LEDAGA® (chlormethine gel) given ‘positive opinion’ by the EMA CHMP for the treatment of MF-CTCL

Last month (16^th December 2016), the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) gave a positive opinion for using 160mg/g chlormethine gel (LEDAGA®) to treat Mycosis Fungoides-Type Cutaneous T-Cell Lymphoma (MF-CTCL) in adult patients.

Actelion’s LEDAGA® received this positive opinion based on pivotal results of the 201 study (NCT00168064), which included 260 patients with stage I and IIA MF-CTCL making it the largest controlled study to be carried out in this disease area so far. Study 201 was a randomized, observer-blinded, active controlled study, and took place over one year in 13 US centers. The aim of the study was to compare the safety and efficacy of chlormethine gel to chlormethine HCI 0.02% compounded in AQUAPHOR® ointment.

• Clinical response via Composite Assessment of Index Lesion Severity (CAILS) score in patients treated with chlormethine gel for ≥6 months = 77%
• Clinical response in compounded control = 59%
• Time to first confirmed response favored chlormethine gel rather than compounded control
• CR was achieved in 19% and 15% of chlormethine gel treated patients and compounded control, respectively
• Mean lesion severity decreases were seen as early as one month into the study; continuing therapy was associated with further reductions
• Most common AEs reported with chlormethine gel were dermatitis (54.7%), pruritus (20.3%), skin infections (11.7%), skin ulceration and blistering (6.3%), and skin hyperpigmentation (5.5%)
• No systemic absorption of chlormethine was reported

Chlormethine is an alkylating agent. LEDAGA® is chlormethine formulated as a topical, once-daily, colorless gel. Since 2013, chlormethine gel has been commercially available in the US under the brand name VALCHLOR® (mechlorethamine) and, since 2016, in Israel through special import authorization procedures. A temporary “Authorization for Use” program, initiated in the later stages of 2014, renders the drug available to patients in France.