Danish company TopoTarget A/S reported European approval and a U.S. approvable letter for its first product, a drug designed to protect a cancer patient's tissue from the effects of anthracyclines upon accidental exposure during the administration of chemotherapy.
The company filed a new drug application with the FDA in January - and later received priority-review status - to market to U.S. hospitals Totect, a catalytic inhibitor of topoisomerase II. In its approvable letter, the agency requested some clarifying issues relating to the drug's manufacturing at European facilities.
"The good news is that we are only facing questions regarding the production of the product," said Peter Buhl Jenson, CEO of the Copenhagen, Denmark-based firm. The facilities are up to par with European regulations, and those represent "about a 95 percent overlap with FDA requirements."
Jenson declined to provide a specific timeline for satisfying the issues, but said he believes that "we will have the product in the U.S. in 2007, which has been our goal all along."
Totect is a small-molecule drug for treating extravasation, the accidental leakage of intravenously administered anthracycline chemotherapeutics, the "cornerstone of treatment in many cancer therapies," into healthy tissue, Jenson said.
It's a rare occurrence - happening is an estimated 0.1 percent to 1 percent of treatments - but extravasation results in skin necrosis that can lead to scarring, injury and postponement of further therapy.
"Occasionally, the [anthracycline] gets out of the veins," he said, "and the drug gets directly into the subcutaneous tissue. No one can predict when this happens, but when it does, it's very painful."
Jenson compared Totect to "a fire extinguisher" that hospitals can have on hand in case of extravasation anthracycline treatment. The product has to be administered within six hours of the leakage.
Upon approval, Totect would be the first product in the U.S. to treat extravasation. The FDA previously granted orphan drug designation.
The drug also has been given orphan status in the European Union, and the product, which is marketed in Europe under the name Savene, is expected to hit the U.S. market by the end of the year.
TopoTarget has established a sales force in Europe, and is "looking into different options," for getting the product to market in the U.S., Jenson told BioWorld Today.
The company already has some U.S. connections, including an ongoing collaboration with Branford, Conn.-based CuraGen Corp. to develop compounds from TopoTarget's histone deacetylase (HDAC) inhibitor library, including PXD101, in Phase I and Phase II trials in multiple cancer indications. That deal, signed in June 2004, could be worth up to $100 million to TopoTarget. (See BioWorld Today, June 7, 2004.)
Other drugs in its pipeline include Savicol, an HDAC inhibitor that began a pivotal Phase II trial in February against colon polyps in patients with familial adenomatous polyposis. Savicol has received orphan designation in both the U.S. and Europe.
TopoTarget also has the HDAC inhibitors Bececa, which is in a Phase II study in basal-cell carcinoma, and Avugane, which is in Phase II as a topical treatment for acne vulgaris.
TopoTarget reported a loss of DKK37.1 million (US$6.37 million) for the first quarter of 2006. As of March 31, it had cash, cash equivalent and marketable securities totaling DKK261 million.
The company's stock (CSE:TOPO) closed at DKK40 Thursday, up DKK1.40.