{"entity":{"id":"thalidomide","kind":"drug","name":"Thalidomide","aka":[],"tldr":"Thalidomide is the drug behind the 1960s birth-defect tragedy, rehabilitated as the first immunomodulatory myeloma drug and the parent of lenalidomide and pomalidomide.","summary":"Singhal (NEJM 1999) showed activity in refractory myeloma; approved 2006 with dexamethasone for newly diagnosed disease under the strict REMS-type distribution programme first built for its leprosy indication (1998). Still used in low-cost regimens worldwide (thalidomide-based induction/maintenance, e.g. in Myeloma XI and LMIC settings). Neuropathy, thrombosis and sedation limit it.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Thalidomide","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Thalidomide"}],"tags":["gap-fill","historic"],"related":[],"cancers":["multiple-myeloma"],"sections":[],"technologies":["protac-degrader","antiangiogenic"],"targets":["ikzf1"],"drugs":[],"companies":["bms"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Thalomid","modality":"Oral cereblon modulator (first IMiD)","mechanism":"Binds cereblon, altering E3 ligase substrate specificity (IKZF1/3 degradation), with anti-angiogenic and immunomodulatory effects; the mechanism explained decades after its teratogenic disaster.","approvals":[{"region":"US","year":1998,"indication":"Erythema nodosum leprosum"},{"region":"US","year":2006,"indication":"Newly diagnosed multiple myeloma with dexamethasone"},{"region":"EU","year":2008,"indication":"Untreated multiple myeloma ≥65 or transplant-ineligible, with melphalan-prednisone"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2006-05-25","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Newly diagnosed multiple myeloma"},{"date":"2014-06-19","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2006 converted to traditional approval 8.1 years after it was granted.","indication":"Newly diagnosed multiple myeloma"}]},"route":"/drugs/thalidomide/","neighbours":{"cancer":[{"id":"multiple-myeloma","kind":"cancer","name":"Multiple myeloma","route":"/cancers/multiple-myeloma/"},{"id":"myeloma-transplant-ineligible","kind":"cancer","name":"Newly diagnosed multiple myeloma, transplant-ineligible","route":"/cancers/myeloma-transplant-ineligible/"}],"technology":[{"id":"antiangiogenic","kind":"technology","name":"Anti-angiogenic therapy","route":"/technologies/antiangiogenic/"},{"id":"cipn-recovery-and-treatment","kind":"technology","name":"Nerve damage from chemotherapy: what recovers, and what helps","route":"/technologies/cipn-recovery-and-treatment/"},{"id":"protac-degrader","kind":"technology","name":"PROTACs & molecular glues (targeted protein degradation)","route":"/technologies/protac-degrader/"}],"target":[{"id":"ikzf1","kind":"target","name":"IKZF1 (Ikaros)","route":"/targets/ikzf1/"}],"company":[{"id":"bms","kind":"company","name":"Bristol Myers Squibb","route":"/companies/bms/"}],"trial":[{"id":"cassiopeia","kind":"trial","name":"CASSIOPEIA","route":"/trials/cassiopeia/"},{"id":"nct00083382","kind":"trial","name":"Combination Bisphosphonate and Anti-Angiogenesis Therapy With Pamidronate and Thalidomide","route":"/trials/nct00083382/"},{"id":"nct00033332","kind":"trial","name":"Dexamethasone With or Without Thalidomide in Treating Patients With Newly Diagnosed Multiple Myeloma","route":"/trials/nct00033332/"},{"id":"nct06017284","kind":"trial","name":"Thalidomide to Chemotherapy Related Nausea and Vomiting in Pancreatic Cancer","route":"/trials/nct06017284/"}],"paper":[{"id":"paper-cassiopeia-lancet-2019","kind":"paper","name":"CASSIOPEIA: daratumumab added to bortezomib, thalidomide and dexamethasone before and after transplant in newly diagnosed myeloma","route":"/key-papers/paper-cassiopeia-lancet-2019/"}],"term":[{"id":"imid","kind":"term","name":"Immunomodulatory drugs (IMiDs) and CELMoDs","route":"/terms/imid/"},{"id":"vte","kind":"term","name":"Venous thromboembolism (VTE)","route":"/terms/vte/"}]}}