CellAct Pharma, a developer of innovative treatments for cancer, has announced that the Committee for Orphan Medicinal Products of the European Medicines Agency (EMA) has granted orphan drug designation to the company's CAP7.1, an adapted version of the well-established anticancer agent etoposide, for the treatment of biliary tract cancers. European Union (EU) orphan drug designation is given to products for the diagnosis, prevention or treatment of rare diseases that are life-threatening or very serious. A disease is defined as rare in the EU if it affects fewer than five in 10,000 people. Biliary tract cancer affects approximately 1.7 in 10,000 people in the EU. This is equivalent to a total of around 87,000 people.
The granting of EU orphan drug designation provides CellAct with development and commercial incentives, including a 10-year period of market exclusivity, access to a centralized review process, protocol assistance and scientific advice during product development, waiving or reduction of certain fees, and eligibility for grants and R&D support initiatives. CellAct is currently recruiting patients for a randomized, multicenter, proof-of-concept phase 2 study with CAP7.1 in adults with refractory biliary tract carcinomas in Germany (www.cap7-1.com).
"Following diagnosis, there are very few treatments options for biliary tract cancer patients. Granting of orphan drug designation could help expedite the regulatory process, if our clinical studies prove to be successful, and potentially get CAP7.1 to patients sooner," stated Nalân Utku, M.D., Chief Executive Officer of CellAct. "Recruitment into our phase 2 clinical study in Germany is progressing on schedule, with additional centers coming online each month."
A previous clinical study in adults with multiple and heavily pretreated solid tumors, including biliary tract cancers, showed promising safety and tolerability following treatment with CAP7.1. Many of these patients also showed signs of efficacy.
In addition to biliary tract cancers, a phase II study is also recruiting patients with therapy-refractory non-small and small cell lung cancers.