NewLink Genetics Corporation, a biopharmaceutical company focused on discovering, developing and commercializing novel immunotherapeutic products, has announced that the European Commission (EC) has designated HyperAcute-Pancreas Immunotherapy (algenpantucel-L) as an orphan medicinal product under Regulation (EC) No. 141/2000 of the European Parliament and of the Council of 16 December 1999 on Orphan Medicinal Products.  

NewLink filed the application for orphan designation in June 2012. A positive opinion was adopted by the Committee for Orphan Medicinal Products (COMP) and was formally accepted by the European Commission.

Finalization of the Public Summary is projected for November, 2012.  Algenpantucel-L is an "off-the-shelf" product candidate currently being studied in IMPRESS (Immunotherapy Pancreas Resected Survival Study), an open-label, randomized, controlled, multi-centre, phase III clinical trial of approximately 700 Stage I and Stage II surgically-resected pancreatic cancer patients, which is being performed under a Special Protocol Assessment (SPA) with the United States Food and Drug Administration (FDA).

As a result of the orphan designation NewLink will have access to multiple incentives for the development of the drug in the EU, including reduced fees during development, access to the centralized authorization procedure (a single application for all EU countries), ten years of market exclusivity, and reduced fees for marketing authorization applications, pre-marketing inspections and multiple post-approval fees. Currently, algenpantucel-L has both orphan drug designation and Fast-track status in the United States.

"As emphasised during National Pancreatic Cancer Awareness Month in November, there is a desperate need for new treatment options for this disease and the European Commission's decision to grant orphan drug designation to algenpantucel-L should help us to address this unmet medical need," said Dr Charles Link, CEO and chairman of NewLink Genetics. "We believe that orphan designation in the European Union will significantly enhance our ability to bring this product globally to patients who have pancreatic cancer."

"Orphan drug designation provides significant incentives for us to accelerate clinical development in the EU," said Dr Nick Vahanian. "We believe that this designation, in combination with data from our ongoing 700 patient IMPRESS Phase 3 study of algenpantucel-L, will expedite advancement of this product through the approval process in Europe.”

As established by the European Medicines Agency (EMA), orphan designation is granted to product candidates intended for the diagnosis, prevention or treatment of life-threatening or chronically debilitating conditions affecting no more than five in 10,000 people in the EU and for whom satisfactory alternative therapies are not available or where they do exist, the orphan product will be of significant benefit to those affected by that condition. Applications are reviewed by the COMP and encompass evaluations of the underlying science and clinical experience with the product.

NewLink's algenpantucel-L is an "off-the-shelf" immunotherapy product candidate that consists of a group of two allogeneic pancreatic cancer tumor cell lines that were genetically modified to express Alpha-Gal.  These cell lines were chosen to provide a broad coverage of pancreatic cancer antigens.  Each of the modified cell lines is grown in large cultures, harvested, irradiated and packaged.  Approximately 150 million cells of each HyperAcute Pancreas cell line are given by intradermal injection with each treatment.

Pancreatic cancer has generally been recognized as an aggressive form of cancer with non-specific initial symptoms, making it difficult to diagnose at an early stage. Due to the difficulty in diagnosis and the aggressive nature of this cancer, the National Cancer Institute estimates a 96% mortality rate is associated with this disease, and the American Cancer Society estimates one-year and five-year overall survival rates of about 24% and 5%, respectively.

Pancreatic cancer can generally be divided into three broad categories: local disease, in which the cancer is confined to the pancreas and can be removed surgically, which is called resection; locally advanced disease, in which the cancer has spread locally and may or may not be eligible for resection because it has invaded tissues that should not be removed, such as key nerves and arteries; and metastatic disease, in which the tumour has spread beyond the region of the pancreas. NewLink's algenpantucel-L is being studied in phase III studies in both local disease and locally advanced disease.

NewLink Genetics Corporation is a biopharmaceutical company focused on discovering, developing and commercializing novel immunotherapeutic products to improve treatment options for cancer patients.