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Published: Tuesday, Jun 22, 2010
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Lenalidomide Receives Orphan Medicinal Product Designation from the European Commission for Chronic Lymphocytic Leukemia

Chronic lymphocytic leukemia (CLL) is a hematological malignancy that affects approximately 165,000 people in the European Union (EU), based on an estimated prevalence rate of 3.5 per 10,000.

Lenalidomide (Revlimid) has been granted orphan medicinal product designation by the European Commission (EC) for treatment of chronic lymphocytic leukemia (CLL) following the favorable opinion of the European Medicines Agency’s (EMEA) Committee for Orphan Medicinal Products (COMP). Orphan medicinal product designation is granted by the EC to promote development of drugs to treat rare diseases or conditions.

Lenalidomide is an immunomodulatory agent that stimulates T-cells, inhibits the production of tumor necrosis factor alpha and vascular endothelial growth factor, and impairs the interaction between bone marrow stroma and hematopoietic cells.

Orphan designation will give lenalidomide access to the Centralized Procedure for the application for marketing approval, reduce fees associated with applying for marketing approval and protocol assistance, and provide 10 years of market exclusivity once approved for treatment of CLL.

Alessandra Ferrajoli, MD, Assistant Professor of Medicine, University of Texas MD Anderson Cancer, Houston, Texas, recently reported preliminary results of a study showing that lenalidomide, delivered at low doses in a continuous schedule, induces complete and partial responses in patients with refractory CLL. Dr. Ferrajoli stated, “Lenalidomide shows impressive results in helping these treatmentrefractory patients achieve complete and partial remission—and the effect looks durable.”

The investigators have enrolled 45 subjects in the ongoing study. Each patient receives lenalidomide 10mg/day for the first four weeks. The dosing is then escalated by 5 mg every 28 days to a maximum of 25 mg/day. Data on the first 35 subjects (median follow-up, 9 mo) were analyzed for the interim report. At the time of this analysis, the other 10 subjects had been on treatment for less than three months. Median subject age at enrollment was 64 years (range 49–86 yr), and the median number of prior treatments was four (range 1–15).

The investigators evaluated treatment responses according to the National Cancer Institute— Working Group criteria. They reported that 13 subjects (38%) have shown a response, with three achieving complete remission (CR, 9%), one achieving nodule response—partial remission (nPR, 3%), and nine achieving partial remission (PR, 26%). Median response duration was 11+ months. Eight patients with stable disease continue to be treated. Twelve patients failed, with two dying early of complications (on days 11 and 22).

Using flow cytometry, the researchers assessed complete responders for minimal residual disease (MRD). No MRD was found in two of the three subjects. The most common toxicity was myelosuppression, with Grade >3 neutropenia and thrombocytopenia found in 17% and 14% of the subjects, respectively.

“Remarkably, our patients did not develop lymphopenia and the T-cell count remained stable or increased during treatment,” the authors noted.

The researchers reported Grade >3 fatigue, diarrhea, and tumor flare in 9%, 6%, and 6% of the patients, respectively. There were 12 serious infections, eight pneumonias, two fevers of unknown origin, one case of fatal mucormycosis, and one case of enteric cryptosporidiosis. None of the patients received routine antibiotic prophylaxis.

“The decision by the EC to designate Revlimid as an orphan medicinal product for treatment of CLL supports our efforts to move Revlimid as quickly as possible through the clinical and regulatory development process worldwide,” said Graham Burton MD, SVP, Global Regulatory Affairs and Pharmacovigilance for Celgene Corporation. “We continue to be encouraged by the growing body of published and presented data on Revlimid by key opinion leaders at major medical meetings, and based on these findings, we are committed to accelerating wherever possible our efforts to help address the unmet medical needs of patients with CLLworldwide.”

n the EU, Iceland, and Norway, Revlimid is authorized for marketing and, in combination with dexamethasone, is indicated for the treatment of multiple myeloma in patients who have received at least one prior therapy. Revlimid is also currently approved in the United States by the U.S. Food and Drug Administration and in Switzerland for multiple myeloma in patients who have received at least one prior therapy. In addition, Revlimid is approved in the United States for treatment of transfusion-dependent anemia because of low- or intermediate-1-risk myelodysplastic syndrome associated with a deletion 5q cytogenetic abnormality with or without additional cytogenetic abnormalities.

Revlimid has already obtained orphan drug designation in the EU, United States, Switzerland, and Australia for the treatment of multiple myeloma and in the EU, Australia, and United States for treatment of myelodysplastic syndrome.

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