasts in bone marrow. Of the 185 patients evaluated in the trial, 41.6 percent (77/185; 95 percent CI: 34.4-49.1) achieved complete remission or complete remission with partial hematologic recovery (CR/CRh*) within two cycles of treatment with BLINCYTO, which was the primary endpoint of the study. The majority of responses (81 percent [62/77]) occurred within the first cycle of treatment. Among patients who achieved CR/CRh*, 39 percent (30/77) went on to HSCT, and 75.3 percent (58/77 95 percent CI: 64.2-84.4) achieved minimal residual disease (MRD) response, a measure of eradication of residual disease at the molecular level.
“The approval of BLINCYTO represents a significant milestone in immunotherapy research, providing clinicians the opportunity to offer a new single-agent therapy to patients fighting this highly aggressive cancer with previously limited options,” said Anthony S. Stein, M.D., clinical professor, Hematology/Oncology at City of Hope.
BLINCYTO has a BOXED WARNING in its product label regarding Cytokine Release Syndrome (CRS) and Neurological Toxicities.
Cytokine Release Syndrome (CRS), which may be life-threatening or fatal, occurred in patients receiving BLINCYTO. Interrupt or discontinune BLINCYTO as recommended. Neurological toxicities, which may be severe, life-threatening, or fatal, occurred in patients receiving BLINCYTO. Interrupt or discontinue BLINCYTO as recommended.
BLINCYTO is contraindicated to patients with known hypersensitivity to blinatumomab or to any component of the product formulation.
Monitor patients for signs and symptoms of infection and treat appropriately.
Advise patients to refrain from driving and engaging in hazardous occupations or activities such as driving, operating heavy or potentially dangerous machinery while BLINCYTO is being administered.
It is important to strictly follow instructions for preparation (including admixing) and administration to prevent overdose and underdose.
The most common adverse reactions (≥ 20 percent) were pyrexia (62 percent), headache (36 percent), peripheral edema (25 percent), febrile neutropenia (25 percent), nausea (25 percent), hypokalaemia (23 percent), rash (21 percent), tremor (20 percent) and constipation (20 percent). Serious adverse reactions were reported in 65 percent of patients. The most common serious adverse reactions (≥ 2 percent) included febrile neutropenia, pyrexia, pneumonia, sepsis, neutropenia, device-related infection, tremor, encephalopathy, infection, overdose, confusion, Staphylococcal bacteremia and headache.
The FDA has also approved a risk evaluation and mitigation strategy (REMS) for BLINCYTO. The purpose of the BLINCYTO REMS is to inform healthcare providers of the serious risks of CRS, neurological toxicities, and preparation and administration errors. Additional information about the BLINCYTO REMS program can be found at http://www.BLINCYTOREMS.com.
Please contact Amgen Medinfo at 800-77-AMGEN (800-772-6436) regarding BLINCYTO availability.
Amgen and its subsidiary Onyx Pharmaceuticals, Inc., which will commercialize BLINCYTO in the U.S., have announced the availability of Onyx Pharmaceuticals 360™ (Onyx 360), to patients receiving BLINCYTO in the U.S. Onyx 360 is a comprehensive patient and caregiver support and services program designed to help patients navigate the treatment journey, including reimbursement and payment support, treatment support and referrals to third-party organizations for day-to-day needs and emotional support. Dedicated Oncology Nurse Advocates are available Monday through Friday from 9 a.m. to 8 p.m. Eastern Standard Time at 1-855-ONYX-360 (1-855-669-9360) to assist patients, caregivers and healthcare providers.
Patients diagnosed with adult ALL are often young adults, with a median age at diagnosis of 34-39.4 In adult patients with relapsed or refractory ALL, median overall survival is just three to five months.5
BLINCYTO is the first BiTE® antibody construct and the first single-agent immunotherapy to be approved by the U.S. Food and Drug Administration (FDA).3 BLINCYTO was granted breakthrough therapy and priority review designations by the FDA, and is now approved in the U.S. for the treatment of Philadelphia chromosome-negative (Ph-) relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL).
Bispecific T cell engager (BiTE®) antibody constructs are a type of immunotherapy being investigated for fighting cancer by helping the body’s immune system to detect and target malignant cells. The modified antibodies are designed to engage two different targets simultaneously, thereby juxtaposing T cells (a type of white blood cell capable of killing other cells perceived as threats) to cancer cells. BiTE® antibody constructs help place the T cells within reach of the targeted cell, with the intent of allowing T cells to inject toxins and trigger the cancer cell to die (apoptosis). BiTE® antibody constructs are currently being investigated for their potential to treat a wide variety of cancers. For more information, visit www.biteantibodies.com.
BLINCYTO is indicated for the treatment of Philadelphia chromosome-negative relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL).
This indication is approved under accelerated approval. Continued approval for this indication may be contingent upon verification of clinical benefit in subsequent trials.
WARNING: CYTOKINE RELEASE SYNDROME and NEUROLOGICAL TOXICITIES
Cytokine Release Syndrome (CRS), which may be life-threatening or fatal, occurred in patients receiving BLINCYTO™. Interrupt or discontinue BLINCYTO™ as recommended.
Neurological toxicities, which may be severe, life-threatening or fatal, occurred in patients receiving BLINCYTO™. Interrupt or discontinue BLINCYTO™ as recommended.
BLINCYTO™ is contraindicated in patients with a known hypersensitivity to blinatumomab or to any component of the product formulation.
Please see full Prescribing Information and medication guide for BLINCYTO at www.BLINCYTO.com.
Amgen is committed to unlocking the potential of biology for patients suffering from serious illnesses by discovering, developing, manufacturing and delivering innovative human therapeutics. This approach begins by using tools like advanced human genetics to unravel the complexities of disease and understand the fundamentals of human biology.
Amgen focuses on areas of high unmet medical need and leverages its biologics manufacturing expertise to strive for solutions that improve health outcomes and dramatically improve people’s lives. A biotechnology pioneer since 1980, Amgen has grown to be the world’s largest independent biotechnology company, has reached millions of patients around the world and is developing a pipeline of medicines with breakaway potential.
For more information, visit www.amgen.com and follow us on www.twitter.com/amgen.
Based in South San Francisco, California, Onyx Pharmaceuticals, Inc., an Amgen subsidiary, is a global biopharmaceutical company engaged in the development and commercialization of innovative therapies for improving the lives of people with cancer. The company is focused on developing novel medicines that target key molecular pathways. For more information about Onyx, visit the company's website at www.onyx.com. Onyx Pharmaceuticals is on Twitter. Sign up to follow our Twitter feed @OnyxPharm at http://twitter.com/OnyxPharm.
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Kristen Davis, 805-447-3008 (Media)
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