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FDA Approves Valchlor (Mechlorethamine Gel) for Lymphoma Patients

First topical formulation of nitrogen mustard (August 26)

The FDA has granted marketing approval for the orphan drug Valchlor (mechlorethamine gel, Ceptaris Pharmaceuticals) for the topical treatment of stage IA and IB mycosis fungoides-type cutaneous T-cell lymphoma (CTCL) in patients who have received prior skin-directed therapy. It is the first FDA-approved topical formulation of mechlorethamine, commonly known as nitrogen mustard.

Mechlorethamine is a chemotherapeutic agent previously approved for intravenous treatment of mycosis fungoides, the most common type of CTCL. Topical mechlorethamine preparations are currently recommended for the treatment of early-stage CTCL by the National Comprehensive Cancer Network (NCCN).

Prior to the approval of Valchlor, there were no FDA-approved topical mechlorethamine products; only nonstandardized, pharmacy-compounded petroleum ointment or aqueous-based topical preparations were available.

The agency’s approval of Valchlor was based on a randomized, observer-blinded, non-inferiority pivotal trial that compared the product with a pharmacy-compounded mechlorethamine preparation in 260 patients with stage IA–IIA mycosis fungoides-type CTCL. The patients had received at least one skin-directed therapy.

In the study, 60% of patients treated with Valchlor showed a confirmed response — defined as reduction of at least 50% in the Composite Assessment of Index Lesion Severity (CAILS) score — at 12 months, compared with 48% of those treated with the compounded control.

The results of this study were published earlier this year in JAMA Dermatology.

Source: Ceptaris Phermaceuticals; August 26, 2013.

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