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

Rhea-AI Summary

Kazia Therapeutics Limited (ASX: KZA; NASDAQ: KZIA) has received Orphan Drug Designation (ODD) from the US FDA for paxalisib, aimed at treating malignant glioma, including the aggressive childhood cancer DIPG. This designation, following the Rare Pediatric Disease Designation received on August 7, 2020, provides several incentives such as up to seven years of exclusivity and potential grant funding. CEO Dr. James Garner emphasized that these approvals will expedite paxalisib's commercialization and development processes, with initial efficacy data expected in late 2020.

Positive

• FDA granted Orphan Drug Designation (ODD) for paxalisib, enhancing its development prospects.
• ODD offers up to seven years of exclusivity, which strengthens competitive positioning.
• Kazia expects to present initial efficacy data from the phase I study of paxalisib in DIPG in the latter half of 2020.

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 the significance of the FDA granting Orphan Drug Designation to KZIA?

The Orphan Drug Designation for paxalisib allows KZIA to benefit from exclusive marketing rights and potential financial incentives, which are crucial for developing treatments for rare diseases.

When did KZIA receive Orphan Drug Designation for paxalisib?

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

What diseases does paxalisib target?

Paxalisib targets malignant glioma, including Diffuse Intrinsic Pontine Glioma (DIPG), a rare and aggressive childhood brain cancer.

What are the next steps for KZIA after receiving Orphan Drug Designation?

KZIA plans to present initial efficacy data from the ongoing phase I study of paxalisib in DIPG and continue its phase II study in glioblastoma.

What financial benefits does the Orphan Drug Designation provide for KZIA?

The designation includes a waiver of nearly $3 million in New Drug Application fees and potential eligibility for orphan grants.