FDA News

FDA grants orphan drug designation to GDC-0084 for glioblastoma multiforme

The FDA granted orphan drug designation to GDC-084 for the treatment of patients with glioblastoma multiforme, the most aggressive form of primary brain cancer, according to the agent’s manufacturer.

GDC-0084 is a small molecule inhibitor of the PI3K/AKT/mTOR pathway. Kazia Therapeutics licensed the agent from Genentech in 2016.

“We are very pleased to have successfully completed this important regulatory step in the development of GDC-0084,” James Garner, Kazia CEO said in a press release. “We share FDA's recognition of the need for new treatments in this very challenging disease, and we believe that GDC-0084 has great promise as a potential new therapy. We anticipate an imminent start of the phase 2 clinical study and look forward to working closely with the participating clinicians."

A phase 2 trial is set to begin next month.

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.

The FDA granted orphan drug designation to GDC-084 for the treatment of patients with glioblastoma multiforme, the most aggressive form of primary brain cancer, according to the agent’s manufacturer.

GDC-0084 is a small molecule inhibitor of the PI3K/AKT/mTOR pathway. Kazia Therapeutics licensed the agent from Genentech in 2016.

“We are very pleased to have successfully completed this important regulatory step in the development of GDC-0084,” James Garner, Kazia CEO said in a press release. “We share FDA's recognition of the need for new treatments in this very challenging disease, and we believe that GDC-0084 has great promise as a potential new therapy. We anticipate an imminent start of the phase 2 clinical study and look forward to working closely with the participating clinicians."

A phase 2 trial is set to begin next month.

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.