Regulatory Affairs

EC approves Actelion's topical chlormethine gel to treat MF-Type CTCL

PBR Staff Writer Published 08 March 2017

Actelion has secured marketing authorization from the European Commission (EC) for its Ledaga (chlormethine gel) 160 micrograms/g to treat mycosis fungoides-type cutaneous T-cell lymphoma (MF-CTCL).

Ledaga is formulated as a topical, once-daily and colorless gel.

MF-CTCL is a rare and potentially life-threatening immune system cancer that forms on the skin. It is the common form of cutaneous T-cell lymphoma.

The approval was based on the results of the pivotal 201 study, which included around 260 patients.

In the randomized controlled study, around 77% of patients within the efficacy evaluable (EE) population who have been treated for about six months with chlormethine gel showed 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.

The 201 is a multicenter, observer-blinded, active-controlled and 12-month trial that included Stage I and IIA MF-type CTCL patients.

The trials was carried out in the 13 US centers to assess the efficacy and safety of chlormethine gel compared against chlormethine HCl 0.02% compounded in Aquaphor ointment.

Actelion has randomized 260 patients1:1 ratio to topical treatment with chlormethine gel or chlormethine HCl 0.02% compounded in Aquaphor ointment once daily for up to 12 months.

Since 2013, the chlormethine gel has been marketing in the US under the brand name Valchlor (mechlorethamine).

In France, the drug secured approval under temporary authorization for use (ATU) program, which started in the second half of 2014.

Earlier this year, Johnson & Johnson (J&J) agreed to acquire Actelion for $30bn. The Swiss biopharmaceutical firm discovers, develops and commercializes new drugs for diseases with significant unmet medical need.

Image: Actelion Research Center. Photo: courtesy of Actelion Pharmaceuticals Ltd.