AbbVie Cancer Drug Gains Orphan Drug StatusBy
AbbVie reports that the European Medicines Agency (EMA) and the US 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. Glioblastoma multiforme is the most common and most aggressive type of malignant primary brain tumor.
â€‹Results from the Phase I clinical program evaluating ABT-414 in patients with recurrent or unresectable glioblastoma multiforme were presented at the 50th American Society of Clinical Oncology (ASCO) meeting in Chicago earlier this year.
Orphan drug designation is a status assigned to a medicine intended for use in rare diseases. In the US, 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 US or that are not expected to recover the costs of developing and marketing a treatment. 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. Orphan status provides sponsors with development and commercial incentives for designated compounds and medicines.