Kazia Therapeutics (ASX:KZA) - CEO, James Garner
CEO, James Garner
Source: Kazia Therapeutics
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  • The US FDA has awarded Kazia Therapeutics (KZA) Orphan Drug Designation (ODD) for its paxalisib drug to treat a rare type of childhood brain cancer
  • The designation is for paxalisib to treat atypical rhabdoid/teratoid tumours which is a rare or ‘orphan’ type of childhood brain cancer
  • The FDA awards this designation to drugs that show good potential in treating rare diseases that often don’t have existing treatments
  • The ODD will provide Kazia with benefits such as up to seven years exclusivity, as well as remove fees associated with a marketing application, potentially saving the company more than US$3 Million
  • Kazia has already received ODD for paxalisib on two other types of brain cancer
  • KZA shares are up 2.7 per cent, trading at 76 cents at 3:50 pm AEST.

Kazia Therapeutics’ (KZA) paxalisib drug has been awarded Orphan Drug Designation for treating a rare type of childhood brain cancer, by the US Food and Drug Administration (FDA).

The Orphan Drug Designation (ODD) is for paxalisib to treat atypical rhabdoid/teratoid tumours (AT/RT) – an ‘orphan’ type of aggressive brain cancer found in children.

An Orphan disease is one that is considered rare, with less than 200,000 cases annually in the United States.

The FDA awards ODD to drugs that show good potential in treating these rare diseases, providing incentives such as up to seven years exclusivity, as a motivator to develop the drugs for diseases that have less treatments available.

Receiving Orphan Drug Exclusivity (ODE), for Kazia will mean that other drug developers won’t be able to use Kazia’s data to recreate generic versions of paxalisib.

It will also remove fees associated with a marketing application, which are over US$3 million (A$4.3 million) in FY22, and provide opportunities for grant funding and protocol assistance, and tax credits.

This is the third time paxalisib has been granted ODD, after it received the designation for malignant glioma which is a type of brain cancer affecting adults, and diffuse intrinsic pontine glioma (DIPG) which affects children.

Kazia CEO Dr James Garner said brain cancer in children has become an “important area of focus for the paxalisib program.”

“We have been working for some years with several world-leading researchers in DIPG, one of the most aggressive childhood cancers.

“Recent data presented at the AACR conference by Dr Jeffery Rubens and colleagues
from Johns Hopkins Medical School has shown the potential of the drug to also add benefit in AT/RT, another form of childhood brain cancer that is very poorly served by existing treatments.

“This represents an important new opportunity for paxalisib, and one that we continue to explore enthusiastically with our collaborators and advisors.”

KZA shares were up 2.7 per cent, trading at 76 cents at 3:50 pm AEST.

KZA by the numbers
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