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 multiform. [1] Glioblastoma multiforme or GBM 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]

Prior to diagnosis, most patients with glioblastoma multiforme experience a serious symptoms such as a seizures. [6] Typically patients succumb to the disease approximately 15 months after diagnosis. [2][6] Treatment remains challenging and, to-date, no long-term treatments are available. Standard treatment is surgical resection, radiotherapy and concomitant adjunctive chemotherapy. [2]

Investigational drug: ABT-414 
ABT-414 is an investigational anti-EGFR (epidermal growth factor receptor) monoclonal antibody drug conjugate or  ADC being evaluated for the treatment of patients with various cancer and tumor types. As an ADC, ABT-414 is designed to be stable in the bloodstream and only release the potent cytotoxic agent once inside targeted cancer cells. Developed by AbbVie researchers with components in-licensed from Life Science Pharmaceuticals, Inc. and Seattle Genetics, ABT-414 is currently being investigated for the treatment of glioblastoma multiforme, the most common and most aggressive malignant primary brain tumor. ABT-414 is also in clinical trials for the treatment of patients with squamous cell tumors. ABT-414 is an investigational compound and its efficacy and safety have not been established by the FDA.

“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,” explained Gary Gordon, M.D., vice president, oncology clinical development at AbbVie. Commenting on the EMA and FDA decisions, Gordon further noted “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.” AbbVie is a research-based biopharmaceutical company formed in 2013 following separation from Abbott Laboratories.

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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. [3]

Orphan Drug Designation: Europe and U.S.
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.

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