Saturday, May 25, 2013

Celgene Announces U.S. FDA Grants Priority Review for ABRAXANE® sNDA in Advanced Pancreatic Cancer

ME Newswire / Business Wire

BOUDRY, Switzerland - Saturday, May 25th 2013

European Medicines Agency Accepts Regulatory Submission of Type II Variation for ABRAXANE for the Treatment of Advanced Pancreatic Cancer

Celgene International Sàrl, a subsidiary of Celgene Corporation (Celgene) (NASDAQ: CELG) today announced that the U.S. Food and Drug Administration (FDA) has assigned a Priority Review designation to the supplemental New Drug Application (sNDA) for the use of ABRAXANE® (paclitaxel protein-bound particles for injectable suspension) (albumin-bound) in combination with gemcitabine for the first–line treatment of patients with advanced pancreatic cancer.

The FDA grants Priority Review to medicines that, if approved, have the potential to offer significant improvement compared to marketed products or provide a treatment where no adequate therapy exists. The goal for completing a Priority Review is six months. The Prescription Drug User Fee Act (PDUFA) date for the sNDA for ABRAXANE is set for September 21, 2013.

In April 2013, the European Medicines Agency (EMA) has also accepted for review a Type II Variation to the current Marketing Authorization Application (MAA) for ABRAXANE, in combination with gemcitabine, for the first–line treatment of patients with advanced pancreatic cancer. Celgene plans to submit dossiers for registration in other countries/regions during 2013.

Both applications included data from an open-label, phase III, randomized, international study, Metastatic Pancreatic Adenocarcinoma Clinical Trial (MPACT) involving 861 patients with metastatic pancreatic cancer. Results from this study were recently presented at the American Society of Clinical Oncology’s (ASCO) 2013 Gastrointestinal Cancers Symposium in January.

Celgene is preparing a development plan for a phase III, international, multicenter, randomized controlled trial evaluating the activity of ABRAXANE plus gemcitabine in the adjuvant pancreatic cancer setting.

ABRAXANE is not currently approved for the treatment of advanced pancreatic cancer.

About Pancreatic Cancer

Pancreatic cancer is the eighth leading cause of cancer-related death worldwide and the fourth leading cause of cancer-related death in the US. The pancreas is composed of two main cell types: exocrine and endocrine. Exocrine tumors are by far the most common type of pancreatic cancer, with adenocarcinoma accounting for about 95 percent of cancers of the pancreas. For all stages of pancreatic cancer combined, the 5-year overall survival rate is about 6 percent, which is the lowest 5-year overall survival rate of any cancer in the US. In Europe, the reported survival rate is less than 10 percent survival at five years.

About ABRAXANE®

ABRAXANE is an albumin-bound form of paclitaxel that is manufactured using patented nab® technology. ABRAXANE is formulated with albumin, a human protein, and is free of solvents.

In the United States, ABRAXANE was first approved in January 2005 for the treatment of breast cancer after failure of combination chemotherapy for metastatic disease or relapse within 6 months of adjuvant chemotherapy. Prior therapy should have included an anthracycline unless clinically contraindicated. ABRAXANE is also approved in Canada, India, European Union/European Economic Area (EU/EEA), South Korea, China, Australia, Bhutan, United Arab Emirates, Nepal, New Zealand, Japan, Russia, Sri Lanka, and Argentina for the treatment of metastatic breast cancer.

In October 2012, ABRAXANE was approved by the FDA for the first-line treatment of locally advanced or metastatic non-small cell lung cancer, in combination with carboplatin, in patients who are not candidates for curative surgery or radiation therapy. ABRAXANE is also approved in Japan and Argentina for the treatment of non-small cell lung cancer and is approved in Japan for the treatment of gastric cancer.

ABRAXANE is currently in various stages of investigation for the potential treatment of the following cancers: melanoma, bladder, ovarian, and expanded applications for breast, lung, and pancreatic cancer.

U.S. Regulatory Information for ABRAXANE

ABRAXANE® for Injectable Suspension (paclitaxel protein-bound particles for injectable suspension)(albumin-bound) is indicated for the treatment of breast cancer after failure of combination chemotherapy for metastatic disease or relapse within 6 months of adjuvant chemotherapy. Prior therapy should have included an anthracycline unless clinically contraindicated.

ABRAXANE is indicated for the first-line treatment of locally advanced or metastatic non-small cell lung cancer, in combination with carboplatin, in patients who are not candidates for curative surgery or radiation therapy.

Important Safety Information

WARNING - NEUTROPENIA

    Do not administer ABRAXANE therapy to patients who have baseline neutrophil counts of less than 1,500 cells/mm3. In order to monitor the occurrence of bone marrow suppression, primarily neutropenia, which may be severe and result in infection, it is recommended that frequent peripheral blood cell counts be performed on all patients receiving ABRAXANE
    Note: An albumin form of paclitaxel may substantially affect a drug’s functional properties relative to those of drug in solution. DO NOT SUBSTITUTE FOR OR WITH OTHER PACLITAXEL FORMULATIONS

CONTRAINDICATIONS

Neutrophil Counts

    ABRAXANE should not be used in patients who have baseline neutrophil counts of < 1,500 cells/mm3

Hypersensitivity

    Patients who experience a severe hypersensitivity reaction to ABRAXANE should not be rechallenged with the drug

WARNINGS AND PRECAUTIONS

Hematologic Effects

    Bone marrow suppression (primarily neutropenia) is dose-dependent and a dose-limiting toxicity of ABRAXANE. In clinical studies, Grade 3-4 neutropenia occurred in 34% of patients with metastatic breast cancer (MBC) and 47% of patients with non-small cell lung cancer (NSCLC)
    Monitor for myelotoxicity by performing complete blood cell counts frequently, including prior to dosing on Day 1 for MBC and Days 1, 8, and 15 for NSCLC
    Do not administer ABRAXANE to patients with baseline absolute neutrophil counts (ANC) of less than 1,500 cells/mm3
    In the case of severe neutropenia (<500 cells/mm3 for 7 days or more) during a course of ABRAXANE therapy, reduce the dose of ABRAXANE in subsequent courses in patients with either MBC or NSCLC
    In patients with MBC, resume treatment with every-3-week cycles of ABRAXANE after ANC recovers to a level >1,500 cells/mm3 and platelets recover to >100,000 cells/mm3
    In patients with NSCLC, resume treatment if recommended at permanently reduced doses for both weekly ABRAXANE and every-3-week carboplatin after ANC recovers to at least 1,500 cells/mm3 and platelet count of at least 100,000 cells/mm3 on Day 1 or to an ANC of at least 500 cells/mm3 and platelet count of at least 50,000 cells/mm3 on Days 8 or 15 of the cycle

Nervous System

    Sensory neuropathy is dose- and schedule-dependent
    The occurrence of Grade 1 or 2 sensory neuropathy does not generally require dose modification
    If ≥ Grade 3 sensory neuropathy develops, treatment should be withheld until resolution to Grade 1 or 2 for MBC or until resolution to ≤ Grade1 for NSCLC followed by a dose reduction for all subsequent courses of ABRAXANE

Hypersensitivity

    Severe and sometimes fatal hypersensitivity reactions, including anaphylactic reactions, have been reported
    Patients who experience a severe hypersensitivity reaction to ABRAXANE should not be re-challenged with this drug

Hepatic Impairment

    Because the exposure and toxicity of paclitaxel can be increased with hepatic impairment, administration of ABRAXANE in patients with hepatic impairment should be performed with caution
    The starting dose should be reduced for patients with moderate or severe hepatic impairment

Albumin (Human)

    ABRAXANE contains albumin (human), a derivative of human blood

Use in Pregnancy: Pregnancy Category D

    ABRAXANE can cause fetal harm when administered to a pregnant woman
    If this drug is used during pregnancy, or if the patient becomes pregnant while receiving this drug, the patient should be apprised of the potential hazard to the fetus
    Women of childbearing potential should be advised to avoid becoming pregnant while receiving ABRAXANE

Use in Men

    Men should be advised not to father a child while receiving ABRAXANE

ADVERSE REACTIONS

Randomized Metastatic Breast Cancer (MBC) Study

    The most common adverse reactions (≥20%) with single-agent use of ABRAXANE vs. Paclitaxel injection in the MBC study were alopecia (90%, 94%), neutropenia (all cases 80%,82%; severe 9%,22%), sensory neuropathy (any symptoms 71%, 56%; severe 10%, 2%), abnormal ECG (all patients 60%, 52%; patients with normal baseline 35%, 30%), fatigue/asthenia (any 47%, 39%; severe 8%, 3%), myalgia/arthralgia (any 44%, 49%; severe 8%, 4%), AST elevation (any 39%, 32%), alkaline phosphatase elevation (any 36%, 31%), anemia (all cases 33%, 25%; severe 1%, <1%), nausea (any 30%, 22%; severe 3%, <1%), diarrhea (any 27%, 15%; severe <1%, 1%) and infections (24%, 20%), respectively
    Sensory neuropathy was the cause of ABRAXANE discontinuation in 7/229 (3%) patients
    Other adverse reactions of note with the use of ABRAXANE vs. Paclitaxel injection included vomiting (any 18%,10%; severe 4%, 1%), fluid retention (any 10%,8%; severe 0%,<1%); mucositis (any 7%, 6%; severe <1%, 0%), hepatic dysfunction (elevations in bilirubin 7%, 7%), hypersensitivity reactions (any 4%,12%; severe 0%, 2%), thrombocytopenia (any 2%, 3%; severe <1%, <1%), and injection site reactions (<1%, 1%), respectively. Dehydration and pyrexia were also reported
    Renal dysfunction (any 11%, severe 1%) were reported in patients treated with ABRAXANE (n = 229)
    In all ABRAXANE treated patients (n=366) ocular/visual disturbances were reported (any 13%; severe 1%)
    Severe cardiovascular events possibly related to single-agent ABRAXANE occurred in approximately 3% of patients and included cardiac ischemia/infarction, chest pain, cardiac arrest, supraventricular tachycardia, edema, thrombosis, pulmonary thromboembolism, pulmonary emboli, and hypertension
    Cases of cerebrovascular attacks (strokes) and transient ischemic attacks have been reported

Non-Small Cell Lung (NSCLC) Cancer Study

    Adverse reactions with a difference of ≥2%, Grade 3 or higher, with combination use of ABRAXANE and carboplatin vs. combination use of Paclitaxel injection and carboplatin in NSCLC were anemia (28%, 7%); neutropenia (47%, 58%); thrombocytopenia (18%, 9%), peripheral neuropathy (3%, 12%), and peripheral edema (0%, <1%), respectively
    Adverse reactions with a difference of ≥5%, Grades 1-4, with combination use of ABRAXANE and carboplatin vs. combination use of Paclitaxel injection and carboplatin in NSCLC were anemia (98%,91%), neutropenia (85%, 83%), thrombocytopenia (68%, 55%), peripheral neuropathy (48%, 64%), peripheral edema (10%, 4%), and epistaxis (7%, 2%), respectively
    The most common adverse reactions (≥20%) of ABRAXANE in combination with carboplatin for NSCLC were anemia, neutropenia, thrombocytopenia, alopecia, peripheral neuropathy, nausea, and fatigue
    The most common serious adverse reactions of ABRAXANE in combination with carboplatin for NSCLC were anemia (4%) and pneumonia (3%)
    The most common adverse reactions resulting in permanent discontinuation of ABRAXANE were neutropenia (3%), thrombocytopenia (3%), and peripheral neuropathy (1%)
    The most common adverse reactions resulting in dose reduction of ABRAXANE were neutropenia (24%), thrombocytopenia (13%), and anemia (6%)
    The most common adverse reactions leading to withholding or delay in ABRAXANE dosing were neutropenia (41%), thrombocytopenia (30%), and anemia (16%)
    The following common (≥10% incidence) adverse reactions were observed at a similar incidence in ABRAXANE plus carboplatin-treated and paclitaxel injection plus carboplatin-treated patients: alopecia 56%, nausea 27%, fatigue 25%, decreased appetite 17%, asthenia 16%, constipation 16%, diarrhea 15%, vomiting 12%, dyspnea 12%, and rash 10% (incidence rates are for the ABRAXANE plus carboplatin treatment group)

Post-marketing Experience with ABRAXANE and Other Paclitaxel Formulations

    Severe and sometimes fatal hypersensitivity reactions have been reported with ABRAXANE. The use of ABRAXANE in patients previously exhibiting hypersensitivity to paclitaxel injection or to human albumin has not been studied
    There have been reports of congestive heart failure and left ventricular dysfunction with ABRAXANE, primarily among individuals with underlying cardiac history or prior exposure to cardiotoxic drugs
    There have been reports of extravasation of ABRAXANE. Given the possibility of extravasation, it is advisable to monitor closely the ABRAXANE infusion site for possible infiltration during drug administration

DRUG INTERACTIONS

    Caution should be exercised when administering ABRAXANE concomitantly with medicines known to inhibit or induce either CYP2C8 or CYP3A4

USE IN SPECIFIC POPULATIONS

Nursing Mothers

    It is not known whether paclitaxel is excreted in human milk. Because many drugs are excreted in human milk and because of the potential for serious adverse reactions in nursing infants, a decision should be made to discontinue nursing or to discontinue the drug, taking into account the importance of the drug to the mother

Pediatric

    The safety and effectiveness of ABRAXANE in pediatric patients have not been evaluated

Geriatric

    No toxicities occurred notably more frequently among patients ≥65 years of age who received ABRAXANE for MBC
    Myelosuppression, peripheral neuropathy, and arthralgia were more frequent in patients ≥65 years of age treated with ABRAXANE and carboplatin in NSCLC

Renal Impairment

    The use of ABRAXANE has not been studied in patients with renal impairment

DOSAGE AND ADMINISTRATION

    Dose adjustment is recommended for patients with moderate and severe hepatic impairment and patients who experience severe neutropenia or severe sensory neuropathy during treatment with ABRAXANE
    Withhold ABRAXANE if AST >10 x ULN or bilirubin >5 x ULN
    Dose reductions or discontinuation may be needed based on severe hematologic or neurologic toxicities
    Monitor patients closely

Please see full Prescribing Information, including Boxed WARNING, CONTRAINDICATIONS, WARNINGS AND PRECAUTIONS, and ADVERSE REACTIONS, please visit

http://www.abraxane.com/docs/Abraxane_PrescribingInformation.pdf

About Celgene

Celgene Corporation, headquartered in Summit, New Jersey, is an integrated global pharmaceutical company engaged primarily in the discovery, development and commercialization of innovative therapies for the treatment of cancer and inflammatory diseases through gene and protein regulation.

Celgene International Sárl, located in Boudry, Switzerland, is a wholly owned subsidiary and international headquarters of Celgene Corporation. For more information, please visit the Company's website at www.celgene.com.

Forward-Looking Statements

This press release contains forward-looking statements, which are generally statements that are not historical facts. Forward-looking statements can be identified by the words "expects," "anticipates," "believes," "intends," "estimates," "plans," "will," “outlook” and similar expressions. Forward-looking statements are based on management’s current plans, estimates, assumptions and projections, and speak only as of the date they are made. We undertake no obligation to update any forward-looking statement in light of new information or future events, except as otherwise required by law. Forward-looking statements involve inherent risks and uncertainties, most of which are difficult to predict and are generally beyond our control. Actual results or outcomes may differ materially from those implied by the forward-looking statements as a result of the impact of a number of factors, many of which are discussed in more detail in our Annual Report on Form 10-K and our other reports filed with the Securities and Exchange Commission.

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