Healthcare Industry News: myelodysplastic syndromes
News Release - March 3, 2006
FDA Grants Priority Review for REVLIMID(R) sNDA for Treatment of Relapsed or Refractory Multiple MyelomaJune 30, 2006 PDUFA Date Established
Expanded Access Program to Remain Open Through FDA Action on REVLIMID sNDA
SUMMIT, N.J., March 3 (HSMN NewsFeed) - Celgene Corporation (Nasdaq: CELG ) announced that the U.S. Food and Drug Administration (FDA) has granted a Priority Review designation to its Supplemental New Drug Application (sNDA) for REVLIMID (lenalidomide) for the treatment of relapsed or refractory multiple myeloma. The Prescription Drug User Fee Act (PDUFA) date is June 30, 2006. The Company is seeking approval to market REVLIMID in combination with dexamethasone as a proposed indication for the treatment of multiple myeloma patients who have received at least one prior therapy subject to FDA review and approval. Priority Review is granted to a pharmaceutical product that, if approved, would be a significant improvement compared to existing marketed products or approved therapies in the treatment, diagnosis, or prevention of a disease.
The REVLIMID sNDA submission is based upon the safety and efficacy results of two large randomized pivotal Phase III special protocol assessment trials, North American Trial MM-009 and International Trial MM-010, evaluating REVLIMID plus dexamethasone in multiple myeloma patients that have received at least one prior therapy. Based on a pre-specified interim analysis, both studies achieved the primary endpoint of time to disease progression (TTP) with combination therapy of lenalidomide and dexamethasone over that of placebo and dexamethasone. The clinical data, both from MM-009 and MM-010, were presented during a plenary session at the December 2005 meeting of the American Society of Hematology (ASH).
The Celgene Expanded Access Program, available to qualified patients with relapsed or refractory multiple myeloma, will remain open to ensure broad access to REVLIMID while the REVLIMID sNDA is under review by the FDA.
REVLIMID® (lenalidomide) Capsules 5 mg & 10 mg
1. POTENTIAL FOR HUMAN BIRTH DEFECTS.
LENALIDOMIDE IS AN ANALOGUE OF THALIDOMIDE. THALIDOMIDE IS A KNOWN HUMAN TERATOGEN THAT CAUSES SEVERE LIFE-THREATENING HUMAN BIRTH DEFECTS. IF LENALIDOMIDE IS TAKEN DURING PREGNANCY, IT MAY CAUSE BIRTH DEFECTS OR DEATH TO AN UNBORN BABY. FEMALES SHOULD BE ADVISED TO AVOID PREGNANCY WHILE TAKING REVLIMID® (lenalidomide).
Special Prescribing Requirements
BECAUSE OF THIS POTENTIAL TOXICITY AND TO AVOID FETAL EXPOSURE TO REVLIMID® (lenalidomide), REVLIMID® (lenalidomide) IS ONLY AVAILABLE UNDER A SPECIAL RESTRICTED DISTRIBUTION PROGRAM. THIS PROGRAM IS CALLED "RevAssist(SM)." UNDER THIS PROGRAM, ONLY PRESCRIBERS AND PHARMACISTS REGISTERED WITH THE PROGRAM ARE ABLE TO PRESCRIBE AND DISPENSE THE PRODUCT. IN ADDITION, REVLIMID® (lenalidomide) MUST ONLY BE DISPENSED TO PATIENTS WHO ARE REGISTERED AND MEET ALL THE CONDITIONS OF THE RevAssist(SM) PROGRAM.
2. HEMATOLOGICAL TOXICITY
(NEUTROPENIA AND THROMBOCYTOPENIA)
REVLIMID® (lenalidomide) IS ASSOCIATED WITH SIGNIFICANT NEUTROPENIA AND THROMBOCYTOPENIA. PATIENTS SHOULD HAVE THEIR CBC CHECKED WEEKLY FOR THE FIRST 8 WEEKS OF REVLIMID® (lenalidomide) TREATMENT AND AT LEAST MONTHLY THEREAFTER TO MONITOR FOR CYTOPENIAS. MOST DELETION 5q MDS PATIENTS STUDIED REQUIRED A DOSE ADJUSTMENT FOR NEUTROPENIA AND/OR THROMBOCYTOPENIA.
3. DEEP VEIN THROMBOSIS AND PULMONARY EMBOLISM
REVLIMID® (lenalidomide) HAS DEMONSTRATED SIGNIFICANT RISK OF DEEP VEIN THROMBOSIS AND PULMONARY EMBOLISM IN SOME PATIENTS WITH CERTAIN MEDICAL CONDITIONS.
IMPORTANT SAFETY INFORMATION
Hypersensitivity: REVLIMID® (lenalidomide) is contraindicated in any patients who have demonstrated hypersensitivity to the drug or its components.
Other adverse events: Other most frequently reported adverse events were diarrhea, pruritis, rash, fatigue, constipation, nausea, nasopharyngitis, arthralgia, pyrexia, back pain, peripheral edema, cough, dizziness, headache, muscle cramp, dyspnea, and pharyngitis. REVLIMID® (lenalidomide) is substantially excreted by the kidney, so the risk of toxic reactions may be greater in patients with impaired renal function.
REVLIMID is a member of a proprietary group of novel immunomodulatory compounds, IMiDs®. Celgene continues to evaluate REVLIMID in a broad range of hematology and oncology conditions. The IMiD pipeline, including REVLIMID, is covered by a comprehensive intellectual property estate of U.S. and foreign issued and pending patent applications including composition-of-matter and use patents.
REVLIMID is approved by the FDA for treatment of patients with transfusion-dependent anemia due to low- or intermediate-1-risk myelodysplastic syndromes (MDS) associated with a deletion 5q cytogenetic abnormality with or without additional cytogenetic abnormalities. REVLIMID is not approved by the FDA or any other regulatory agencies as a treatment for any other indication and is currently being evaluated in clinical trials for efficacy and safety for future regulatory applications.
FOR FURTHER INFORMATION ABOUT REVLIMID® AND THE RevAssist(SM) PROGRAM, YOU MAY GO TO THE INTERNET AT www.REVLIMID.com OR BY CALLING THE MANUFACTURER'S TOLL FREE NUMBER 1-888-4CELGENE. RevAssist(SM) is a proprietary risk-management restrictive distribution program, tailored specifically for REVLIMID patients, to prevent the potential for human birth defects and ensure prompt and convenient access to REVLIMID.
About Multiple Myeloma
Multiple myeloma (also known as myeloma or plasma cell myeloma) is a cancer of the blood in which malignant plasma cells are overproduced in the bone marrow. Plasma cells are white blood cells that help produce antibodies called immunoglobulins that fight infection and disease. However, most patients with multiple myeloma have cells that produce a form of immuno-globulin called paraprotein (or M protein) that does not benefit the body. In addition, the malignant plasma cells replace normal plasma cells and other white blood cells important to the immune system. Multiple myeloma cells can also attach to other tissues of the body, such as bone, and produce tumors. The cause of the disease remains unknown. In the year 2005, there were approximately 200,000 people worldwide suffering from multiple myeloma. An estimated 74,000 new cases of multiple myeloma are expected in 2006. The estimated number of deaths from multiple myeloma expected in 2006 is approximately 60,000 worldwide.
About myelodysplastic syndromes
myelodysplastic syndromes (MDS) are a group of hematologic malignancies that affect approximately 300,000 people worldwide. myelodysplastic syndromes occur when blood cells remain in an immature or "blast" stage within the bone marrow and never develop into mature cells capable of performing their necessary functions. Eventually, the bone marrow may be filled with blast cells suppressing normal cell development. According to the American Cancer Society, 10,000 to 20,000 new cases of MDS are diagnosed each year in the United States, with mean survival rates ranging from approximately six months to six years for the different classifications of MDS. MDS patients must often rely on blood transfusions to manage symptoms of anemia and fatigue and may develop life-threatening iron overload and/or toxicity from frequent transfusions, thus underscoring the critical need for new therapies targeting the cause of the condition rather than simply managing its symptoms.
About Deletion 5q Chromosomal Abnormality
Chromosomal (cytogenetic) abnormalities are detected in more than half of patients with myelodysplastic syndrome (MDS), and involve a deletion in all or part of one or more specific chromosomes. The most common cytogenetic abnormalities in MDS are deletions in the long arm of chromosomes 5, 7, and 20. Another common abnormality is an extra copy of chromosome 8. A deletion involving the 5q chromosome may be involved in 20 percent to 30 percent of all MDS patients. The World Health Organization has also recently identified a unique subset of MDS patients with a "5q- Syndrome" where the only chromosomal abnormality is a specific portion of the 5q chromosome.
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. For more information, please visit the Company's website at http://www.celgene.com.
REVLIMID® is a registered trademark of Celgene Corporation.
RevAssist(SM) is a service mark of Celgene Corporation.
This release contains forward-looking statements which are subject to known and unknown risks, delays, uncertainties and other factors not under the Company's control, which may cause actual results, performance or achievements of the Company to be materially different from the results, performance or other expectations expressed or implied by these forward-looking statements. These factors include results of current or pending research and development activities, actions by the FDA and other regulatory authorities, and other factors described in the Company's filings with the Securities and Exchange Commission such as our 10K, 10Q and 8K reports.
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