Sun Industries B V — Odomzo EMA EPAR public assessment report (EMEA/H/C/002839, 2015)
Sun Industries B V · 2015 · EMA EPAR public assessment report
Report summary
The EMA's 2015 assessment of Odomzo (sonidegib) for locally advanced basal cell carcinoma (BCC) not amenable to surgery or radiation recommended a 200 mg daily oral dose. Non-clinical studies revealed potent Hedgehog pathway inhibition but also significant dose-dependent toxicities, including skeletal, dental, reproductive, and neurological effects. Human pharmacokinetics showed slow elimination (half-life 13-28 days) and increased absorption with high-fat meals. The pivotal Phase II BOLT study demonstrated objective response rates of 54.8% (200mg) and 47.3% (800mg) in laBCC, with median progression-free survival of 22.1 and 19.4 months, respectively. The metastatic BCC indication was withdrawn due to lower efficacy. Safety concerns include muscle-related events, reproductive toxicity, and potential secondary malignancies, necessitating a pregnancy prevention program.
Key findings — cited to the page
| blood CPK elevation (Grade 3-4, sonidegib 200 mg) | 6.3 % | A2201 · p.97 |
| patients with CTC grade 3/4 toxicity (sonidegib 800 mg) | 56 % | A2201 · p.97 |
| patients with grade 3-4 AEs suspected to be study drug related | 23.3 % | X2101 · p.95 |
| patients with Grade 3-4 events (sonidegib 200 mg) | 30.4 % | A2201 · p.97 |
| muscle spasms (Grade 3-4, sonidegib 200 mg) | 2.5 % | A2201 · p.97 |
| probability of grade 3 or 4 CK elevation at median Cmax for 800 mg qd | 0.112 | · p.49 |
| NOEC (Daphnia magna Reproduction Test) | 14.9 µg/L | · p.32 |
| patients experiencing grade 3-4 AEs | 60 % | X2101 · p.94 |
| increased blood creatine phosphokinase (grade 3-4, drug-related) | 17.5 % | X2101 · p.95 |
| increased AST (grade 3-4, drug-related) | 3.9 % | X2101 · p.95 |
| probability of grade 3 or 4 CK elevation at median Cmax for 200 mg qd | 0.039 | · p.49 |
| CTC grade 4 AE (elevation of blood CPK) | 2.5 % | A2201 · p.99 |
Source: Sun Industries B V ↗ · 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 →