US FDA Awards Orphan Drug Designation (ODD) To Paxalisib For Malignant Glioma, Including DIPG

Rhea-AI Summary

Kazia Therapeutics Limited (KZIA) has received Orphan Drug Designation (ODD) from the FDA for its drug paxalisib, targeting malignant glioma, including Diffuse Intrinsic Pontine Glioma (DIPG), a critical childhood brain cancer. This designation follows the Rare Pediatric Disease Designation awarded earlier in August 2020. ODD grants up to seven years of exclusivity and financial benefits, including fee waivers for new applications. Initial data from a phase I study in DIPG is anticipated in the latter half of 2020, with further data on glioblastoma expected at an upcoming conference.

Positive

• FDA granted Orphan Drug Designation (ODD) to paxalisib for malignant glioma, enhancing its market position.
• This designation provides potential seven years of Orphan Drug Exclusivity (ODE) and financial incentives like fee waivers.

Negative

• None.

SYDNEY, Aug. 23, 2020 /PRNewswire/ -- Kazia Therapeutics Limited (ASX: KZA; NASDAQ: KZIA), an Australian oncology-focused biotechnology company, is pleased to announce that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Kazia's paxalisib (formerly GDC-0084) for the treatment of malignant glioma, which includes Diffuse Intrinsic Pontine Glioma (DIPG), a rare and highly aggressive childhood brain cancer.

Key Points

• Orphan Drug Designation (ODD) is a special status accorded to drugs which are considered promising potential treatments for rare ('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
• Receipt of ODD follows award of Rare Pediatric Disease Designation (RPDD) for DIPG on 7 August 2020

Kazia CEO, Dr 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."

He added, "This award of ODD concludes a program of regulatory optimisation that Kazia has initiated for paxalisib over the past six months. As we orient paxalisib towards commercialization, these special designations from FDA will allow us to move forward in the swiftest and most effective way possible."

Orphan Drug Designation

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. In addition, drugs with ODD may be eligible for orphan grants by FDA.

Kazia previously received ODD for paxalisib in glioblastoma in February 2018.

Summary of Paxalisib Regulatory Status

	Glioblastoma Most common and most aggressive adult brain cancer	DIPG Highly aggressive childhood brain cancer
Orphan Designation	February 2018	August 2020
Fast Track Designation	August 2020
Rare Pediatric Disease Designation	n/a	August 2020

Next Steps

Kazia expects to present further data from its ongoing phase II study of paxalisib in glioblastoma at the Society for Neuro-Oncology (SNO) Annual Meeting in November 2020.

Initial efficacy data from the ongoing phase I study of paxalisib in DIPG at St Jude Children's Research Hospital is expected during 2H CY2020. Precise timing remains uncertain due to pandemic-related disruption in conference schedules, but Kazia expects to provide an update to investors at the earliest opportunity.

Paxalisib has been selected to join the international GBM AGILE pivotal study in glioblastoma, and recruitment is expected to begin in 2H CY2020.

 

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SOURCE Kazia Therapeutics Ltd

FAQ

What is Kazia Therapeutics' stock symbol?

Kazia Therapeutics is listed under the stock symbol KZIA.

When did Kazia receive Orphan Drug Designation for paxalisib?

Kazia received Orphan Drug Designation for paxalisib on August 23, 2020.

What does Orphan Drug Designation mean for paxalisib?

Orphan Drug Designation means paxalisib is recognized as a promising treatment for a rare disease, providing benefits like market exclusivity and financial support.

What is the significance of the FDA's designation for Kazia?

The FDA's designation is crucial for Kazia as it enhances the drug's commercial viability and provides financial incentives for development.

What are the next steps for Kazia following the ODD award?

Kazia plans to present data from ongoing studies of paxalisib in glioblastoma and DIPG, with updates expected in late 2020.