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Thalidomide

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.

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Thalidomide
PubChem
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.
Brand / code
Thalomid
Medicare
Part D (self-administered)

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.

Commercial insurance
covered with prior authorisation

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.

NICE recommendedNICE TA228 · 2011SMC: accepted
Appraised for
Untreated multiple myeloma with an alkylating agent and a corticosteroid (transplant-ineligible)
NHS England
Routinely funded for the appraised indication (or via managed access)

Sources: NICE TA228 · SMC advice: thalidomide. Funding decisions are indication-specific and change monthly; verify with NICE and your treating team.

Regulatory

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Approvals

RegionYearIndication
US1998Erythema nodosum leprosum
US2006Newly diagnosed multiple myeloma with dexamethasone
EU2008Untreated multiple myeloma ≥65 or transplant-ineligible, with melphalan-prednisone

Trials

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ClinicalTrials.gov · phase 2/3
refreshed 2026-09-09
367 studies2 recruiting77 Phase 232 Phase 3
Search “Thalidomide” on ClinicalTrials.gov →
Counts are from a name search and may include unrelated studies; up to 100 studies are summarised.

Recruiting now (live from ClinicalTrials.gov)

Recruiting trials near you · live from ClinicalTrials.gov
Thalidomide
intervention: Thalidomide
Open on ClinicalTrials.gov →

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Latest papers

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Latest papers · live from Europe PMC
Open in Europe PMC

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.

Connected

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