CHMP recommends Ledaga (chlormethine gel) to treat mycosis fungoides-type cutaneous T-cell lymphoma (MF-CTCL) -Actelion.

Actelion Ltd announced that the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMA), issued a positive opinion for the use of chlormethine gel 160 micrograms/g (Ledaga) for the treatment of mycosis fungoides-type cutaneous T-cell lymphoma (MF-CTCL) in adult patients and recommended that the European Commission approves the product.

The CHMP opinion is based on the results of the pivotal 201 study, the largest randomized controlled study ever conducted in MF-CTCL involving 260 patients. In this study 77% of patients who were treated for at least 6 months with chlormethine gel achieved a clinical response, in the Composite Assessment of Index Lesion Severity (CAILS) score, while 59% of those treated with the compounded control had a clinical response. A response was defined as an at least 50% improvement in the baseline CAILS score. Complete response was achieved in 19% of patients versus 15% of patients treated with the compounded control. Reductions in mean lesion severity were seen as early as four weeks into the study, with further reductions observed with continuing therapy. The time to first confirmed response favored chlormethine gel (Ledaga) compared to the compounded control.

MF-CTCL is a rare, potentially life-threatening immune system cancer that appears in the skin. MF-CTCL is usually a chronic disease and the course of disease in individual patients is unpredictable. In around 10% of cases, MF-CTCL cells can metastasize to other body tissues, including the liver, spleen and lungs.

Comment: Chlormethine gel, under the brand name Valchlor(mechlorethamine) is commercially available in the US since 2013 and in Israel through special import authorization procedure since 2016. In France, patients benefit from the drug under a temporary authorization for use ("ATU") program initiated during the second half of 2014.