Ceptaris nets first-ever FDA approval for topical lymphoma chemo gel

Ceptaris Therapeutics won a first-of-its-kind approval from the FDA for its topical treatment for mycosis fungoides, the most common form of cutaneous T-cell lymphoma. The gel, applied directly to the skin, delivers the chemotherapeutic agent mechlorethamine, previously only approved for intravenous treatment.

The FDA granted market approval for Pennsylvania-based Ceptaris' orphan cancer drug Valchlor, designed to treat stage IA and IB mycosis fungoides-type cutaneous T-cell lymphoma (CTCL), itself a rare form of non-Hodgkin's lymphoma, for patients who have received prior skin-directed therapies. Because CTCL is characterized by lesions on the skin caused by malignant T cells, topical treatments of mechlorethamine--otherwise known as nitrogen mustard--have been used before, but Valchlor is the first with U.S. approval.

And with the FDA's go-ahead, Ceptaris met a milestone on its way to wrapping up a $250 million deal with Swiss biotech Actelion ($ATLN), which signed a deal to acquire the privately held company at the end of July. Last year, the U.S. agency denied Valchlor's approval, the reason for which was never specified.

In the 260-patient study, results of which were published in JAMA Dermatology, 60% of the patients treated with Valchlor had a confirmed response at 12 months, according to the company. Also, no systemic absorption of the cytotoxic mechlorethamine was detected in the Valchlor treatment.

"The use of topical mechlorethamine has been documented over several decades, but this is the first time that a product has gone through the rigorous FDA approval process," said Stuart R. Lessin, lead investigator in the pivotal trial and president of the board of directors of the Cutaneous Lymphoma Foundation. "Not only is Valchlor manufactured under FDA's good manufacturing practices, but it will also be accompanied by patient support and assistance programs which are not currently available with compounded mechlorethamine."

"With this approval, we look forward to working with Actelion to close the merger and make Valchlor available to patients," Ceptaris CEO Stephen Tullman said in a statement.

- here's the release
- and here's FierceBiotech's take