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Thalidomide is the drug behind the 1960s birth-defect tragedy, rehabilitated as the first immunomodulatory myeloma drug and the parent of lenalidomide and pomalidomide.
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.
Binds cereblon, altering E3 ligase substrate specificity (IKZF1/3 degradation), with anti-angiogenic and immunomodulatory effects; the mechanism explained decades after its teratogenic disaster. Connects to IKZF1 (Ikaros).
1.Thalidomide binds IKZF1 (Ikaros) with one end and an E3 ubiquitin ligase with the other.
Oral, self-administered, so it is a Part D drug: covered through a stand-alone Part D plan or Medicare Advantage drug benefit, usually on the specialty tier with 25 to 33% coinsurance until the annual cap ($2,000 in 2025, $2,100 in 2026). Generic thalidomide is available; REMS-restricted dispensing.
Covered for FDA-labelled and NCCN-listed uses, but almost always behind prior authorisation confirming diagnosis, biomarker and line of therapy; dispensed through a specialty pharmacy.
Part D out-of-pocket capped at $2,000 (2025) / $2,100 (2026). Medicare patients cannot use manufacturer co-pay cards; charity funds (PAN, HealthWell, CancerCare) and the Extra Help subsidy are the routes.
Sources: Medicare.gov: Drug coverage (Part D) · Medicare.gov: Costs for Medicare drug coverage (annual out-of-pocket cap). Not medical or financial advice; verify with your plan.
Sources: NICE TA228 · SMC advice: thalidomide. Funding decisions are indication-specific and change monthly; verify with NICE and your treating team.
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Newly diagnosed multiple myeloma
Accelerated approval on a surrogate endpoint, with a confirmatory trial required.
Newly diagnosed multiple myeloma
Confirmed: the accelerated approval of 2006 converted to traditional approval 8.1 years after it was granted.
| Region | Year | Indication |
|---|---|---|
| US | 1998 | Erythema nodosum leprosum |
| US | 2006 | Newly diagnosed multiple myeloma with dexamethasone |
| EU | 2008 | Untreated multiple myeloma ≥65 or transplant-ineligible, with melphalan-prednisone |
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Query for this drug: (TITLE:"Thalidomide" OR ABSTRACT:"Thalidomide" OR TITLE:"Thalomid" OR ABSTRACT:"Thalomid") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Thalidomide, not a curated reading list.
Shares Combination Bisphosphonate and Anti-Angiogenesis Therapy With Pamidronate and Thalidomide, Dexamethasone With or Without Thalidomide in Treating Patients With Newly Diagnosed Multiple Myeloma, Multiple myeloma.
Shares Immunomodulatory drugs (IMiDs) and CELMoDs, Newly diagnosed multiple myeloma, transplant-ineligible, Multiple myeloma.
Shares IKZF1 (Ikaros), Newly diagnosed multiple myeloma, transplant-ineligible, Multiple myeloma.
Shares Immunomodulatory drugs (IMiDs) and CELMoDs, Newly diagnosed multiple myeloma, transplant-ineligible, Multiple myeloma.
Shares Newly diagnosed multiple myeloma, transplant-ineligible, PROTACs & molecular glues (targeted protein degradation), Multiple myeloma.
Shares CASSIOPEIA, Multiple myeloma.
Shares Combination Bisphosphonate and Anti-Angiogenesis Therapy With Pamidronate and Thalidomide, Dexamethasone With or Without Thalidomide in Treating Patients With Newly Diagnosed Multiple Myeloma, Multiple myeloma.
Shares Newly diagnosed multiple myeloma, transplant-ineligible, PROTACs & molecular glues (targeted protein degradation), Multiple myeloma.