FDA grants orphan drug designation for cholangiocarcinoma therapy

Mundipharma EDO and Imbrium Therapeutics announced that the FDA granted orphan drug designation to etoposide toniribate for the treatment of cholangiocarcinoma, according to a press release.

“We are pleased that the FDA has recognized etoposide toniribate as a potential treatment for relapsed/refractory biliary tract cancer,” Thomas Mehrling, MD, PhD, CEO of Mundipharma EDO, said in the release. “As a company we are focused on developing treatments for rare and difficult-to-treat cancers and getting them to patients as rapidly as possible. We look forward to accelerating the development of etoposide toniribate, in conjunction with Imbrium Therapeutics, with a global phase 3 trial with sites in [the European Union], U.S., Australia and other countries.”

Etoposide toniribate, a small molecule topoisomerase inhibitor, demonstrated positive results in a phase 2 trial. The inhibitor is hydrolyzed at the site of the tumor by carboxylesterase, an enzyme overexpressed by some tumors, to produce activated etoposide, which binds to and inhibits topoisomerase II to break down tumor DNA.

“This designation represents Imbrium’s first milestone in oncology and underscores our commitment to advance the clinical development of oncology chemotherapeutics while actively collaborating to advance treatments across our therapeutic portfolio,” Paul Medeiros, president of Imbrium Therapeutics, said in the release.

Reference: www.mundipharma.com; www.imbriumthera.com