title: Pomalidomide: first global approval.
abstract: Pomalidomide (Pomalyst(®)) is a small molecule analogue of thalidomide under development with Celgene Corporation for the oral treatment of haematological and connective tissue diseases. Pomalidomide has been approved in the USA and is awaiting approval in the EU for use with low-dose dexamethasone for the treatment of relapsed and refractory multiple myeloma that has progressed following at least two prior therapies, including lenalidomide and bortezomib. The efficacy and safety of pomalidomide as monotherapy in patients with relapsed and refractory multiple myeloma has also been evaluated in a phase III trial. The agent is in phase III clinical development for the treatment of myelofibrosis and in phase II development for systemic sclerosis. Pomalidomide is also being investigated in patients with amyloidosis, prostate cancer, small cell lung cancer, pancreatic cancer, graft-versus-host disease, and Waldenstrom's macroglobulinaemia. This article summarizes the milestones in the development of pomalidomide leading to this first global approval for relapsed and refractory multiple myeloma.
authors.affiliation: Adis R & D Insight, 41 Centorian Drive, Private Bag 65901, Mairangi Bay, North Shore, 0754 Auckland, New Zealand. dru@adis.com
authors.forename: Shelley
authors.initials: S
authors.lastname: Elkinson
authors.forename: Paul L
authors.initials: PL
authors.lastname: McCormack
mesh_terms: D020533:Angiogenesis Inhibitors / Q000737:chemistry* / Q000493:pharmacokinetics / Q000627:therapeutic use*; D000818:Animals; D002986:Clinical Trials as Topic / Q000379:methods; D017277:Drug Approval / Q000379:methods*; D006086:Graft vs Host Disease / Q000188:drug therapy / Q000378:metabolism; D006801:Humans; D009101:Multiple Myeloma / Q000188:drug therapy / Q000378:metabolism; D013792:Thalidomide / Q000031:analogs & derivatives* / Q000737:chemistry / Q000493:pharmacokinetics / Q000627:therapeutic use
chemical_list: D020533:Angiogenesis Inhibitors; D013792:Thalidomide; C467566:pomalidomide