AbbVie Gets Both FDA And EMA Orphan Status For GBM Drug

AbbVie reported that it has received Orphan Drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for its investigational drug ABT-414 for the treatment of glioblastome multiforme (GBM).

ABT-414 is an anti-epidermal growth factor receptor antibody drug conjugate designed to be stable in the blood stream and release cytotoxic agents only once inside cancer cells. AbbVie developed the drug with in-licensed components from Seattle Genetics and Life Science Pharmaceuticals. The compound is currently undergoing investigation in GBM and squamous cell tumors.

GBM is the leading and most aggressive type of malignant primary brain tumor. Two to three people out of every 100,000 are diagnosed in the U.S. and EU every year with GBM. The disease has a very poor prognosis with only about 4 percent of patients reaching a survival rate of five years.

Gary Gordon, VP of oncology clinical development at AbbVie, said, “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. 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.”

Earlier this year AbbVie presented results from the ABT-414’s Phase I clinical program investigating the drug in patients with recurrent or unresectable glioblastoma multiforme at the 50th American Society of Clinical Oncology (ASCO) meeting held in Chicago. The company reported that ABT-414 achieved four partial responses including one complete response in patients with recurrent or unresectable GBM. “The complete response and three partial responses call for further evaluation of this compound in this extremely difficult-to-treat patient population,” said Hui Gan, medical oncologist and senior research fellow at the Austin Health and Ludwig Institute for Cancer Research in Heidelberg, Australia.

The company said later stage clinical trials are in the works to further investigate ABT-414 in patients with GBM.