Helsinn Birex — Ledaga EMA EPAR public assessment report (EMEA/H/C/002826, 2017)
Helsinn Birex · 2017 · EMA EPAR public assessment report
Report summary
The EMA's Public Assessment Report for Ledaga (chlormethine gel) details its regulatory approval for topical treatment of mycosis fungoides-type cutaneous T-cell lymphoma (MF-type CTCL) in adults. The hybrid medicinal product, referencing Caryolysine, received a positive opinion on December 15, 2016. The report covers the product's chemical properties, manufacturing, and stability. Non-clinical studies confirmed chlormethine's genotoxic potential but showed no significant systemic exposure or toxicity with topical application. Pivotal clinical trials (Study 201 and 202) demonstrated the non-inferiority of 0.02% Ledaga compared to a compounded formulation, with comparable efficacy (CAILS, SWAT) and safety. Higher doses in Study 202 showed increased response rates. The safety profile primarily involved skin-related adverse events, with no measurable plasma concentrations of chlormethine. Risk management strategies address identified and potential risks.
Key findings — cited to the page
| Grade 4 AEs in AP arm | 10 n | · p.71 |
| patients with Grade 4 AEs in chlormethine gel arm | 5 n | · p.71 |
| patients with Grade 4 AEs in AP arm | 9 n | · p.71 |
| Skin irritation events | 17 events | · p.71 |
| Grade 4 AEs in chlormethine gel arm | 9 n | · p.71 |
| Complete CAILS Response (CR) | 14.7 % | Study 201 · p.41 |
| Overall CAILS response rate (CR+PR) | 58.9 % | Study 201 · p.41 |
| Partial CAILS Response (PR) | 44.2 % | Study 201 · p.41 |
| Number of patients in EE Analysis Set | 185 patients | Study 201 · p.39 |
| Number of patients in ITT excluding Site #7 Analysis Set | 242 patients | Study 201 · p.39 |
| Partial CAILS Response (PR) | 57.8 % | Study 201 · p.41 |
| Overall CAILS response rate (CR+PR) | 76.7 % | Study 201 · p.41 |
Source: Helsinn Birex ↗ · 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 →