Actelion has bagged European Union Approval for Ledaga, its treatment for mycosis fungoides-type cutaneous T-cell lymphoma (MF-CTCL), a rare potentially life-threatening immune system cancer that appears in the skin.
The condition is the most common form of cutaneous T-cell lymphoma, and its course in patients is unpredictable. In about 34 percent of cases disease progression is observed, and in the most advanced stages, MF-CTCL cells can metastasise to other body tissues, including the liver, spleen and lungs.
MF-CTLC typically appears in patients over 50 years of age and is more common in men. The disease presents first as dry skin and a red rash, with or without itching. Because of this, MF-CTLC is often mistaken for eczema or psoriasis, delaying diagnosis. It goes on to form scaly plaques on the skin, which can cover small or large areas of the skin. Large bumps or tumour nodules may also develop, and lymph nodes may be involved.
Current research suggests that patients who are diagnosed in early stages of MF-CTCL have a normal life expectancy, however, the average time to diagnosis ranges from two to seven years. A key therapeutic aim is to prevent disease progression.
Ledaga (chlormethine) is an alkylating drug formulated as a topical, once-daily, colourless gel. Its approval was based on results of the pivotal 201 study, the largest randomised controlled study ever conducted in early stage MF-CTCL, involving 260 patients.
This showed that 77 percent of evaluable patients who were treated for at least six months with Ledaga achieved a clinical response in the Composite Assessment of Index Lesion Severity (CAILS) score, while 59 percent of those treated with the compounded control had a clinical response.
Also, a complete response was achieved in 19 percent of patients treated with the drug versus 15 percent in the control group, with reductions in mean CAILS scores seen as early as four weeks into the study.
The most frequent adverse reactions reported with Ledaga were skin related: dermatitis (54.7 percent), pruritus (20.3 percent), skin infections (11.7 percent), skin ulceration and blistering (6.3 percent), and skin hyperpigmentation (5.5 percent).
Actelion said it is now “working diligently” to launch the drug in the EU “as rapidly as possible”.