FDA grants orphan designation to Kazia's GDC-0084


Kazia Therapeutics (ASX:KZA), an Australian oncology-focused biotechnology company formerly known as Novogen, has announced the US FDA has granted Orphan Drug Designation (ODD) to its investigational new drug, GDC-0084, for the treatment of glioblastoma multiforme, the most common and most aggressive form of primary brain cancer.

The company received written notification from the regulator on Friday 23 February 2018.

ODD is a special status accorded to drugs considered promising potential treatments for rare (‘orphan’) diseases. The designation can provide drug developers with up to seven years of exclusivity, extending the effective life of a commercial product. It also provides opportunities for grant funding, protocol assistance, and financial benefits, such as a waiver of New Drug Application fees and tax credits.

GDC-0084 was licensed from Genentech in October 2016 after demonstrating favourable results in a phase 1 study of 47 patients with advanced brain cancer.

It is due to commence a phase 2 clinical trial in glioblastoma in late March or early April this year. Glioblastoma multiforme (GBM) is an area of significant unmet medical need. More than 130,000 patients are diagnosed worldwide each year, and the prognosis remains poor, with median survival of 12-15 months on best available care. Existing drug treatments are largely ineffective in almost two-thirds of patients, and there remains an urgent need for new therapies.

Kazia said it intends to shortly commence an international phase 2 clinical study designed to provide definitive evidence of efficacy.

This study will initially be conducted predominantly at leading centres in the US. It is anticipated to provide an initial data read-out in early calendar 2019.

According to Kazia CEO, Dr James Garner, “We are very pleased to have successfully completed this important regulatory step in the development of GDC-0084. 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 II clinical study, and look forward to working closely with the participating clinicians.”