ROCKVILLE, Maryland—FDA has granted orphan drug designation to Cephalon's investigational agent Treanda (bendamustine HCl) for the treatment of chronic lymphocytic leukemia.

Orphan drug status is granted by FDA to promote the development of products that demonstrate promise for the treatment of rare diseases affecting fewer than 200,000 Americans annually. An estimated 15,000 new cases of CLL are diagnosed each year in the United States.

The orphan drug status entitles Cephalon to a 7-year period of marketing exclusivity in the United States for Treanda, if FDA approves the agent for the treatment of CLL.

In a press release, Cephalon said it has submitted a new drug application to FDA for Treanda for the treatment of CLL, based on data from a European study. This large multicenter phase III trial evaluated the safety and efficacy of Treanda, compared with chlorambucil(Drug information on chlorambucil), in patients with previously untreated CLL. The trial met both of its primary endpoints, overall response and progression-free survival, and demonstrated an acceptable tolerability profile, according to Cephalon. The company expects the data to be presented at the upcoming American Society of Hematology annual meeting in December 2007.

Treanda, designed as a purine analog/alkylator hybrid, combines the moieties of an antimetabolite and an alkylator.

Preclinical data show that the agent induces rapid, sustained single- and double-strand DNA damage, which results in apoptosis in the tumor, Cephalon said. These novel dual-action anti-tumor effects of Treanda may be attributed to its unique chemical design.

NHL trials

The company said it is also studying Treanda for the treatment of patients with indolent non-Hodgkin's lymphoma who are refractory to rituximab(Drug information on rituximab) (Rituxan) and in combination with rituximab in patients who have relapsed indolent non-Hodgkin's lymphoma or mantle cell lymphoma.