Kazia Therapeutics Ltd (NASDAQ:KZIA) gained ground Monday, after it disclosed that the U.S. Food and Drug Adminstration has granted Orphan Drug Designation to its paxalisib for the treatment of malignant glioma.
The Australian-based drug company went on to say malignant glioma also includes Diffuse Intrinsic Pontine Glioma (DIPG), a rare and highly aggressive childhood brain cancer.
Orphan Drug Designation (ODD) is a special status accorded to drugs which are considered promising potential treatments for rare (or 'orphan') diseases, generally defined as those which affect less than 200,000 cases per annum in the United States.
ODD can provide drug developers with up to seven years of Orphan Drug Exclusivity (ODE), 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
CEO James Garner, commented, "Taken together, RPDD and ODD provide a powerful suite of incentives, opportunities, and protections for the development of paxalisib in DIPG.
"We look forward to seeing initial data from the ongoing phase I study in DIPG at St Jude Children's Research Hospital during the second half of calendar 2020. In parallel, we are working closely with collaborators, advisors, and researchers to determine the best path forward for paxalisib in this devastating disease."
ODD exists to recognise the development of a drug for a rare disease, which may affect adults or children. ODD provides an additional period of 7.5 years data exclusivity (for a paediatric disease), which allows companies to better defend their products against competition.
It also results in a waiver by FDA of fees for a marketing application, under the Prescription Drug User Fees Act (PDUFA fees), which are just under US$ 3 million in FY2020.
KZIA shares began Monday up 27.7 cents, or 3.7%, from Friday’s close to $7.7569.
