SYDNEY, Aug. 24, 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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