FDA awards Fast Track Designation to Paxalisib for newly diagnosed glioblastoma
The United States Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) to Kazia's paxalisib (formerly GDC-0084) for the treatment of glioblastoma, the most common and most aggressive form of primary brain cancer.The Kazia Therapeutics Limited an Australian oncology-focused biotechnology company has been granted the approval for the same.
Key Points
Fast Track Designation (FTD) is designed to expedite development of pharmaceutical products which demonstrate the potential to address unmet medical needs in serious or life-threatening conditions
FTD provides Kazia with substantially enhanced access to FDA, including opportunities for face-to-face meetings and written consultation throughout the remaining development of paxalisib
Drugs with FTD are eligible to apply for Accelerated Approval and Priority Review at the time of a New Drug Application (NDA) submission, which may result in faster product approval .
FTD also allows for 'rolling review', whereby Kazia may submit completed sections of the paxalisib NDA as they become available, rather than at the end of development
Kazia
consequently plans to begin initial preparatory activities for NDA filing for
paxalisib in CY2021
Kazia CEO, Dr James Garner, commented, "in awarding Fast Track Designation
to paxalisib, FDA has recognised the drug's potential to meaningfully improve
outcomes for patients with glioblastoma. This is a very powerful acknowledgement.
The opportunities that Fast Track Designation creates, as we move towards an
NDA filing, are of great value and have the potential to substantially
accelerate the commercialisation of paxalisib. In particular, the 'rolling
review' process enables Kazia to complete and submit substantial sections of
our NDA filing in advance, saving time and reducing risk for the product. We
look forward to working closely with FDA as we move into the final stage of
development for paxalisib."
The
specific indication for which FTD has been approved is "for the treatment
of patients with newly diagnosed glioblastoma with unmethylated
O6-Methylguaninemethyltransferase (MGMT) promotor status who have completed
initial radiation with concomitant temozolomide." This language precisely
reflects the patient population studied in the ongoing phase II study, and is
the primary proposed population for the GBM AGILE pivotal study, and is the
intended indication at commercial launch.
Kazia announced on 7 August 2020 that FDA had granted paxalisib Rare Pediatric
Disease Designation (RPDD) for DIPG, an aggressive childhood brain cancer. For
clarity, this granting of FTD for glioblastoma is not specifically connected to
the prior granting of RPDD in DIPG.
Fast Track Designation
Introduced
under the FDA Modernization Act (1997), Fast Track Designation (FTD) may be
awarded by FDA to investigational drugs which treat a serious or
life-threatening condition, and which fill an unmet medical need. FDA notes
that 'the purpose [of the Fast Track program] is to get important new drugs to
the patient earlier.'[1] FTD must be requested by the sponsor company and must
be accompanied by a detailed review of both preclinical and clinical data. To
be awarded FTD, drugs must generally be able to show some potential advantage
over existing therapies, either in terms of safety or efficacy.
The key benefits of FTD comprise enhanced access to FDA, with regular and more
frequent opportunities for consultation and discussion. In addition, drugs with
FTD may be eligible for Accelerated Approval, in which a new medicine is
approved prior to the availability of definitive data, and Priority Review, in
which the standard 12-month review process is reduced to six months. Drugs with
FTD may also enter a 'rolling review' of their NDA submission, in which
sections are submitted and reviewed as they become available, substantially
expediting the approval process.
Next Steps
Kazia completed recruitment to its phase II clinical trial of paxalisib in newly diagnosed glioblastoma in February 2020, and interim clinical data was presented at the American Association of Cancer Research (AACR) Virtual Annual Meeting II in June 2020. Overall survival was calculated at 17.7 months, which compares favourably to a historical figure of 12.7 months for temozolomide, the existing FDA-approved standard of care.
Kazia expects to present further data from this study in 2H CY2020, and to conclude the study in early CY2021.
Paxalisib
has been selected to join the international GBM AGILE pivotal study in
glioblastoma, and recruitment is expected to begin in 2H CY2020.