Ledaga(chlormethine gel)
Oncology· 249 cited findings· 1 source document
Company: Helsinn Birex
What the regulatory reviews say
Ledaga (chlormethine gel) is a medicinal product for which a marketing authorization application was submitted on 17 April 2015, following its orphan medicinal product designation on 22 May 2012. The gel has a concentration of 0.016% w/w chlormethine. The CHMP issued a positive opinion on 15 December 2016. The US approval date for chlormethine gel was August 2013.
In a clinical trial with 260 enrolled patients, the overall CAILS response rate (CR+PR) was 76.7%, with a complete CAILS response (CR) of 18.9% and a partial CAILS response (PR) of 57.8%. The ratio of CAILS response rates (Gel/AP) was 1.301 (95% CI: 1.065–1.609). In the ITT analysis set, the overall CAILS response rate (CR+PR) was 58.5%, with a ratio of CAILS response rates (Gel/AP) of 1.226 (95% CI: 0.974–1.552).
Safety findings included a pregnancy rate of 12.5%. Studies also reported chromosomal aberrations at various chlormethine concentrations: 6.9% at 0.1 mg/kg, 48.7% at 0.5 mg/kg, and 82.5% at 1 mg/kg. Gross malformations were observed in 40% and external malformations in 90% of fetuses. The NOAEL for DEGEE topical was 1000 mg/kg/day.
Key figures — cited to the source page
| Overall CAILS response rate (CR+PR) | 76.7 | Helsinn Birex — Ledaga EMA EPAR public assessment report (EM, p.41 |
| Ratio of CAILS response rates (Gel/AP) | 1.301 | Helsinn Birex — Ledaga EMA EPAR public assessment report (EM, p.41 |
| 95% CI for Ratio of CAILS response rates (Gel/AP) | 1.065–1.609 | Helsinn Birex — Ledaga EMA EPAR public assessment report (EM, p.41 |
| pregnancy rate | 12.5 | Helsinn Birex — Ledaga EMA EPAR public assessment report (EM, p.22 |
| chromosomal aberrations (chlormethine 1 mg/kg) | 82.5 | Helsinn Birex — Ledaga EMA EPAR public assessment report (EM, p.23 |
| gross malformations | 40 | Helsinn Birex — Ledaga EMA EPAR public assessment report (EM, p.23 |
| Marketing authorization application submission date | 17 April 2015 | Helsinn Birex — Ledaga EMA EPAR public assessment report (EM, p.5 |
| US approval date | August 2013 | Helsinn Birex — Ledaga EMA EPAR public assessment report (EM, p.5 |
Source documents
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Figures are as stated in the cited regulatory review documents (different drugs are studied in different trials — values are not head-to-head comparisons). Educational reference only — not medical or investment advice.