Shares in Kazia Therapeutics Ltd (NASDAQ:KZIA, ASX:KZA), an oncology-focused biotech firm, saw shares add 10% in New York after the all powerful US FDA gave it key orphan drug status.
It was for its new investigational drug GDC-0084 for the treatment of glioblastoma multiforme - the most aggressive form of brain cancer.
Imminent start of Phase II clinical study
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.
Orphan drug designation (ODD) is a special status accorded to candidates, which are considered promising potential treatments for rare, or orphan diseases, generally defined as those which affect less than 200,000 cases per year in the United States.
It also provides potential for grant funding and financial benefits, such as a waiver of new drug application fees, and tax credits.
"We are very pleased to have successfully completed this important regulatory step in the development of GDC-0084," said Kazia chief executive Dr James Garner.
"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."