Aslan Pharmaceuticals, a biotech company focused on the development of immunotherapies and targeted agents for Asia prevalent tumor types, has received Orphan Drug designation for its pan-HER inhibitor ASLAN001 (varlitinib) from the FDA Office of Orphan Products Development (OOPD). The indication for the Orphan Drug designation is cholangiocarcinoma, a rare and very aggressive form of bile duct cancer that has no cure.
The FDA awards Orphan Drug designation to advance the evaluation and development of drugs and products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. The OOPD evaluates scientific and clinical data submissions from sponsors to identify and designate products as promising for rare diseases and to further advance scientific development of such promising medical products. Orphan Drug designation will provide Aslan certain incentives including extended marketing exclusivity to develop ASLAN001 in cholangiocarcinoma.
ASLAN001 currently is being tested in phase II studies in breast cancer and cholangiocarcinoma. Studies in gastric cancer also are being planned.
Cholangiocarcinoma is a heterogeneous cancer, which may show genetic variation in different populations, and its low prevalence in the West makes it difficult to study. In Asia, the incidence is more than 10 times higher. The access to patients and physicians with knowledge of the disease will be key in studying the effects of ASLAN001 in this disease.
“The Orphan Drug designation is an important step in the development of ASLAN001. With no approved targeted therapies for cholangiocarcinoma, the prognosis for patients in both Asia and the West is poor,” said Dr. Carl Firth, chief executive officer of ASLAN.