CANADA approves POMALYST one year after USA

Pomalyst (Pomalidomide) Approved By FDA For Relapsed And Refractory Multiple Myeloma The Myeloma Beacon Staff Published: Feb 8, 2013 The United States Food and Drug Administration (FDA) has approved pomalidomide, which will be marketed under the brand name Pomalyst, for the treatment of relapsed and refractory multiple myeloma. Specifically, Pomalyst has been approved for use in patients with multiple myeloma who have received at least two prior therapies including Rev­limid (lenalido­mide) and Velcade (bortezomib) and have demon­strat­ed disease progression on or within 60 days of completion of the last therapy. Celgene (NASDAQ:CELG), the company that will market Pomalyst, has told The Beacon that the drug will be available in U.S. pharmacies in about two weeks. Pomalyst is the second drug, following Kyprolis (carfilzomib), that the FDA has approved for the treatment of multiple myeloma over the past year. Prior to Kyprolis, the last FDA approval of a novel myeloma therapy was in June 2006, when the agency approved Revlimid as a treatment for myeloma. Pomalyst also is being reviewed by the European Medicines Agency for approval in Europe. Celgene submitted the European marketing authorization application for the drug in June of last year. In recent discussions with financial analysts, the company has said that it expects a decision on this application by the second half of this year. Pomalyst is chemically similar to Revlimid and thalidomide. All three drugs are administered orally as a capsule or tablet, and the three drugs together make up the class of myeloma therapies known as immunomodulatory agents. Celgene, which developed Pomalyst and will sell it in the U.S., also markets Revlimid and thalidomide in the U.S. and internationally. The FDA’s decision regarding Pomalyst’s new drug application is likely to be its last approval of a new myeloma therapy for at least a year. There are a number of potential new myeloma therapies in the later stages of development, including elotuzumab, panobinostat, and perifosine. However, many analysts believe it may not be until 2015, and possibly even later, until one of these drugs becomes the next novel anti-myeloma therapy to gain FDA approval. Celgene has not yet said publicly what the price of Pomalyst will be. However, it has confirmed with The Beacon published reports that Pomalyst will cost about $10,500 per 28-day cycle, based on a 21-out-of-28-day dosing regimen. At that price, Pomalyst will be the most expensive treatment for multiple myeloma. In comparison, Revlimid costs $8,400 per 28-day period at the FDA-approved 21-out-of-28-day dosing; Velcade costs between $4,100 and $8,200 per 28-day period, depending on the frequency of dosing; and Kyprolis costs $10,000 per 28-day cycle at the recommended dose for a patient of average size. Pomalyst’s FDA approval is based on data from the Phase 2 “MM-002” clinical trial of Pomalyst plus dexamethasone in relapsed and refractory myeloma patients. Updated results from this study were presented at the American Society of Hematology (ASH) meeting in December (see related Beacon news). They showed that the combination of Pomalyst and dexamethasone had an overall response rate of 34 percent in myeloma patients with a median of five previous lines of therapy. Median progression-free survival in these patients was 4.6 months and median overall survival was 16.5 months. Also presented at the ASH meeting were initial results of the Phase 3 “MM-003” clinical trial comparing Pomalyst plus low-dose dexamethasone to only high-dose dexamethasone, also in heavily pretreated myeloma patients. These results showed that the combination of Pomalyst and dexamethasone had a statistically significant overall survival advantage compared to treatment with with dexamethasone alone (see related Beacon news). In Pomalyst’s clinical trials, the most frequent serious side effects were low red blood cell, white blood cell, and platelet counts, as well as infections such as pneumonia.