NORTH CHICAGO, Ill., Aug. 4, 2014 /PRNewswire/ -- AbbVie (NYSE:ABBV) today announced the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) have granted orphan drug designation to AbbVie's investigational compound ABT-414, an anti-epidermal growth factor receptor antibody drug conjugate, which is being evaluated for safety and efficacy in patients with glioblastoma multiforme. 1 Glioblastoma multiforme is the most common and most aggressive type of malignant primary brain tumor. Each year in the U.S. and Europe, two to three out of every 100,000 people are diagnosed with glioblastoma multiforme, which has a five year survival rate of approximately four percent. 2
"The orphan drug designation is an important regulatory advancement as we further our development in recurrent glioblastoma multiforme, a disease that is uniformly fatal with limited treatment options," said Gary Gordon, M.D., vice president, oncology clinical development, AbbVie. "We are pleased to continue developing ABT-414 in Phase II trials in patients with glioblastoma multiforme based on the results of our Phase I program."
Results from the Phase I clinical program evaluating ABT-414 in patients with recurrent or unresectable glioblastoma multiforme were presented at the 50 th American Society of Clinical Oncology (ASCO) meeting in Chicago earlier this year. 3
About Orphan Drug Designation Orphan drug designation is a status assigned to a medicine intended for use in rare diseases. 4 In the U.S., the Orphan Drug Designation program provides orphan status to medicines intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. or that are not expected to recover the costs of developing and marketing a treatment. 5 In Europe, a medicine must meet similar criteria to be granted orphan status. The criteria include that the medicine must be intended for the treatment, prevention or diagnosis of a disease that is life threatening and has a prevalence in the EU of up to five in 10,000, and the intended medicine must aim to provide significant benefit to those affected by the condition. 4 Orphan status provides sponsors with development and commercial incentives for designated compounds and medicines.The approval of an orphan designation request does not alter the standard regulatory requirements and process for obtaining marketing approval for an investigational use. Sponsors must establish safety and efficacy of a compound in the treatment of a disease through adequate and well-controlled studies.