DRUG APPROVALS BY DR ANTHONY MELVIN CRASTO
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Pomalyst (pomalidomide) Capsules
Company: Celgene Corporation
Date of Approval: February 8, 2013
Treatment for: Multiple Myeloma
Pomalyst (pomalidomide) is a thalidomide analogue indicated for the treatment of patients with multiple myeloma.
The U.S. Food and Drug Administration today approved Pomalyst (pomalidomide) to treat patients with multiple myeloma whose disease progressed after being treated with other cancer drugs.
Multiple myeloma is a form of blood cancer that primarily affects older adults and arises from plasma cells in the bone marrow. According to the National Cancer Institute, approximately 21,700 Americans are diagnosed with multiple myeloma and 10,710 die yearly from the disease.
Pomalyst is a pill that modulates the body’s immune system to destroy cancerous cells and inhibit their growth. It is intended for patients who have received at least two prior therapies, including lenalidomide and bortezomib, and whose disease did not respond to treatment and progressed within 60 days of the last treatment (relapsed and refractory).
“Pomalyst is the third drug in a class of immunomodulatory agents that includes lenalidomide and thalidomide, and is the second drug approved in the past year to treat multiple myeloma,” said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research. “Treatment for multiple myeloma is tailored to meet individual patient’s needs, and today’s approval provides an additional treatment option for patients who have not responded to other drugs.”
- FDA Approves Pomalyst for Advanced Multiple Myeloma – February 8, 2013
- Celgene Corporation Provides Update on FDA Advisory Committee for Pomalidomide – October 3, 2012
- The International Myeloma Foundation Says Pomalidomide, an Important New Drug for Patients, Has Been Submitted for FDA Approval – April 27, 2012
pomalidomide. 4-Amino-2-(2,6-dioxopiperidin-3-yl)isoindole-1,3-dione
Pomalidomide (INN, originally CC-4047 or 3-amino-thalidomide, marketed as Pomalyst by Celgene), is a derivative of thalidomide that is anti-angiogenic and also acts as an immunomodulator. Pomalidomide was approved on February 8, 2013 by the U.S. Food and Drug Administration (FDA) as a treatment for relapsed and refractory multiple myeloma.[1] An application for approval to treat multiple myeloma also has been submitted by Celgene to the European Medicines Agency, and a decision on that application is expected by the second half of 2013.[1]
Origin and development
The parent compound of pomalidomide, thalidomide, was originally discovered to inhibit angiogenesis in 1994.[2] Based upon this discovery, thalidomide was taken into clinical trials for cancer, leading to its ultimate FDA approval for multiple myeloma. Further structure activity studies done in Dr. Robert D’Amato’s lab at Boston Children’s Hospital led to the first report in 2001[3] that 3-amino-thalidomide was able to directly inhibit both the tumor cell and vascular compartments of myeloma cancers. This dual activity of pomalidomide makes it more efficacious than thalidomide in vitro and in vivo.[4]
Clinical trials
Phase I trial results showed tolerable side effects.[5]
Phase II clinical trials for multiple myeloma and myelofibrosis reported ‘promising results’.[6][7]
Phase III results were reported at ASH in 2012 and showed significant extension of progression-free survival (median 3.6 months vs. 1.8 months; P < 0.001), and overall survival in patients taking pomalidomide and dexamethasone.[8]
- “Pomalyst (Pomalidomide) Approved By FDA For Relapsed And Refractory Multiple Myeloma”. The Myeloma Beacon. Retrieved 2013-02-08.
- D’Amato, Robert J.; Loughnan, Michael S.; Flynn, Evelyn; Folkman, Judah (1994). “Thalidomide is an inhibitor of angiogenesis”. Proceedings of the National Academy of Sciences of the United States of America 91 (9): 4082–5. Bibcode 1994PNAS…91.4082D. doi:10.1073/pnas.91.9.4082. JSTOR 2364596. PMC 43727. PMID 7513432.
- D’Amato, R; Lentzsch, S; Anderson, KC; Rogers, MS (2001). “Mechanism of action of thalidomide and 3-aminothalidomide in multiple myeloma”. Seminars in Oncology 28 (6): 597–601. doi:10.1016/S0093-7754(01)90031-4. PMID 11740816.
- Lentzsch, S; Rogers, MS; Leblanc, R; Birsner, AE; Shah, JH; Treston, AM; Anderson, KC; D’Amato, RJ (2002). “S-3-Amino-phthalimido-glutarimide inhibits angiogenesis and growth of B-cell neoplasias in mice”. Cancer research 62 (8): 2300–5. PMID 11956087.
- Streetly, Matthew J.; Gyertson, Kylie; Daniel, Yvonne; Zeldis, Jerome B.; Kazmi, Majid; Schey, Stephen A. (2008). “Alternate day pomalidomide retains anti-myeloma effect with reduced adverse events and evidence of in vivo immunomodulation”. British Journal of Haematology 141 (1): 41–51. doi:10.1111/j.1365-2141.2008.07013.x. PMID 18324965.
- “Promising Results From 2 Trials Highlighting Pomalidomide Presented At ASH” (Press release). Celgene. December 11, 2008. Retrieved October 28, 2012.
- Tefferi, Ayalew (December 8, 2008). “Pomalidomide Therapy in Anemic Patients with Myelofibrosis: Results from a Phase-2 Randomized Multicenter Study”. 50th ASH Annual Meeting and Exposition. San Francisco. Retrieved October 28, 2012.
- “Phase III Study (MM-003) of Pomalidomide Plus Low-Dose Dexamethasone Demonstrates Significant Progression-Free and Overall Survival Improvement for Patients with Relapsed or Refractory Multiple Myeloma.”. 11 Dec 2012.
- This new drug is specifically indicated for patients who have received at least 2 prior therapies, including lenalidomide (Revlimid, Celgene) and bortezomib (Velcade, Millennium Pharmaceuticals), and whose disease did not respond to treatment and progressed within 60 days of the last treatment.
New Drug Approvals 