FDA grants orphan drug designation to TREANDA

Aug 29 2007

Cephalon, Inc. announced today that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development granted orphan drug designation for the company's investigational therapy, TREANDA (bendamustine HCl), for the treatment of chronic lymphocytic leukemia (CLL).

According to the National Cancer Institute, CLL is a slowly progressing blood and bone marrow disease with an estimated 15,000 new cases diagnosed every year in the United States.

Orphan drug status is granted by the FDA to promote the development of products that demonstrate promise for the treatment of rare diseases affecting less than 200,000 Americans annually. The orphan drug designation would entitle Cephalon to a seven-year period of marketing exclusivity in the United States for TREANDA, if approved by FDA for the treatment of CLL.

In the third quarter of 2007, Cephalon plans to submit a new drug application to the FDA for TREANDA for the treatment of patients with CLL. The company also is studying TREANDA for the treatment of patients with indolent (or slowly-progressing) non-Hodgkin's lymphoma (iNHL), who are refractory to the monoclonal antibody rituximab.

Cephalon is committed to advancing medications for orphan diseases. The company's first product, PROVIGIL(R) (modafinil) Tablets [C-IV], was launched with an orphan drug designation for narcolepsy in 1999. Other Cephalon therapies with orphan drug designation include TRISENOX(R) (arsenic trioxide) injection and CEP-701 (lestaurtinib), which is in development.

TREANDA is the first rationally designed purine analog / alkylator hybrid, combining the moieties of an antimetabolite and an alkylator. Preclinical data show that TREANDA induces rapid, sustained single- and double-strand DNA damage, which results in apoptosis, or programmed cell death in the tumor. TREANDA also induces mitotic checkpoint inhibition, which results in non-apoptotic cell death. These novel dual-action, anti-tumor effects of TREANDA may be attributed to its unique chemical design.

Cephalon holds exclusive rights to market and develop TREANDA in the United States. TREANDA is licensed from Astellas Deutschland GmbH. Bendamustine HCl, the active ingredient in TREANDA, is marketed in Germany by Astellas' licensee, MundiPharma International Limited, under the tradename RIBOMUSTIN(R). In Germany, RIBOMUSTIN is indicated as a single-agent or in combination with other anti-cancer agents for indolent NHL, multiple myeloma, and CLL. SymBio Pharmaceuticals Ltd holds exclusive rights to market and sell bendamustine HCl in Asia.

In 2005, Cephalon created a strategic business unit, Cephalon Oncology, focused on the development and commercialization of oncology products and resources for patients and healthcare providers. The Cephalon Oncology portfolio includes a number of promising investigational and marketed compounds. In addition to TREANDA, the Cephalon Oncology therapeutic portfolio in the United States includes TRISENOX, a product approved in the United States for the treatment of patients with relapsed or refractory acute promyelocytic leukemia (APL), and CEP-701, an oral small molecule inhibitor of tyrosine kinases including FLT-3, TRK and JAK-2, in phase 3 development for acute myeloid leukemia (AML).

http://www.cephalon.com/