H A C — Targretin EMA EPAR public assessment report (EMEA/H/C/000326, 2001)
H A C · 2001 · EMA EPAR public assessment report
Report summary
Targretin (bexarotene) is approved for advanced cutaneous T-cell lymphoma (CTCL) refractory to prior systemic treatment, with a recommended initial dose of 300 mg/m2/day. Preclinical studies demonstrated anti-cancer activity but also dose-related toxicities in animals, including coagulopathy, liver damage, and cataracts. Clinical trials, including two pivotal studies in refractory CTCL, showed objective response rates of 27-67%, varying by dose and disease stage. Common adverse events include hyperlipidemia (especially hypertriglyceridemia) and hypothyroidism, with pancreatitis being a serious risk linked to hyperlipidemia. Despite these, the overall benefit-risk profile for advanced CTCL patients is considered favorable.
Key findings — cited to the page
| overall response rate to Targretin (prior irradiation therapies) | 31 % | Targretin · p.33 |
| TSH concentration at baseline | 1.60 µU/ml | L1069-24 · p.30 |
| patients requiring treatment discontinuation due to hypertriglyceridemia (300 mg/m²/day initial dose) | 2.4 % | Targretin capsules · p.30 |
| serious adverse events | 3.1 % | · p.30 |
| deaths considered possibly related to bexarotene | 1 | · p.30 |
| treatment-related deaths in non-CTCL oncology patients | 2 | · p.30 |
| Therapeutic indication | Treatment of skin manifestations of advanced stage CTCL patients refractory to at least one systemic treatment | Targretin · p.1 |
| CTCL patients experiencing drug-related adverse events | 97.4 % | · p.31 |
| hypertriglyceridemia/hypercholesterolemia as dose-limiting toxicity (recommended posology) | 39 % | · p.31 |
| neutropenia/leucopenia as dose-limiting toxicity (recommended posology) | 3.6 % | · p.31 |
| objective response rate (CA) in refractory CTCL patients | 34 % | · p.32 |
| deaths in study L1069-24 | 17 | L1069-24 · p.30 |
Source: H A C ↗ · License: EMA - reuse permitted with acknowledgement. Summary and findings are extracted from the published document; every figure cites its page. Informational only — not investment advice. Explore the Oncology library →