Duquesne Compound to Fight Brain Cancer Earns Orphan Drug Status

The promise of a Duquesne-created compound to treat one of the deadliest forms of brain cancer has led to a special designation by the U.S. Food and Drug Administration and the European Medicines Agency.

GangjeeThe Orphan Drug Designation for the compound FLAG-003, discovered in the lab of Dr. Aleem Gangjee, Distinguished Professor of medicinal chemistry, and licensed to FLAG Therapeutics Inc., will help to advance the new therapy through clinical testing and regulatory processes, said Dr. Frank Sorgi, FLAG's president and chief executive officer.

"The Orphan Drug Designation, which can lead to an expedited review, acknowledges drugs that are very important and show great promise, but impact a small group of people," said Dr. Alan Seadler, associate academic vice president for research. "I don't know of any other drug developed at Duquesne that has gotten this far in the process."

FLAG-003 has received the designation for treating glioma, which are the most aggressive and deadly forms of brain cancer. Survival rates are only 27 percent at two years and 10 percent at five years.

"This compound is an unprecedented, first-of-its-kind drug with multiple mechanisms of action in a single agent, providing combination chemotherapy to attack brain tumors on several fronts without pathways for escape," Gangjee explained.

Preliminary results indicate that FLAG-003 inhibits tumor growth significantly better than the currently approved chemotherapy, temozolomide (TMZ). FLAG-003 is a small molecule compound using dual action to inhibit both the growth of blood vessels in the tumors and the synthesis of DNA, stalling the growth of the tumor itself.

"The decision to grant FLAG-003 Orphan Drug Designation in both the U.S. and the European Union is an important milestone for our clinical program and underscores the urgency for a safe and effective treatment for these rare and deadly forms of brain cancer," said Sorgi.

North Carolina-based FLAG expects to initiate its Phase 1 study in the fourth quarter of 2016.

The Orphan Drug Designation is granted to drugs that are intended for safe and effective treatment, prevention or diagnosis of rare diseases/disorder affecting fewer than 200,000 Americans annually and fewer than five of 10,000 people in the European Union (EU). With this designation, FLAG Therapeutics is entitled to seven years of marketing exclusivity after product launch in the U.S. and 10 years in the EU, and is eligible to apply for research funding, tax credits, waiver or partial payment of application fees, protocol assistance and access to the central authorization procedure within the EU.

Originally printed in the Duquesne University Times

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